Biden has touted his signing of the Inflation Reduction Act, legislation that lets Medicare set prices for some small-molecule drugs 9 years after FDA approval, versus 13 years for biologics. The law has drawn criticism—and litigation—from several biopharma giants. They contend that the law hinders drug development. Georgetown University Law Center’s O’Neill Institute for National and Global Health Law tracks 14 lawsuits seeking to overturn the measure—two each by AstraZeneca, Bristol Myers Squibb, and Janssen Pharmaceuticals (Johnson & Johnson); and one each by Astellas Pharma, Boehringer Ingelheim, Merck & Co., Novartis, and Novo Nordisk.

Trump points to his 2020 signing of executive orders that directed federally qualified health centers to pass along insulin and epinephrine discounts to low-income Americans; allowed importation of prescription drugs from Canada and other countries where prices for identical drugs were lower; barred discount-reducing agreements between drug manufacturers and pharmacy benefit manages; and ensured that the United States paid the lowest price available among economically advanced countries for Medicare Part B drugs.

A year after Deloitte pegged the average cost of developing a new drug at approximately $2.3 billion among the top 20 global biopharma companies it studied, there’s no reason to think it will fall significantly—and thus no reason to expect a drip in drug prices soon.

Below is GEN’s updated A-List of top 10 best-selling prescription drugs based on 2023 sales. This year marks the first time in the 11 years that GEN has compiled such rankings that AbbVie’s Humira (adalimumab) didn’t finish on top. Humira led GEN’s yearly best-selling drug lists since the first list published in 2013, when the drug generated $10.66 billion. That translates to $14.346 billion in today’s dollars, which means Humira’s sales grew only 0.4% over the past decade when inflation is accounted for.

Humira showed its first year-over-year sales decline in 2019, reflecting direct biosimilar competition in Europe and other countries that shrank the drug’s 2019 sales beyond the United States by 31% from 2018. Last year, Humira saw its first biosimilar competition in the United States, starting with Amgen’s Amjevita (adalimumab-atto), which generated $626 million in 2023 product sales.

Top-selling drugs are ranked based on sales or revenue reported for 2023 by biopharma companies in press announcements, annual reports, investor materials, and/or conference calls. Each drug is listed by name, sponsor(s), 2023 sales, 2022 sales, and the percentage change between both years.

Seven of last year’s 10 top-selling drugs registered year-over-year sales gains, with 5 of the 7 racking up double-digit increases. The rest of the list saw declines in sales from 2023, typically reflecting the launch of generic or biosimilar competitors, plus insurer discounts.

The total aggregate value of the top 10 best-selling drugs last year was $145.495 billion, down 27% from $198.073 billion in 2022, when two COVID-19 vaccines and one antiviral drug to treat the virus contributed to a sales spike that accounted for nearly half (47%) of total sales. Only one of the COVID-19 vaccines generated enough in sales to make last year’s top 10. However, the 2023 top 10 generated 44% more in sales than the $101.15 billion racked up in 2019 for the top 10 drugs listed by GEN in March 2020, as the pandemic wreaked havoc on the world.

Ranking number 11 through number 15 in 2023 were treatments that generated between $7.1 billion and $9.7 billion in revenues:

• Darzalex/Darzalex Faspro (daratumumab) from Janssen Biotech (Johnson & Johnson).
• Trikafta/Kaftrio (elexacaftor/tezacaftor/ivacaftor and ivacaftor) from Vertex Pharmaceuticals.
• Gardasil/Gardasil 9 (Human Papillomavirus Quadrivalent (Types 6, 11, 16, and 18) Vaccine, Recombinant/Human Papillomavirus 9-valent Vaccine, Recombinant) from Merck & Co.
• AbbVie’s Skyrizi (risankizumab‐rzaa) from AbbVie.
• Trulicity (dulaglutide) from Eli Lilly and Company.

References

1. Includes sales of Eylea^® HD, which won FDA approval in August 2023, and Eylea. In 2023, Eylea HD generated $165.8 million in U.S. sales.

2. Sanofi records global net product sales of Dupixent, with each company recording its half-share of profits on global sales of the drug.

3. Figure includes product revenue for both companies and royalty revenue disclosed by Ono.

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One way to meet this need, she suggested, would be to expand delivery options from ex vivo approaches to in vivo approaches. With ex vivo treatments, it is necessary to remove and replace cells or suppress the immune system. With in vivo treatments, it is possible for treatments to be administered completely inside the patient’s body. Accordingly, in vivo treatments are more convenient for patients and healthcare providers alike—so convenient, in fact, that their administration is practical at healthcare facilities of all kinds, not just large urban academic hospitals.

As reported by Alvin Powell in the Harvard Gazette, Doudna offered the following observation:

“[Figuring out how] we can achieve in vivo genome editing … is the bottleneck in this field. Broadly speaking, what we need to be addressing is how these editors are going to get into target cells in the body. It’s a really interesting, really big challenge, and there are many people working on it.”

Among them are Harvard Medical School researchers at the Massachusetts Eye and Ear Hospital who recently led a successful proof-of-concept study for a gene editing therapy. In the Phase I/II BRILLIANCE trial (NCT03872479), 11 of 14 Leber congenital amaurosis patients who were treated with an in vivo CRISPR-Cas9-based therapy, Editas Medicine’s EDIT-101, showed measurable improvement on at least one key vision test. Six showed improvement on two or more vision outcomes.

But at prices exceeding $1 million, gene editing therapies have been administered to only 250 people. Institutions working to raise that number include the companies in this A-List, which highlights the top 10 public developers of gene editing therapies or of platforms enabling development of therapies that apply editing technologies. Some of the listed have had success with ex vivo therapies; others, such as Editas, report progress with in vivo therapies.

The companies have been ranked on a composite scale based on:

•  Portfolio: Number of approved treatments and clinical programs. (Portfolio assessments considered how far the clinical programs had advanced).
•  Cash position: Cash, cash equivalents, and marketable securities, as disclosed by the companies in regulatory filings and press releases.
•  Market capitalization: Product of the share price and the number of outstanding shares.

When this A-List was prepared, information about 2023 revenues wasn’t available for all of the companies we considered for inclusion. Indeed, revenue figures were available for only about half of the companies that ultimately made the list. We report the revenue figures we obtained even though we didn’t rely on them to determine company rankings.

Just missing the list at No. 11 was metagenomics-driven gene editing toolbox developer Metagenomi (MGX). Metagenomi went public in February, raising $81.1 million in net proceeds, providing a cash runway to support two IND applications and two additional development candidate nominations. At deadline, none of Metagenomi’s 13 programs were in the clinic. But in April, Moderna terminated a 2-1/2-year collaboration of undisclosed value to co-develop Metagenomi’s gene editing technologies, including base editors and RNA-mediated integration systems, helping shrink a stock whose value has tumbled 47% from its 52-week high of $12.74 on February 15, to $6.80 on May 8.

1. CRISPR Therapeutics (CRSP)
CRISPR Therapeutics cemented its gene editing leadership last December when it and partner Vertex Pharmaceuticals received the FDA’s first approval for a CRISPR-based gene edited therapy, Casgevy (exagamglogene autotemcel or “exa-cel”), for sickle cell disease. The following month, the FDA approved Casgevy to treat transfusion-dependent β-thalassemia. Headquartered in Zug, Switzerland, CRISPR Therapeutics ranked second in 2023 revenue with $201.206 million (adding only $504,000 in Q1) but first in all other criteria, from clinical activity (five programs, two of which are in Phase I/II trials) to market cap ($4.468 billion) to cash position ($1.694 billion).

2. Beam Therapeutics (BEAM)
Beam Therapeutics said on May 7 that it expects to treat 45 patients in the expansion cohort of its BEACON Phase I/II trial assessing co-lead candidate BEAM-101 in severe sickle cell disease, after successfully completing sequential dosing and engraftment for the three-patient sentinel cohort. Beam expects to report data from “multiple” patients in the BEACON trial in the second half of this year. Based in Cambridge, MA, Beam led gene editing companies in 2023 revenue with $377.709 million (it added $7.41 million in Q1) and was second in cash position with $1.19 billion (dipping to $1.095 billion in Q1). Beam finished third in market cap with $1.872 billion, but seventh in clinical progress (two programs, both Phase I/II).

