They discussed general insights on how diverse the topics have been. Julianna discussed David Liu’s, PhD, keynote on Wednesday and Corinna shared about an interview she had with Frederic Revah, PhD, Genethon.

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Here, they take a moment to chat about the start of the meeting and what they are looking forward to over the next few days. Davies discusses how this year’s conference is different from the meetings five years ago, opening workshops featuring Y. Esther Tak, PhD, Arena Bioworks, Samagya Banskota, PhD, Boston University, Jonathan Levy, PhD, Prime Medicine, Makoto Saito, PhD, Broad Institute, and Cameron Glasscock, PhD, University of Washington. They both share their thoughts on an interview with Mammoth’s CSO Lucas Harrington, and the news about the DAYLIGHT trial. Singleman shares her first impressions of ASGCT as a first-time attendee, some interesting talks during the “Prospects for Prenatal Gene and Cell Therapy” session Tuesday morning, and how things are going in the exhibition hall. 

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Here, they take a moment to chat about the first day at the meeting, with a focus on the opening plenary session featuring Aviv Regev from Genentech, Jakob Nikolas Kather, from Technical University Dresden, Ben Cravatt, Scripps Research, and Carolyn Bertozzi, Stanford University.

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In her first trip to U.S. HUPO, Julianna LeMieux, PhD, deputy editor in chief at GEN is on the ground—in the talks and exhibition hall. She took a minute to chat with Jonathan Grinstein, PhD, a senior editor at GEN, on her takeaways from the meeting and the field of proteomics.

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Her remarkable recovery since then, foreshadowing the successful treatment of dozens of other SCD patients in the trial, led to the swift regulatory approval by the U.S. Food and Drug Administration in December 2023. Casgevy, as the therapy is now called, has also been approved by agencies in the U.K. and the European Union.

Gray’s inspiring story has been shared in a series of interviews since 2019 with National Public Radio and in a brief presentation at the Human Genome Editing conference in London in March 2023. But this is her first extended video interview, recorded for the inaugural State of Cell and Gene Therapy virtual summit, broadcast on January 24, 2024.

In an exclusive 45-minute interview with GEN’s editorial director, Kevin Davies, PhD, and GEN senior editor, Uduak Thomas, Gray shares her story of being diagnosed at three months, years of living with debilitating pain and doctors’ visits, and the sustaining power of her Christian faith. She brings us into the hospital room at the Sarah Cannon Cancer Institute at TriStar Health in Nashville, TN, where she received the infusion of her gene-edited “supercells.” Gray also discusses the arduous weeks of preparation and testing prior to treatment, the road to recovery, her new life back home with her family post-treatment, and finding her voice as a public speaker. 

The transcript of Victoria’s interview has been published in The CRISPR Journal (February 2024).

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To look back at the year that is ending, and look ahead to 2024, GEN invited CEOs of three companies to offer some valuable perspective on the biopharma industry’s strengths and challenges, and the strengths and challenges of their own companies.

• Neil McFarlane, CEO of Zevra Therapeutics
• Karen Zaderej, CEO of Axogen
• Simon Arkell, CEO of Ryght

McFarlane assumed his current role as CEO of Zevra in October, the latest chapter in a career stretching over 25 years of global biopharma and life sciences experience.

Just a month ago Zevra propelled itself into the commercial phase by completing the acquisition of Acer Therapeutics for up to $91 million—$15 million plus $76 million in contingent value rights tied to milestones. Zevra is also moving toward resubmission of its new drug application (NDA) for Arimoclomol by year’s end, as a treatment for Niemann-Pick disease type C (NPC), an ultra-rare, genetic, progressive and fatal neurological disease.

Zaderej joined Axogen in 2006, and has served as president, CEO, and a board member since 2011. Seven years later, she became chairman of Axogen, a developer of products for peripheral nerve regeneration and repair. Axogen is headquartered in Alachua, FL, with a second corporate campus in Tampa, FL, a global distribution facility in Burleson, TX, and a tissue processing center in Dayton, OH.

2023 was a memorable year for Axogen. In August, it began processing Avance^® Nerve Graft at its processing center in Dayton, intended to support the company’s long-term growth as well as a Biologics License Application (BLA) submission planned for the first half of 2024 to transition the product into a biologic.

A month later in September, Axogen launched Axoguard HA+ Nerve Protector^TM (HA+), which combines a remodeling extracellular matrix base-layer for long-term protection with a short-term resorbable hyaluronate-alginate gel coating for enhanced nerve gliding and minimization of soft tissue attachments. In 2024, Axogen plans to launch another product, Avive+ Soft Tissue Matrix in Q1.

Arkell was an Olympic pole vaulter for the Australian national team who won several championship honors in Australia and in the U.S. before co-founding Ryght, the developer of a generative-AI data analytics platform for biopharmas.

Based in Laguna Beach, CA, the company officially launched in June as Synthetica Bio, and rebranded in November to reflect its focus on providing accurate and reliable insights for driving biopharma decision-making in clinical trial operations and commercial activities.

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