Around the Helix: Cell and Gene Therapy Company Updates – April 10, 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 Ide-Cel's Expanded Indication to Second-Line, Triple-Class Refractory Multiple Myeloma

The FDA has approved Bristol Myers Squibb and 2seventy bio’s supplemental biologics license application (sBLA) for idecabtagene vicleucel's (ide-cel; Abecma) chimeric antigen receptor (CAR) T-cell therapy, expanding the indication to patients with relapsed or refractory multiple myeloma (MM) after 2 or more prior lines of therapy including an immunomodulatory agent (IMiD), a proteasome inhibitor (PI), and an antiCD38 monoclonal antibody.

2. FDA Approves Janssen and Legend Biotech’s Carvykti for Expanded Indication in Earlier Line Multiple Myeloma

The FDA has approved Janssen’s and Legend Biotech's ciltacabtagene autoleucel (cilta-cel; marketed as Carvykti) for an expanded indication in adult patients with relapsed and lenalidomide-refractory MM who have been treated with at least 1 prior line of therapy, including a PI and anIMiD.

3. BrainStorm Reaches Accord With FDA on New Phase 3b Trial Design for ALS Cell Therapy NurOwn

Under a special protocol agreement, BrainStorm Cell Therapeutics has received written agreement from the FDA regarding the protocol and statistical analysis for its planned phase 3b clinical trial (BCT-006-US; NCT identifier pending) to evaluate debamestrocel (NurOwn), an investigational autologous mesenchymal stem cell neurotrophic factor–secreting cell therapy product that is intended to treat amyotrophic lateral sclerosis (ALS).

4. IASO Bio’s CAR-T Eque-Cel Cleared for US Trial in Generalized Myasthenia Gravis

IASO Biotechnology’s equecabtagene autoleucel (eque-cel, also known as CT103A), an investigational autologous BCMA-directed CAR-T therapy, has received clearance of an investigational new drug application from the FDA for a trial in generalized myasthenia gravis.

5. Forge’s Krabbe Disease Gene Therapy Garners Innovation Passport

Forge Biologics has received an innovation passport designation from the United Kingdom’s Medicines and Healthcare Products Regulatory Agency for FBX-101, which allows access to the Innovative Licensing and Access Pathway. FBX-101 is an investigational adeno-associated virus (AAV) vector-based gene therapy currently in clinical development for the treatment of Krabbe disease.

6. New Biotech Company Nvelop Therapeutics Launches WithIntent of Delivering Genetic Medicines Via Nonviral Methods

Nvelop, which has received financing from investors such as Newpath Partners and Atlas Ventur, is seeking to use 2 modular platforms for in vivo, nonviral delivery for cargo such as base and prime editor ribonucleoproteins. “With the combination of best-in-class technologies, scientific co-founders with a deep track record in cell and gene therapy, and a team that has pioneered the development of some of the first approved therapies in the field, Nvelop is uniquely positioned to unlock the promise of genetic medicines,” Jeff Walsh, MBA, the CEO of Nvelop, said in a statement.

7. Ginkgo Scoops Up CAR-T Assets From Modulus

Ginkgo Bioworks has acquired CAR and switch receptor libraries from Modulus Therapeutics and intends to use the assets to support customers looking to improve CAR therapy performance in solid tumors, autoimmune diseases, and other indications. “We are thrilled to contribute our innovative designs to the Ginkgo ecosystem, and look forward to seeing these tools deployed across a range of Ginkgo-partnered programs,” Max Darnell, PhD, the CEO and cofounder of Modulus, said in a statement.

8. Charles River and Axovia Team Up to Tackle Ciliopathy With Gene Therapy

Under a new collaboration agreement, contract development and manufacturing organization Charles River Laboratories will provide Axovia Therapeutics with plasmid DNA for use in the phase 1 clinical evaluation of AXV101, an AAV vector-based gene therapy intended to treat Bardet-Biedl syndrome.