3. Intellia Therapeutics (NTLA)
Intellia Therapeutics is enrolling patients in its pivotal Phase III MAGNITUDE trial (NCT06128629) assessing in vivo CRISPR-based candidate NTLA-2001 (Intellia leads development, with partner Regeneron Pharmaceuticals) in amyloidogenic transthyretin (ATTR) amyloidosis with cardiomyopathy. The first patients were dosed in March. Intellia plans to launch by year’s end two other Phase III trials—one evaluating NTLA-2001 in hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN), the other investigating NTLA-2002 in hereditary angioedema. Cambridge, MA-based Intellia is second in both clinical progress (two programs, including Phase I/II and long-term studies for NTLA-2002) and market cap ($2.319 billion), and it is third in cash position ($1.012 billion, sinking 6% to $953.384 million in Q1). It finished ninth in 2023 revenue with $36.275 million (adding $28.935 million in Q1).

4. Editas Medicine (EDIT)
Editas Medicine said on May 8 that it completed enrollment of the adult cohort of the Phase I/II/III RUBY trial (NCT04853576) assessing renizgamglogene autogedtemcel (reni-cel, formerly EDIT-301) in sickle cell disease, with “substantive” clinical data to be released later this year. Reni-cel is also under study in transfusion-dependent β-thalassemia in the Phase I/II EdiTHAL trial (NCT05444894), with data also set for release later this year. Based in Cambridge, MA, Editas scored fourth in clinical progress (both reni-cel programs), fifth in revenue ($78.123 million), sixth in cash position ($427.135 million, sliding 12% to $376,776 in Q1), but eighth in market cap ($451.469 million).

5. Wave LifeSciences (WVE)
Wave Life Sciences said on May 9 that it expects to deliver proof-of-mechanism data later this year from its Phase Ib/IIa RestorAATion-2 (NCT06405633) trial now underway, part of a clinical program assessing WVE-006 as a first-/best-in-class treatment for α1-antitrypsin deficiency. WVE-006 is a GalNAc-conjugated, subcutaneously delivered RNA editing oligonucleotide for which GSK has an exclusive global license. Singapore-registered Wave, whose U.S. headquarters is in Cambridge, MA, is fourth in both 2023 revenue ($113.305 million, adding $12.538 million in Q1) and market cap ($735.749 million), but ninth in both cash position ($200.351 million, declining 10% to $180.922 million in Q1) and clinical progress (one editing program, two trials including Phase I RestorAATion-1 [NCT06186492] and two nonediting programs).

6. Caribou Biosciences (CRBU)
Caribou Biosciences expects by year’s end to launch its Phase I GALLOP trial assessing CRB-010 in lupus nephritis and extrarenal lupus, reflecting expanded clinical development for the allogeneic anti-CD19 chimeric antigen receptor T-cell therapy with a PD-1 knockout. CRB-010 has shown encouraging initial safety and efficacy in patients with relapsed or refractory B-cell non-Hodgkin’s lymphoma. Co-founded by CEO and president Rachel Haurwitz, PhD, and Nobel laureate Jennifer Doudna, PhD, Berkeley, CA-based Caribou tied for 5th in the clinic (three programs, all in Phase I), and placed 5th in cash position ($328.827 million), but finished 9th in market cap ($331.918 million) and 10th in revenue ($34.477 million).

7. Verve Therapeutics (VERV)
Verve Therapeutics in April voluntarily paused an early-stage clinical trial of VERVE-101—the first base editing treatment to reach the clinic—after one patient showed an elevated level of a liver enzyme and a low platelet count. Investors sent shares tumbling 35%, but analysts were far more forgiving. The setback came five months after Verve enthusiastically presented the first human proof-of-concept data for the in vivo base editing therapy. Boston-based Verve placed 8th in clinical progress (two programs, both Phase Ib) and just 11th in 2023 revenue ($11.758 million, adding $5.695 million in Q1) but is 4th in cash position ($623.95 million) and 6th in market cap ($546.615 million).

8. Poseida Therapeutics (PSTX)
Poseida Therapeutics promised investors a renewed focus on its genetic medicine portfolio, as well as advancements in its emerging platform technologies. Among these is its Cas-CLOVER high-fidelity nuclease for enabling clean site-specific gene editing. Poseida uses Cas-CLOVER in manufacturing its three clinical programs (all Phase I, tied for fifth). Two are partnered with Roche—P-BCMA-ALLO1 for relapsed/refractory multiple myeloma, and P-CD19CD20-ALLO1 for B-cell malignancies—and the other is wholly owned P-MUC1C-ALLO1 for multiple solid tumor indications. San Diego-based Poseida also ranks 6th in revenue ($64.703 million), 8th in cash position ($202.202 million), and 11th in market cap ($260.252 million).

9. Sangamo Therapeutics (SGMO)
Sangamo Therapeutics showcased its Modular Integrase (MINT) platform and other next-generation genome engineering capabilities at the recent American Society of Gene and Cell Therapy annual meeting and in a bioRxiv preprint (“Systematic Development of Reprogrammed Modular Integrases Enables Precise Genomic Integration of Large DNA Sequences”). MINT is a protein-guided genome editing method designed to integrate large sequences of DNA into the genome. A pioneer in zinc finger nuclease editing, Richmond, CA-based Sangamo is third in clinical progress with three programs, (including TX200, a gene edited cell therapy to prevent kidney transplant rejection, in Phase I/II) and third in revenue ($176.232 million, adding only $481,000 in Q1). It is 12th in market cap ($107.4 million) and 13th in cash position ($81.002 million, tumbling 33% to $54.417 million in Q1).

10. Prime Medicine (PRME)
Prime Medicine is preparing to launch the first human trials of a prime editing therapy after the FDA cleared the company’s IND application in April for its first clinical candidate. PM359, designed to target the p47phox variant of chronic granulomatous disease, consists of autologous hematopoietic stem cells modified ex vivo using prime editors designed to correct a high percentage of cells containing the disease-causing mutation. The FDA clearance sparked a 27% surge in Prime’s shares, lifting its market cap to fifth with $682.965 million. Based in Cambridge, MA, Prime ranks 10th in clinical progress (PM359’s Phase I/II trial) and 11th in cash position ($121.665 million).

Don’t forget to read GEN’s Top Five Privately Held Gene Editing Therapy Companies.

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It’s a testament to the confidence in gene editing therapy developers shared by venture capital investors that the next five private companies below the top five raised nearly $1 billion in combined capital ($874 million, to be precise). Ranked 6th to 10th were Tome Biosciences ($213 million); Aera Therapeutics ($193 million); EdiGene (RMB 1.182 billion [$164 million]); Tune Therapeutics ($160 million); and SparingVision (€135 million [$144 million]).

1. Tessera Therapeutics
Tessera Therapeutics, a Flagship Pioneering company, has raised approximately $600 million toward developing genetic medicines. Tessera’s Gene Writing technology uses mobile elements called non–long nonterminal repeat retrotransposons, which write genes into the genome through a method that entails binding RNA, binding DNA, nicking DNA, and then priming reverse transcription. “We were surprised to discover that almost no one had thought about how they could be adapted as tools for genome engineering,” Tessera’s co-founder and chief innovation officer Jacob (Jake) Rubens, PhD, told GEN in 2022. Last October, the Galien Foundation awarded its Prix Galien USA 2023 to Somerville,
MA-based Tessera in the Best Startup category.

2. Mammoth Biosciences
Mammoth Biosciences’ CRISPR-based ultracompact gene editing platform has helped it raise $465 million—about $365 million in venture capital and $100 million-plus in equity and nondilutive capital—as well as interest from biopharma partners such as Bayer and Vertex Pharmaceuticals. Regeneron Pharmaceuticals joined them in April, agreeing to combine its delivery antibodies with Mammoth’s platform to develop CRISPR-based gene edited therapies. Regeneron is paying Brisbane,
CA-based Mammoth a $95 million equity investment and a $5 million upfront payment, plus up to $370 million in milestone payments per target developed. Mammoth’s co-founders include CRISPR pioneer and Nobel laureate Jennifer Doudna, PhD.

