Around the Helix: Cell and Gene Therapy Company Updates – January 31, 2024

The cell and gene therapy sectors are growing exponentially, with new players emerging daily and much progress being made both in and out of the lab. CGTLive™’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.

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1. FDA Approves Kite’s Axi-Cel Manufacturing Change to Reduce Turnaround Time

The FDA has approved Kite Pharma’s manufacturing process change for axicabtagene ciloleucel (axi-cel; Yescarta). The new change is expected to reduce median turnaround time from leukapheresis to product release from 16 days to 14 days.

2. FDA Finalizes Sets of Guidelines for Both CAR-T Products and Genome Editing Products

The FDA has finalized 2 guidance documents focused on the development of chimeric antigen receptor T-cell (CAR-T) therapy products and gene therapy products that incorporate human genome editing, respectively.

3. Janssen and Legend Biotech’s Carvykti to be Put in Front of FDA AdComm Regarding Multiple Myeloma Expanded Use BLA

Janssen’s and Legend Biotech's ciltacabtagene autoleucel (cilta-cel; marketed as Carvykti), which is an FDA-approved BCMA-directed autologous CAR-T, will be put in front of the FDA’s Oncologic Drugs Advisory Committee (AdComm) as part of the review process for its biologics license application (BLA) for an expanded indication in adult patients with relapsed and lenalidomide-refractory multiple myeloma who have been treated with at least 1 prior line of therapy, including a proteasome inhibitor and an immunomodulatory agent.

4. Ahead of Announcing Positive Results Seen in Trial for Gene Therapy FBX-101, Forge Biologics’ CEO Makes Comment Supporting Krabbe Disease’s Addition to RUSP

Forge Biologics, which is developing adeno-associated virus (AAV) vector-based gene therapy FBX-101 for the treatment of Krabbe disease, has announced that Timothy J. Miller, PhD, the CEO and president of Forge, made a comment during a public meeting of the Advisory Committee on Heritable Disorders in Newborns and Children on January 30, 2024, supporting the addition of Krabbe disease to the national Recommended Newborn Screening Panel (RUSP).

5. Astellas and Mass General Brigham Join Forces for 5 Year Plan

Under a new 5 year strategic agreement, Astellas Pharma and Mass General Brigham will perform collaborative research in the realm of oncology, rare disease, and cell and gene therapy. “Industry alliances are critical to advancing novel therapies toward commercialization and bringing the ‘bench to the bedside’,” Chris Coburn, MS, the chief innovation officer at Mass General Brigham, said in a statement. “This collaboration supports 3 critical areas of rapid clinical advancement and high patient need...”

6. Gamida Cell Looking to Sell Off Assets or Merge

Gamida Cell announced that it is seeking “strategic alternatives”, which may include sale of its assets, merging with another company, or another form of transaction “in order to maximize value for all stakeholders”. In the meantime, the company noted that it will continue to press forward with commercialization activities for its FDA approved allogeneic hematopoietic stem cell therapy Omisirge (omidubicel-onlv). The first patient was treated with omidubicel-onlv in the commercial setting in October 2023.

7. Regeneron's Newly Formed R&D Unit Will Take On Development of Assets Acquired from 2seventy

Regeneron Cell Medicines, a research and development (R&D) unit formed by Regeneron, will handle the continued development of a slew of preclinical and clinical immune cell therapy programs that the company acquired from 2seventy bio. "By integrating 2seventy’s pipeline of cell therapies and their talented team, we are complementing our own expertise and portfolio of innovative immuno-oncology treatments, which will allow for potentially transformative combinations that can really make a difference in patients’ lives," George D Yancopoulos, MD, PhD, the board cochair, cofounder, president, and chief scientific officer of Regeneron, said in a statement.

8. Ractigen Therapeutics and University Medical Center Utrecht Team Up to Tackle Neurodevelopmental Disorders With saRNA

In a newly announced collaboration between the company an the academic institution, the partners will pursue the development of small activating RNA (saRNA) therapeutics for the treatment of a variety of neurodevelopmental disorder indications. “Our partnership with Utrecht University represents a significant step forward in our mission to develop innovative therapies for challenging genetic disorders," Long-Cheng Li, MD, the CEO of Ractigen Therapeutics, said in a statement. "By combining our RNAa technology and advanced delivery systems with Utrecht’s impactful research, we believe we can make a real difference in the field of neurodevelopmental disorders.”