3. Arbor Biotechnologies
Arbor Biotechnologies expanded its toolbox of novel, next-generation editors on May 8 by acquiring Serendipity Biosciences. Arbor, which has raised $335 million, said the deal expands its potential therapeutic applications in reverse transcriptase–based editing and insertion of large DNA regions. Serendipity’s editing technologies were discovered in the laboratory of Arbor co-founder and CRISPR pioneer Feng Zhang, PhD. Cambridge, MA-based Arbor’s pipeline includes five wholly owned preclinical treatments for liver and central nervous system disorders. On May 9, at the American Society of Gene and Cell Therapy annual meeting, Arbor presented data supporting clinical development of its lead candidate, ABO-101, a therapy for primary hyperoxaluria type 1.

4. Scribe Therapeutics
Scribe Therapeutics has attracted $120 million in venture capital and $140 million in upfront payments from biopharma partners that include Prevail Therapeutics (an Eli Lilly and Company subsidiary), Biogen, and Sanofi. Headquartered in Alameda, CA, Scribe develops CRISPR-based treatments through genetic modification platforms designed to build and apply the company’s suite of CRISPR technologies in hematopoietic disorders and other therapeutic areas, including neurodegenerative diseases; ophthalmological diseases; and multisystem, muscle, and metabolic disorders. Scribe’s co-founders include CRISPR pioneer and Nobel laureate Jennifer
Doudna, PhD.

5. Chroma Medicine
Chroma Medicine has raised $257 million in capital toward developing programmable epigenetic editors that precisely turn genes on or off, or that can alter the expression of several genes at once. By seamlessly silencing, activating, and multiplexing genes in a single platform intended to mimic the cell’s innate mechanisms for controlling gene expression, Boston-based Chroma’s epigenetic technology can become a leading approach for gene regulation. A Chroma co-sponsored study (Cappelluti et al. Nature 2024; 627:416–423) showed that lipid nanoparticle delivery of the company’s engineered transcriptional repressors (editor-encoding mRNAs) to the liver of mice led to epigenetic silencing of the Pcsk9 gene for nearly one year.

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Gene therapies that employ in vivo gene editing technology look especially promising now that the first such therapy, CRISPR-based Casgevy (exagamglogene autotemcel), has secured FDA approval. (In December 2023, it was approved for sickle cell disease; in January 2024, it was approved for beta-thalassemia.) According to Goldman Sachs analysts, peak-year sales for Casgevy are projected to reach $3.9 billion.

Another encouraging development is the success of Sarepta Therapeutics’ Elevidys (delandistrogene moxeparvovec-rokl), the first gene therapy indicated for Duchenne muscular dystrophy. It generated just over $200 million in net product revenue last year, its first year on the market, despite being available to patients only since the summer.

During Sarepta’s Q4 2023 quarterly earnings call, Dallan Murray, the company’s executive vice president and chief customer officer, told analysts, “The success of Elevidys shows that gene therapy can be commercially viable, providing hope for those patients with Duchenne, and for all those with genetic conditions and unmet needs.” He added that Elevidys performed the best of the six gene therapies launched since mid-2022.

Of these six, the second-best performer in terms of revenues was Krystal Biotech’s Vyjuvek (beremagene geperpavec-svdt), which made $50.7 million in 2023—too little to be among the top 10 therapies in this A-List, which ranks cell and gene therapies by 2023 revenues as disclosed by the sponsoring companies in regulatory filings, press releases, financial statements, or quarterly earnings calls.

Farther beyond the top 10, the sales figures drop off considerably. Bluebird Bio’s Zynteglo (betibeglogene autotemcel, “beti-cel”) made $16.7 million; Bluebird’s Skysona (elivaldogene autotemcel, “eli-cel”), $12.4 million; BioMarin Pharmaceutical’s Roctavian (valoctocogene roxaparvovec-rvox, “val-rox”), $3.5 million. Even lower was a cell therapy launched last year, Gamida Cell’s cell therapy Omisirge (omidubicel-onlv), which finished 2023 generating just $1.784 million.

Of the 12 gene therapies on the market in the United States as of December 31, 2023, only 7 had sales figures publicly available. Of the other five, sales figures for four of them were not disclosed by their sponsors: Ferring Pharmaceuticals’ Adstiladrin (nadofaragene firadenovec-vncg); CSL Behring’s Hemgenix (etranacogene dezaparvovec-drlb); BioVex’s (Amgen’s) Imlygic (talimogene laherparepvec); and PTC Therapeutics’ Upstaza (eladocagene exuparvovec). The fifth gene therapy, Bluebird Bio’s Lyfgenia (lovotibeglogene autotemcel, “lovo-cel”) had yet to generate revenue last year, though Bluebird said on March 26 that it expects to recognize revenue from its first infusion of Lyfgenia in the third quarter of this year.

This A-List does not include antisense oligonucleotides (ASOs) even though therapies of this class are sometimes referred to as gene therapies. Our rationale is that ASOs modulate RNA instead of manipulating or inserting genetic DNA. Another reason: ASOs are not included in the FDA’s list of Approved Celllular and Gene Therapy Products. However, if this A-List had included ASOs, we would have listed two treatments from Sarepta Therapeutics for Duchenne muscular dystrophy. Exondys 51 (eteplirsen) and Amondys 45 (casimersen) are for patients who have mutations amenable to exon 51 skipping and exon 45 skipping, respectively. The 2023 sales figures for these therapies are $540.576 million and $273.755 million, respectively.

1. Yescarta (axicabtagene ciloleucel)

2023 Sales: $1.498 billion

Sponsor(s): Kite, a Gilead Company

Type: CD19-directed genetically modified autologous T-cell immunotherapy

Indication(s): Treatment of adults with large B-cell lymphoma that is refractory to first-line chemoimmunotherapy or that relapses within 12 months of first-line chemoimmunotherapy. Also, treatment of adults with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma not otherwise specified, primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma, and diffuse large B-cell lymphoma arising from follicular lymphoma.

Initial FDA Approval Date: October 18, 2017

2. Zolgensma (onasemnogene abeparvovec-xioi)

2023 Sales: $1.214 billion

Sponsor(s): Novartis^a

Type: Adeno-associated virus vector–based gene therapy

Indication(s): Treatment of pediatric patients less than two years of age with spinal muscular atrophy with biallelic mutations in the survival motor neuron 1 gene.

Initial FDA Approval Date: May 24, 2019

3. Kymriah (tisagenlecleucel)

2023 Sales: $508 million

Sponsor(s): Novartis

Type: CD19-directed genetically modified autologous T-cell immunotherapy

Indication(s): Treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia that is refractory or in second or later relapse; adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma not otherwise specified, high grade B-cell lymphoma and diffuse large B-cell lymphoma arising from follicular lymphoma; and adult patients with relapsed or refractory follicular lymphoma after two or more lines of systemic therapy.

FDA Approval Date: August 30, 2017

4. Carvykti (ciltacabtagene autoleucel)

2023 Sales: $500 million

Sponsor(s): Janssen Biotech (Johnson & Johnson) and Legend Biotech

Type: B-cell maturation antigen–directed genetically modified autologous T-cell immunotherapy

Indication(s): Treatment of adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.

Initial FDA Approval Date: February 28, 2022

5. Abecma (idecabtagene vicleucel)

2023 Sales: $472 million

Sponsor(s): Bristol Myers Squibb and 2seventy bio

Type: B-cell maturation antigen–directed genetically modified autologous T-cell immunotherapy

Indication(s): Treatment of adult patients with relapsed or refractory multiple myeloma after two or more prior lines of therapy including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody.

Initial FDA Approval Date: March 26, 2021

6. Tecartus (brexucabtagene autoleucel)

2023 Sales: $370 million

Sponsor(s): Kite, a Gilead Company

Type: CD19-directed genetically modified autologous T-cell immunotherapy

Indication(s): Treatment of adults with relapsed or refractory mantle cell lymphoma. Also, treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia.

Initial FDA Approval Date: July 24, 2020 (accelerated approval)

7. Breyanzi (lisocabtagene maraleucel)

2023 Sales: $364 million

Sponsor(s): Bristol Myers Squibb

Type: CD19-directed genetically modified autologous T-cell immunotherapy

Indication(s): Treatment of adults with large B-cell lymphoma, including diffuse large B-cell lymphoma not otherwise specified (including diffuse large B-cell lymphoma arising from indolent lymphoma), high-grade B-cell lymphoma, primary mediastinal large B-cell lymphoma, and follicular lymphoma grade 3B who have refractory disease to first-line chemoimmunotherapy or relapse within 12 months of first-line chemoimmunotherapy, or refractory disease to first-line chemoimmunotherapy or relapse after first-line chemoimmunotherapy and are not eligible for hematopoietic stem cell transplantation due to comorbidities or age, or relapsed or refractory disease after two or more lines of systemic therapy. Also, treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma who have received at least two prior lines of therapy, including a Bruton’s tyrosine kinase inhibitor and a B-cell lymphoma 2 inhibitor.

Initial FDA Approval Date: February 5, 2021 (accelerated approval)

8. Elevidys (delandistrogene moxeparvovec-rokl)

2023 Sales: $200.356 million

Sponsor(s): Sarepta Therapeutics

Type: Adeno-associated virus vector–based gene therapy

Indication(s): Treatment of ambulatory pediatric patients aged four through five years with Duchenne muscular dystrophy with a confirmed mutation in the Duchenne muscular dystrophy gene.

Initial FDA Approval Date: June 22, 2023 (accelerated approval)

9. MACI (autologous cultured chondrocytes on porcine collagen membrane)

2023 Sales: $164.8 million

Sponsor(s): Vericel

Type: Autologous cellularized scaffold product

Indication(s): Repair of symptomatic, single, or multiple full-thickness cartilage defects of the knee with or without bone involvement in adults.

Initial FDA Approval Date: December 13, 2016

10. Luxturna (voretigene neparvovec-rzyl)

2023 Sales: Approximately CHF 46 million (about $51 million)^b

Sponsor(s): Spark Therapeutics (Roche)

Type: Adeno-associated virus vector–based gene therapy

Indication(s): Treatment of patients with confirmed biallelic RPE65 mutation–associated retinal dystrophy. Patients must have viable retinal cells as determined by the treating physician(s).

Initial FDA Approval Date: December 18, 2017

Footnotes

^a Novartis is the successor to AveXis, which successfully completed the development of Zolgensma in 2019 by receiving FDA approval for the gene therapy. In 2014, AveXis licensed from Regenxbio the AAV9 vector used in the Phase I SMA clinical trial at Nationwide Children’s Hospital. Regenxbio licensed exclusive rights to key intellectual property covering novel recombinant AAV vectors discovered at University of Pennsylvania in the laboratory of James M. Wilson, MD, PhD.

^b Roche has not furnished a 2023 Luxturna sales figure, instead including those sales within the “others” portion of its ophthalmology sales listed in its 2023 annual report, CHF 48 million (about $53 million). That figure is the same as the “others” figure listed in 2022, when the company disclosed CHF 46 million in Luxturna sales.

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In 2024, we will learn if sequencing customers are comfortable enough to switch from Illumina to competing companies. Illumina’s rivals include PacBio (which launched the Onso, a benchtop short-read sequencing platform, at a party featuring Maroon 5 in October 2022); Singular Genomics (which rolled out its G4 Sequencing Platform in late 2021); and Element Biosciences (which announced AVITI Sequencing in March 2022).

“[The newer companies] all have some finite chance of going out of business,” said Alex Dickinson, PhD, a former Illumina senior vice president who is now executive chair of Ryght.AI. “They all have incredible burn rates, and we live in an environment where funding is very scarce.” (This comment, and others, was contributed to a recent GEN roundup of what 2024 holds for NGS and genomics.)

As for Illumina, it continues to double down on the NovaSeq X sequencing system it unveiled in 2022 and commercially launched last year. Illumina says that it received 390 orders for NovaSeq systems in 2023 and shipped 352 to customers, including 79 during the fourth quarter.

“NovaSeq X has been the most successful high-throughput product launch in our history,” Illumina CEO Jacob Thaysen, PhD, declared to analysts February 8 on the company’s Q4 2023 earnings call.

Regardless of how Illumina and its rivals fare this year, NGS has only scratched the surface of its commercial potential, according to a report issued by Research and Markets on March 7. The market research firm projects that the NGS market will more than triple over the next seven years, from $10.63 billion this year to $34.19 billion in 2030—sustaining a compound annual growth rate of 18.16%.

This A-List article presents two lists of sequencing instrument providers. One list includes eight companies that provide NGS instruments. The other includes three companies that provide Sanger instruments. (Since one company, Thermo Fisher Scientific, appears in both lists, the total number of sequencing instrument providers is 10.) Each company is listed by name, followed by a short description of recent company activity, including financial activity from 2023 (either revenue—if it had been disclosed—or total capital raised). Because only some of the instrument providers disclose how much revenue they generate from NGS, they are listed alphabetically, not ranked. (The most recent ranking of NGS companies appeared in 2019.)

Besides companies that provide sequencing instruments, the sequencing industry includes companies that provide workflow solutions. Three workflow giants—Agilent Technologies, QIAGEN, and Roche—appear in their own section of this A-List article. (Again, these companies appear alphabetically because they do not specify what portion of their revenues comes from enabling sequencing.)

PROVIDERS OF NGS INSTRUMENTATION

Element Biosciences

Element Biosciences last month announced a series of partnerships focused on workflows for its AVITI System. With Volta Labs, Element will partner to optimize sequencing throughput by launching Volta’s Callisto Sample Prep System for AVITI. With Twist Bioscience, Element will develop the Twist for Element, Exome 2.0 plus Comprehensive Exome Spike-in Workflow for AVITI, an exome sequencing solution. And with Integrated DNA Technologies, Element has launched a suite of adapters, universal blockers, and library amplification primer mixes designed exclusively for AVITI. Element disclosed preliminary 2023 revenues of $25 million in January at the 42nd Annual J.P. Morgan Healthcare Conference.

Illumina

Through its new CEO, Jacob Thaysen, PhD, the longtime NGS giant has laid out its priorities for 2024, which include boosting its “topline” or revenues by increasing placements of all its instruments—especially the NovaSeq X sequencing system, unveiled in 2022—as well as enhancing its commercial processes and driving more sequencing activity. Thaysen’s appointment in September 2023 capped a turbulent year that saw predecessor Francis deSouza resign after a partially successful proxy campaign by Carl C. Icahn. Illumina finished 2023 with a $1.161 billion net loss, improved from 2022’s $4.404 billion net loss, while revenue slid 2% year-over-year to $4.5 billion.

MGI Tech

MGI Tech—whose U.S. subsidiary is Complete Genomics—announced March 4 that Eurofins Genomics has ordered the DNBSEQ-T20×2 (T20) ultra-high throughput sequencer, along with the genomics data center ZTRON Appliance and numerous MGI lab automation products and systems. Launched in February 2023, the T20 is powered by MGI’s DNBSEQ technology and designed to significantly reduce sequencing costs to below $100 per genome when running 50,000 whole genome sequences per year. The T20 aims to address high-throughput processing needs for various types of sequencing technologies. These include whole genome, whole genome bisulfite, single-tube long fragment read, and single-cell sequencing technologies. The same is true for spatiotemporal omics technologies, such as MGI Tech’s Stereo-seq. China’s sluggish economy has affected MGI, which finished last year with a net loss of RMB 597.1 million (about $83 million), compared with net income of RMB 2.03 billion ($282 million) in 2022.

Oxford Nanopore Technologies

Oxford Nanopore Technologies on March 7 announced the early access launch of the PromethION 2 Integrated (P2i), its all-in-one desktop sequencing device. P2i is designed to facilitate real-time base calling and post run analysis directly within the device, eliminating dependency on external computing resources. In December, Oxford Nanopore promoted improvements in DNA sequencing accuracy powered by machine learning–guided enzyme engineering and improved models. The company reached a record of Q28 (99.8 %) in median simplex single molecule accuracy. Oxford Nanopore finished 2023 with a net loss of £154.5 million ($197.4 million) on revenue of £169.7 million ($216.8 million). However, excluding COVID-19 sequencing and the company’s largest customer, The Emirati Genome Program, “underlying” revenue jumped 39% to £149.7 million ($191.5 million).

PacBio

PacBio launched two new high-throughput library preparation kits and workflows optimized for its Revio sequencing system in February. HiFi Prep Kit 96 and HiFi Plex Prep Kit 96 allow users to automate long-read sequencing workflow steps and streamline preparing, pooling, and loading of samples, which according to PacBio will reduce costs 40% and cut workflow time 60%. HiFi Plex Prep Kit 96 customers can run 1,536 samples in a single Revio run. Delivery of the kits is planned for early in the second quarter. PacBio’s net loss dipped 2% in 2023, to $306.735 million, on revenue that soared 56% year-over-year to $200.521 million.

Singular Genomics

Singular Genomics has unveiled an upgrade to its G4 Sequencing Platform—the G4X Spatial Sequencer, a high-throughput in situ spatial sequencing platform designed for simultaneous direct RNA sequencing, targeted transcriptomics, proteomics, and fluorescent hematoxylin and eosin stain analysis from formalin-fixed, paraffin-embedded tissues. G4X is expected to be available by the end of 2024. Singular also announced upgrades to G4 that include a higher-throughput F4 Flow Cell estimated to produce 600 million–800 million paired reads per flow cell, potentially doubling the G4 sequencer run output to 3.2 billion reads. F4 is expected to be released in the second half of 2024.

Thermo Fisher Scientific

Thermo Fisher Scientific’s NGS technology carries the Ion Torrent name. Last year, Thermo Fisher launched two new NGS-based research tools for preimplantation genetic testing-aneuploidy (PGT-A). (This type of testing is used for in vitro fertilization and intracytoplasmic sperm injection.) The Ion ReproSeq PGT-A Kit and the Ion AmpliSeq Polyploidy Kit are the first research-use reproductive health assays available on the company’s Ion Torrent Genexus Integrated Sequencer, and they are designed to deliver complete workflows from sample to aneuploidy analysis result. Also last year, Thermo Fisher began partnering with Pfizer to expand access to NGS-based cancer testing for patients in 30-plus countries across Latin America, Africa, the Middle East, and Asia. Thermo Fisher’s sequencing business is within the Genetic Sciences business of its Life Sciences Solutions segment, which generated 23% ($9.977 billion) of the company’s $42.86 billion in 2023 revenue.

Ultima Genomics

Ultima Genomics last month launched the UG 100 system, which has an ultra-high-throughput sequencing architecture that features an open silicon wafer instead of multiple traditional flow cells. This architecture, Ultima says, will reach and even break through the barrier known as the $100 genome. The UG 100 includes full 24/7 run automation, flexibility for smaller and significantly faster runs, high accuracy for germline applications, and an extreme accuracy mode for somatic applications and rare event detection. According to Ultima, the UG 100’s new ppmSeq technology provides industry-leading raw read accuracy for calling single nucleotide variants for rare event detection applications such as detecting low-level circulating tumor DNA. Privately held Ultima emerged from stealth in 2022 after raising approximately $600 million.

PROVIDERS OF SANGER SEQUENCING INSTRUMENTATION

Hitachi High-Tech

Hitachi High-Tech last month joined Sysmex to announce that the companies agreed to partner on developing genetic testing systems based on capillary electrophoresis (CE) sequencers. Sysmex and Hitachi High-Tech aim to develop more efficient genetic testing systems at a lower cost, and to expand and optimize genetic testing for individual diseases. Hitachi High-Tech will pursue approval for CE sequencers as medical devices while Sysmex will seek development and regulatory approval for testing reagents for use with such devices, plus develop analysis software. Hitachi markets the Compact CE Sequencer DS3000, which applies CE technology developed by Hitachi High-Tech over several decades in a compact system designed to facilitate sequencing analysis and fragment analysis.

Promega

Promega markets the Spectrum Compact CE system, a benchtop
instrument designed for Sanger sequencing analysis. Spectrum Compact supports Sanger sequencing applications for verification of NGS base calls. It is designed to confirm successful genome edits in transformed cultures and to screen secondary clones for successful CRISPR-Cas9 edits. Spectrum Compact CE is designed for use with existing sequencing chemistries using fluorescently labeled dideoxynucleotide triphosphates and 4-, 5-, and 6-dye short tandem repeat kits from Promega, and other commercially available kits. Spectrum Compact CE can also be used for DNA fragment analysis for forensic labs.

Thermo Fisher Scientific

In addition to its NGS tools and technology, Thermo Fisher Scientific markets genetic analyzers and workflow tools for Sanger sequencing under the Applied Biosystems brand. Applied Biosystems’ SeqStudio Series systems are designed to analyze nucleic acids using CE with Sanger sequencing or fragment analysis and to enable applications that range from simple, targeted sequencing to identification of SARS variants of concern. The most recent instrument in the series, Applied Biosystems’ SeqStudio Flex Series Genetic Analyzer, was introduced in 2022. Thermo Fisher’s sequencing business is within the Genetic Sciences business of its Life Sciences Solutions segment, which generated 23% ($9.977 billion) of the company’s $42.86 billion in 2023 revenue.

PROVIDERS OF WORKFLOW SOLUTIONS

Agilent Technologies

Speaking with analysts last November, CEO Mike McMullen quantified Agilent’s genomics business at $500 million—part of the Diagnostics and Genomics Group, which generated $1.409 billion last year, up 1% from 2022. Roughly half of the genomics business relates to sample preparation chemistries, such as Agilent’s SureSelect. The other half enables quality assurance and quality control of NGS samples to validate quality prior to sequencing.

QIAGEN

QIAGEN credits growth in its portfolios of universal NGS solutions for use with third-party NGS systems with a 6% boost in sales (to $239 million) of the company’s Genomics/NGS business in 2023.

Roche

Sequencing investment was among “significant areas of spending,” Roche stated in its Finance Report 2023, without furnishing figures. Roche Sequencing Solutions is within Roche’s Diagnostics Division, which last year generated CHF 14.1 billion ($16.1 billion) in revenue, down 20.5% from 2022 due to lower sales of COVID-19-related tests.

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Employment in biopharma and the rest of the life sciences industry grew by just 0.1% between August 2023 and November 2023. Nonetheless, it grew an even 2% for the period between January 2023 and November 2023, as indicated in a quarterly report issued on February 1 by CBRE, a commercial real estate firm.¹ That’s slightly above the 1.8% increase in total nonfarm payroll growth for the same period, according to the U.S. Bureau of Labor Statistics (BLS).

In its more expansive 2024 U.S. Life Sciences Outlook, CBRE noted that life sciences job growth had slowed to an average of 2,291 jobs per month since July 2022.² This figure was down more than three-fourths from the corresponding figure for 2021, which was 10,000-plus jobs per month. It should be noted that in 2021, many companies were still scrambling to develop COVID-19 drugs and vaccines—and many smaller biotechnology companies, flush with venture capital (VC), were expanding their workforces and operations.

Some further signs of concern for the industry: October 2023 showed the first net loss of life sciences jobs since the pandemic-fueled layoffs of April 2020—though job growth returned in November. Even more sobering, biotech R&D growth from January 2023 through November 2023 inched up just 0.1%.

For 2024, CBRE predicts a “modest” 0.2% dip in life sciences employment in the first half, to be followed by an uptick in the second half to end the year with a net gain in jobs. But which life sciences jobs are likeliest to see the most growth over the next decade?

For an answer to that question, see the list in this article. It contains 10 research and clinical biotech occupation categories that will add jobs through 2032, according to the BLS Occupational Outlook Handbook, which was updated last September.³ The occupation categories are ranked in order of the number of additional jobs that are expected to be created between 2022 and 2032. When two occupations show the same number of additional jobs, the tie between them is broken by ranking higher the occupation that shows growth in the number of job openings that is higher when viewed in proportion to the total number of jobs.

Also, for each occupational category, the following BLS-calculated metrics are given: the number of jobs in 2022, the percentage increase between 2022 and 2032, and the median pay per year in 2022. Accompanying these metrics is an occupational description from the BLS.

This year, as with GEN’s 2023 A-List of top biopharma jobs in demand,⁴ the greatest number of projected jobs over the coming decade were for professionals who collect samples and perform tests to analyze body fluids, tissue, and other substances—namely, clinical laboratory technologists and technicians (also known as medical laboratory scientists).

Interestingly, 7 of the 10 occupations highlighted in this A-List showed a year-over-year decrease in job openings projected over the next decade compared with last year’s A-List. That can be viewed as yet another indication that biopharma employers, both within academia and industry, have mostly begun to slow their hiring amid continuing uncertainty over the broader economy. The largest drop in the employment change projected for 2022–2032 is among biochemists. A projected increase of 2,300 was calculated this year, whereas a projected increase of 5,700 was calculated last year—a decrease of 60%.

The only three occupations showing year-over-year increases in projected job openings are zoologists (up 500 jobs 2022–2032), genetic counselors (up only 100), and epidemiologists (up 500)—the last an apparent sign that at least some laboratories are prioritizing the study of COVID-19, the prevention of future pandemics, or both.

1. Clinical Laboratory Technologists and Technicians

Projected employment change, 2022–2032: 16,800 more jobs

Job openings projected each year on average, 2022–2032: 24,000

Number of jobs, 2022: 342,900

Job outlook, 2022–2032: 5%
(Faster than average)

Median pay, 2022: $57,380/year

About the position: Clinical/medical  laboratory technicians perform medical laboratory tests for the diagnosis, treatment, and prevention of disease.

2. Medical Scientists

Projected employment change, 2022–2032: 11,600 more jobs

Job openings projected each year on average, 2022–2032: 7,500

Number of jobs, 2022: 119,000

Job outlook, 2022–2032: 10%
(Much faster than average)

Median pay, 2022: $99,930/year

About the position: Medical scientists conduct research aimed at improving overall human health. They often use clinical trials and other investigative methods to reach their findings.

3. Biological Technicians

Projected employment change, 2022–2032: 3,900 more jobs

Job openings projected each year on average, 2022–2032: 10,600

Number of jobs, 2022: 81,400

Job outlook, 2022–2032: 5%
(Faster than average)

Median pay, 2022: $49,650/year

About the position: Biological technicians help biological and medical scientists conduct laboratory tests and experiments.

4. Epidemiologists

Projected employment change, 2022–2032: 2,700 more jobs

Job openings projected each year on average, 2022–2032: 800

Number of jobs, 2022: 10,000

Job outlook, 2022–2032: 27%
(Much faster than average)

Median pay, 2022: $78,520/year

About the position: Epidemiologists are public health workers who investigate patterns and causes of disease and injury. They seek to reduce the risk and occurrence of negative health outcomes through research, community education and health policy.

5. Biochemists and Biophysicists

Projected employment change, 2022–2032: 2,300 more jobs

Job openings projected each year on average, 2022–2032: 2,800

Number of jobs, 2022: 34,500

Job outlook, 2022–2032: 7%
(Faster than average)

Median pay, 2022: $103,810/year

About the position: Biochemists and biophysicists study the chemical and physical principles of living things and of biological processes, such as cell development, growth, heredity, and disease.

6. Chemical Technicians

Projected employment change, 2022–2032: 1,900 more jobs

Job openings projected each year on average, 2022–2032: 7,100

Number of jobs, 2022: 58,800

Job outlook, 2022–2032: 3%
(As fast as average)

Median pay, 2022: $50,840/year

About the position: Chemical technicians use laboratory instruments and techniques to help scientists analyze the properties of materials.

7. Microbiologists

Projected employment change, 2022–2032: 1,100 more jobs

Job openings projected each year on average, 2022–2032: 1,700

Number of jobs, 2022: 20,900

Job outlook, 2022–2032: 5%
(Faster than average)

Median pay, 2022: $81,990/year

About the position: Microbiologists study microorganisms such as bacteria, viruses, algae, fungi, and some types of parasites. They try to understand how these organisms live, grow, and interact with their environments.

8. Bioengineers and Biomedical Engineers

Projected employment change, 2022–2032: 1,000 more jobs

Job openings projected each year on average, 2022–2032: 1,200

Number of jobs, 2022: 19,700

Job outlook, 2022–2032: 5%
(Faster than average)

Median pay, 2022: $99,550/year

About the position: Bioengineers and biomedical engineers combine engineering principles with sciences to design and create equipment, devices, computer systems, and software.

9. Genetic Counselors

Projected employment change, 2022–2032: 600 more jobs

Job openings projected each year on average, 2022–2032: 300

Number of jobs, 2022: 3,500

Job outlook, 2022–2032: 16%
(Much faster than average)

Median pay, 2022: $89,990/year

About the position: Genetic counselors assess clients’ risk for a variety of inherited conditions, such as birth defects. They review genetic test results with individuals and families and support them in making decisions based on those results. They also offer information to other healthcare providers.

10. Zoologists and Wildlife Biologists

Projected employment change, 2022–2032: 600 more jobs

Job openings projected each year on average, 2022–2032: 1,500

Number of jobs, 2022: 19,100

Job outlook, 2022–2032: 3%
(As fast as average)

Median pay, 2022: $67,430/year

About the position: Zoologists and wildlife biologists study animals—those both in captivity and in the wild—and how they interact with their ecosystems. They focus primarily on undomesticated animals and their behavior, as well as on the impact humans have on wildlife and natural habitats.

References

1. CBRE. Return to Positive Absorption in Q4 May Signal Improving Life Sciences Market in 2024.
2. CBRE. 2024 U.S. Life Sciences Outlook
3. U.S. Bureau of Labor Statistics. Occupational Outlook Handbook.
4. GEN. Top 10 Life Sciences Jobs over the Next Decade.

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These numbers come from LSEG, the global financial markets infrastructure and data provider that owns the London Stock Exchange. LSEG’s data also reveals marked increases in the dollar values assigned to the biotech companies and pharma companies that are being acquired. The total value of biotech M&A in 2023 stood at $122.2 billion, up 45% from $84.2 billion a year earlier. The total value of pharma M&A jumped 38% year over year, from $98.5 billion in 2022 to $135.5 billion in 2023.

The final quarter of 2023 saw M&A activity heat up, with no fewer than nine announced acquisition deals of $1 billion or more. The largest of these (and the second largest transaction announced in 2023) saw Bristol Myers Squibb (BMS) agree to acquire neuro drug developer Karuna Therapeutics for $14 billion, a deal set to close in the first half of 2024. BMS was very busy in Q4 2023, as it also announced plans to buy out radiopharmaceutical drug developer RayzeBio and oncology drug developer Mirati Therapeutics. The former proposal is set at $4.1 billion; the latter, up to $5.8 billion ($4.8 billion plus another $1 billion in milestone-based contingent value rights).

The M&A revival reflects buyers’ need to generate new revenue and replenish pipelines as longtime blockbusters lose patent exclusivity—as well as buyers’ ability to fund deals as capital set aside for that purpose is at near-record levels.

Another possible reason for the M&A uptick: A jump in FDA approvals of new therapies last year. Indeed, approvals rose 50% last year—from 37 in 2022, to 55 in 2023. The number of Biologics License Applications approved by the agency has risen from 10 in 2018, to 17 in 2023 (same number as in 2018, a year that saw 59 drug approvals).

“If you look at some of the some of the biotech companies that are getting these approvals, then that is really good news for the M&A market, because that really drives interest from the larger buyers either to acquire them or partner with them,” said Subin Baral, global life sciences deals leader, EY. “So, it does play a role in M&A.”

What companies will be among those whose M&A potential is tracked in coming months? Leading candidates are in the following A-List. It includes 10 biopharma companies that analysts and other market watchers have seen as buyout targets in recent months. This list is based on notes to investors and comments in news outlets. For each company mentioned, the list explains where talk of acquisitions has surfaced, and why.

This A-List marks the first compilation of takeover targets by GEN since our September 2019 A-List of top M&A prospects. Investors would have been very wise to follow that list since it included two big targets that were bought out for tens of billions of dollars in the intervening five years: Seattle Genetics (later renamed Seagen and acquired by Pfizer for $43 billion despite antitrust concerns, in a deal completed December 14); and Alexion Pharmaceuticals (acquired by AstraZeneca for $39 billion in a deal completed in 2021).

GEN has also enjoyed earlier success predicting some companies on the cusp of being bought out. GEN’s February 26, 2018, A-List included three companies that later found buyers: AveXis (acquired by Novartis for $8.7 billion in 2018) and two companies that changed hands in 2019: Spark Therapeutics (bought by Roche for $4.8 billion), and Tesaro (snapped up by GlaxoSmithKline, now GSK, for $5.1 billion).

Only 1 of the 10 companies on our 2017 list found a buyer. Juno Therapeutics was purchased for $9 billion by Celgene, which a year later got bought out by BMS for $74 billion.

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For the first time since 2012, when GEN began compiling its A-Lists of top biotechnology companies, we report a year-over-year decline in the market capitalization (the product of the share price and the number of outstanding shares) of the listed companies. As of December 8, 2023, the 25 largest biotechnology companies had a combined market capitalization of $1.711 trillion, down 2.2% from the $1.75 trillion market cap of GEN’s Top 25 Biotech Companies of 2023, an A-List published in May 2023.

However, biotechnology remains a profitable investment in the long term. Over the past five years, the market cap for the top 25 biotechnology companies soared nearly 78% from the combined $963.495 billion reported in GEN’s Top 25 Biotech Companies of 2019.

But the one-year decline is unmistakable evidence of how bearish the market has become for biotechnology companies, with investors reacting to the end of pandemic era surges in public and private financing, plunging COVID-19 vaccine sales, and the “patent cliff” loss of exclusivity by 2030 of more than 20 drugs.

Reporting ups and downs

Enduring this year’s biggest loss of market cap among top biotechnology companies was Illumina. The sequencing giant saw its market cap plummet 41.8% since last year, from $30.79 billion to $17.93 billion, and its ranking on GEN’s A-List fall from No. 17 to No. 24. Illumina’s problems included disappointing earnings, activist investor Carl C. Icahn’s proxy campaign, shareholder ouster of the chairman, the subsequent resignation of CEO Francis deSouza, and U.S. and European regulatory rebukes to its acquisition of cancer blood test developer Grail.

Among this year’s gainers, the biggest was Roche-owned Chugai Pharmaceutical, which climbed three notches from No. 11 to No. 8 thanks to a 32.7% jump in its market cap, from $45.86 billion to $60.86 billion. Chugai saw investors respond to positive announcements that included FDA acceptance of its biologics license application for paroxysmal nocturnal hemoglobinuria treatment crovalimab, and Japanese approval of its combination therapy Phesgo for HER2-positive breast and colorectal cancer.

A spot check by GEN reveals that all of the top 10 electronic transfer funds (ETFs) reported year-over-year losses as of December 8, compared with 6 of the top 10 showing year-over-year gains in GEN’s May 2023 A-List of top 25 biotechnology companies.

By comparison, the overall Standard & Poor’s (S&P) 500 index, which tracks 500 of the largest U.S. public stocks, rose 11.8% from May 2, 2023, through December 8, 2023, from $4,119.50 to $4,604.37—and 16.2% from December 8, 2022, when it stood at $3,963.51, through December 8, 2023.

The biggest one-year decliner among the biotechnology ETFs (down 40.2%) was Direxion Daily S&P Biotech Bull 3X Shares (LABU), the fifth-largest ETF with about $1.111 billion in total assets (dropping from $137.60 to $88.63). The smallest year-over-year loss was reported by SPDR S&P Biotech ETF (XBI), the second-largest biotechnology ETF with total assets of $6.425 billion.

The largest biotechnology ETF with $6.67 billion in total assets, the iShares Biotechnology ETF (IBB), fell 8.8% year over year, from $135.89 to $123.88. Third-ranked ARK Genomic Revolution ETF ($1.822 billion) slipped 8.5%, from $31.26 to $28.60.

Total asset figures were supplied by VettaFi, a provider of ETF and other data, analytics, and thought leadership for asset managers.

Making the list

This A-List presents GEN’s updated list of the top 25 biotechnology companies, ranked by their market caps as of December 8, 2023—as indicated by the exchanges on which the companies’ shares are traded, or by other publicly available sources. Each company is listed by rank, name, market cap in billions of dollars, and percentage change from last year. (Figures are rounded off to the nearest hundredth.)

This list does not include pharmaceutical companies that have a heritage of small-molecule, nonbiologic drug development. (In recent years, GEN has covered these companies through a separate list.)

Going back to the first GEN A-List in 2012, 14 top biotechnology companies that were listed back then do not appear here—because they were acquired, because their market caps fell below those of the top 25 companies, or because they restructured operations to reduce biopharmaceutical drug and life sciences tool development to less than half of total revenue.

Among January 2024’s top 25 biotechnology companies, losers of market cap (16) outnumbered gainers (9)—compared with 16 gainers and 9 losers in May 2023.

Companies headquartered outside the United States accounted for more than half (14) of the companies ranked on this latest A-List of top 25 biotechnology companies, one more than on GEN’s previous A-List published in May 2023, though one ex-U.S. company that made this year’s list did not appear last year.

Of the 14 headquartered outside the United States, three are based in China; two each in Denmark, Japan, and South Korea; and one each in Australia, Germany, India, the Netherlands, and Switzerland.

The smallest company listed this year has a market cap of nearly $18 billion, compared with just under $16 billion for the 25th ranked company on GEN’s May 2023 list.

Just missing the list at number 26 is BioMarin Pharmaceutical, which had a market cap of $17.51 billion on December 8, followed by WuXi Biologics ($15.94 billion), UCB ($15.52 billion), Novozymes ($14.44 billion), and bioMérieux ($12.47 billion).

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Earlier that year, the editors of Nature unanimously recognized the technology—which they called spatially resolved transcriptomics—as Method of the Year 2020. “This maintenance of spatial context is crucial for understanding key aspects of cell biology, developmental biology, neurobiology, tumor biology, and more, as specialized cell types and their specific organization are crucially tied to biological activity and remain poorly explored on the scale of whole tissues and organisms.”

The spatial market is on a growth trajectory, though estimates differ on how quickly it will expand in coming years. BCC Research projected in April that spatial genomics and transcriptomics will grow from $1.5 billion last year at a compound annual growth rate (CAGR) of 10.8, to $2.5 billion by 2027.

That’s more than double the $1.2 billion estimate by 2027 forecast by DeciBio Consulting, which foresees an even faster CAGR of 30% from 2022, placing the value that year at just over $323 million.

Among stakeholders that appreciate the potential of spatial biology is Singapore’s Agency for Science, Technology and Research (A*STAR).

A*STAR’s Genome Institute of Singapore (GIS) joined in October with NanoString Technologies and Next Level Genomics, a long-read next-generation sequencing services provider, to establish a joint laboratory focused on applying spatial biology to identify biomarkers that can predict disease progression and treatment response. The new lab, SpACE-Dx (short for Spatial Atlas of Clinical Evolution of Disease), will drive research in complex diseases—primarily focusing on cancer—by harnessing spatial multiomic (gene and protein expression) technologies.

GEN published an updated spatial biology A-List last year, and is pleased to update that list again here. One interesting trend: Revenues for the top five spatial biology companies are climbing. Between 2021 and 2022, total revenue for those companies rose just 3% over 2021, from $3.239 billion to $3.347 billion. Over the first half of this year, however, revenues for those top five public companies soared 17% from a year earlier, from $1.556 billion to $1.826 billion.

On a brighter note, total capital raised by the top five private companies jumped 40% year over year, from more than $365.3 million in 2022 to more than $511.5 million this year.

This A-List is actually three lists in one—public companies, private companies, and “up and coming” companies of note, updated as of November 6.

Public companies are ranked by their combined revenues for 2022 and the first half of 2023, as disclosed in regulatory filings, including sales of products or services, as well as revenue from collaborations and R&D activity.

Private companies are ranked by the total capital they have raised, as disclosed by the companies themselves, either in press statements or in responses to GEN queries verifying figures compiled by other sources.

Also included in this list are eight “up-and-comers,” companies that have either raised significant capital in recent months, shown positive data for their technologies, and/or launched significant new collaborations with partners.

Each company is listed with a short explanation of their recent activity.

References
1. Total capital disclosed by the company in the February 23, 2022, announcement of Spatial Genomics’ Series A financing. The company at deadline had not responded to GEN queries about any subsequent additional financing since then.
2. Total capital disclosed by RareCyte last year. Company at deadline had not responded to GEN queries seeking an updated total capital raised figure.

Alex Philippidis is senior business editor of GEN.

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Recent developments in the CDMO sector include pandemic-driven demand fluctuations and supply chain disruptions, private equity–driven dealmaking and consolidation, and business model shifts. (These shifts reflect an interest in exploring new product categories, in offering end-to-end services, and in cultivating more lasting relationships with clients.) With all these developments, is it possible to venture any general statements about the CDMO sector?

One thing we can say is that the CDMO sector is poised for growth—at least according to market research firms. For example, in May, Grand View Research issued a report projecting that the global CDMO market will expand at a compound annual growth rate of 6.1% over eight years, from $135.85 billion last year to $232.59 billion by 2030.

The report also noted that in 2022, oncology drugs accounted for the largest share of CDMO activity, 35% ($47.684 billion). Moreover, according to Grand View Research, the oncology drug segment is expected to outgrow the overall CDMO industry with a CAGR of 7.4%–which would bring oncology CDMO activity by 2030 to $90.66 billion. This growth reflects the interest biopharmaceutical companies have in meeting rising demand for new oncology drugs as cancer becomes more prevalent worldwide.

Another thing we can say is that positive views on the CDMO market are not limited to market researchers. Such views are also available from recruitment firms in the life sciences industry. For example, Michael Selwood, a consultant at Mantell Associates, has observed that there is a trend toward greater demand for the services provided by CDMOs. He has suggested that this trend is being driven by six factors: growing complexity of drug development, cost containment and efficiency, focus on core competencies, increasing pipeline of biologics and personalized medicine, globalization and emerging markets, and COVID-19 pandemic impact.

Finally, we can say that CDMOs themselves see opportunities ahead. For example, the CDMO companies in this article are demonstrating their optimism through investments in new technologies and in new and expanded facilities, as well as through commitments to ambitious joint ventures.

These companies populate GEN’s first A-List of Contract Development and Manufacturing Organizations. It is, in fact, a “top 10” list, one in which the CDMOs are ranked by their 2022 revenues—as disclosed by the companies in regulatory filings or in responses to GEN queries.

Details about the top 10 CDMOs are provided in the list that accompanies this text. Besides naming a CDMO and citing its 2022 revenue, each item in the list indicates where the CDMO is headquartered. Finally, each item notes a few selected news developments.

1. Lonza Group
Basel, Switzerland
CHF 6.223 billion ($6.970 billion)

• Joined with Vertex Pharmaceuticals to announce plans for a 130,000-square-foot manufacturing facility in Portsmouth, NH, for Vertex’s type 1 diabetes cell therapies.
• Agreed to acquire Amsterdam-based Synaffix for €100 million ($107 million) in upfront cash and up to €60 million ($64 million) in milestone payments, in a deal intended to strengthen Lonza’s bioconjugates offering.
• Launched a partnership with ABL Bio focused on developing and manufacturing ABL’s new bispecific antibody for immuno-oncology and neurodegenerative diseases.

2. Thermo Fisher Scientific
Waltham, MA
$6.967 billion¹

• Announced a next-generation platform of Dynabeads that is intended to accelerate cell therapy manufacturing.
• Opened a sterile fill-finish facility in Singapore.

3. Catalent
Somerset, NJ
FY 2023 Revenues: $4.276 billion²

• Avoided a proxy battle from Elliott Investment Management by agreeing to add four new independent directors supported by the activist investor.
• Announced that new executive chair John Greisch will head a new committee created to review Catalent’s business, strategy and operations, and capital-allocation priorities. (The agreement is intended to reverse a stock price plunge that sent Catalent’s stock skidding 48% from $92.28 to $47.81 the day Catalent and Elliott came to terms.)

4. WuXi Biologics
Wuxi, China
RMB 15.269 billion ($2.108 billion)

• Announced that it will spin off WuXi XDC Cayman, which originated as a joint venture with Wuxi STA to provide bioconjugate services.
• Announced plans to expand its capacity for drug substance and drug product in Germany, in part by doubling the capacity of its drug substance facility in Wuppertal from 12,000 L to 24,000 L.

5. Samsung Biologics
Incheon, South Korea
KRW 2.338 trillion ($1.773 billion)

• Announced full completion of its Bio Campus I, which offers a capacity of 604,000 L, and a planned expansion of Bio Campus II, which is designed to begin operations in 2025.
• Announced two manufacturing deals with Pfizer totaling $897 million to produce biosimilars with oncology, inflammation, and immunotherapy indications through 2029.
• Announced a $390.9 million drug production deal with Novartis, expanding on an $81 million contract signed last year.

6. Siegfried
Zofingen, Switzerland
CHF 1.23 billion ($1.377 billion)

• Acquired a 95% stake in DiNAMIQS, a Swiss CDMO focused on developing and manufacturing viral vectors. (Siegfried is upgrading DiNAMIQS’s capabilities to commercial scale by building a GMP-compliant facility set to start operations in 2025 with flexible capacities of up to 500 L.)
• Opened a Development Center for Drug Products in Barcelona, Spain.
• Broke ground on a CHF 25 million ($28 million) R&D Center for Drug Substances in Evionnaz, Switzerland.

7. Fujifilm Diosynth Biotechnologies
College Station, TX
Fujifilm Tokyo, Japan
¥ 194.2 billion ($1.314 billion)³

• Named Lars Petersen the new president and CEO. (Peterson was previously chief operating officer of the Fujifilm Diosynth site in Hillerød, Denmark, and head of its large-scale strategic business unit.)
• Created a new Strategic Business Unit (SBU) to offer services and manufacturing capacities tailored to support large- or small-scale biopharma clients.

8. Recipharm
Stockholm, Sweden
€1.227 billion ($1.314 billion)

• Announced that it will help Barcelona-based Ahead Therapeutics develop its lead pipeline candidate, a treatment for myasthenia gravis.
• Agreed to provide analytical and process development capabilities to support toxicology studies, as well as GLP manufacturing of lipid nanoparticles to encapsulate the active pharmaceutical ingredient, an antigen peptide.
• Opened a new analytical lab in Bengaluru, India, designed to enhance its global testing capabilities for nitrosamines, extractables and leachables, and elemental impurity testing.
• Arranta Bio subsidiary partnered with MIT to develop a continuous manufacturing technology for mRNA therapeutics.

9. Boehringer Ingelheim
Ingelheim, Germany
€1.024 billion ($1.097 billion)⁴

• Broke ground in May on a €285 million ($305 million) Chemical Innovation Plant at its headquarters. (Operations are set to begin in 2026. Manufacturing processes are to be developed for producing active pharmaceutical ingredients (APIs) and drugs for clinical trials.)
• Celebrated its new €350 million ($374.5 million), 34,500 square meter (371,355 square-foot) Biologicals Development Center in Biberach an der Riß, Germany, where 500-plus employees will research and develop antibodies and therapeutic proteins.

10. MilliporeSigma
Burlington, MA
Merck Darnstadt, Germany
€956 million ($1.024 million)⁵

• Announced a $25 million expansion of its Lenexa, KS, facility that will add 98,000 square feet of laboratory and production space for manufacturing cell culture media. Lenexa joins dry powder cell culture media manufacturing sites in Nantong, China, and Irvine, Scotland.
• Announced it is stepping up biosafety testing in two sites in Scotland (Glasgow and Stirling) through a €35 million ($37.5 million) expansion that is projected to create nearly 500 jobs.

References

1. Revenues reflect Life Science Services sector, which includes CDMO and contract testing services.

2. Revenue reflects figure for Biopharma Contract Manufacturing operations, furnished by the company as part of its annual reports.

3. Figure is the sum of Q1-Q4 fiscal year 2022 quarterly revenue figures for the “Bio CDMO” business within the Healthcare segment of Fujifilm Holdings Corp., whose subsidiaries include CDMO Fujifilm Diosynth Biotechnologies.  Fujifilm operates on a fiscal year that runs from April 1 of the named fiscal year to March 31. FY 2022 covers the 12 months starting April 1, 2022, and ending March 31, 2023.

4. Catalent operates on a fiscal year that ends June 30. Figure reflects net revenues for the 12 months ending June 30, 2023.

5. Revenue figure reflects the approximately 30% share of revenues generated by the Pharma Services business of Thermo Fisher’s Laboratory Products and Biopharma Services segment, according to a pie chart presented at the company’s Investor Day presentation on May 24. Pharma Services includes Patheon, which Thermo Fisher acquired in 2017 for $7.2 billion. 

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