Around the Helix: Cell and Gene Therapy Company Updates – January 22, 2025

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 Issues CRL for Atara Biotherapeutics’ BLA for T-Cell Immunotherapy Tabelecleucel for EBV+ PTLD

The FDA issued a complete response letter (CRL) to Atara Biotherapeutics regarding its biologics license application (BLA) for tabelecleucel (tab-cel, also known as Ebvallo), an allogeneic Epstein-Barr virus (EBV)-specific T-cell immunotherapy, which is under evaluation for the treatment of patients with relapsed/refractory (r/r) EBV-positive post-transplant lymphoproliferative disease (EBV+ PTLD).

2. All of Atara Biotherapeutics’ T-Cell Immunotherapy Trials Placed on Clinical Hold by the FDA

Several days after the CRL, the FDA placed clinical holds on Atara Biotherapeutics’ active investigational new drug applications, which include clinical trials for tab-cel and ATA3219, an investigational allogeneic CD19-directed chimeric antigen receptor T-cell (CAR-T) therapy consisting of EBV-sensitized T-cells that have not been genetically modified being evaluated for nonHodgkin lymphoma (NHL) and systemic lupus erythematosus.

3. Solid Biosciences’ Friedreich Ataxia Gene Therapy SGT-212 Garners Fast Track Designation

Solid Biosciences’ SGT-212, an adeno-associated virus (AAV) vector-based gene therapy intended to treat Friedreich ataxia (FA), has been granted fast track designation by the FDA.

4. NGGT Biotechnology’s Gene Therapy NGGT001 Continues to Show Encouraging Safety in Bietti’s Crystalline Dystrophy

Next Generation Gene Therapeutics (NGGT) Biotechnology’s NGGT001 (also known as rAAV2-hCYP4V2), an investigational AAV vector-based gene therapy intended to treat Bietti’s crystalline dystrophy, did not show any substantial safety concerns in updated data from an early phase 1 investigator-initiated trial (NCT06302608).

5. Alaya.bio and Michel Sadelain, MD, PhD, Up Their Collaboration on In Situ CAR-T

Alaya.bio has expanded its collaboration with Michel Sadelain, MD, PhD, the inaugural Director of the Columbia [University] Initiative for Cell Engineering and Therapy, and his team, which originally began at the Memorial Sloan Kettering Cancer Center. The current efforts of the collaborators will be focused on advancing the development of Alaya.bio’s proprietary polymeric delivery platform for the generation of CAR-T therapy in situ.

6. Galapagos and Catalent Join Forces to Bring CAR-T Manufacturing Closer to Patients

Under a new collaboration agreement, Galapagos and contract development and manufacturing organization Catalent are seeking to establish decentralized manufacturing for planned clinical trials for Galapagos’ investigational CAR-T therapy, which is being evaluated for r/r NHL. The trials, which will take place in New Jersey, New York, and other nearby areas, will be supported by a Catalent facility in Princeton, NJ, with the expectation of enabling patients to have autologous CAR-T manufactured in 7 days.

7. First Danish Patients Infused With Hemgenix

The first patients in Denmark have been treated with UniQure and CSL Behring’s etranacogene dezaparvovec (EtranaDez), marketed as Hemgenix, which was the first gene therapy product to be approved in Europe and the United States for the treatment of hemophilia B. Notably, patients in Denmark are able to access EtranaDez via an outcome-based agreement, the first such policy for EtranaDez to have been put in action by a European nation.

8. MeiraGTx Snags RPDD for Leber Congenital Amaurosis Gene Therapy

MeiraGTx had received rare pediatric disease designation (RPDD) from the FDA for AAV8-RK-RetGC, an investigational AAV vector-based gene therapy in development for the treatment of Leber congenital amaurosis caused by mutations in GUCY2D. Notably, it is the fourth RPDD designation in 3 months that the company has garnered from the FDA for an ophthalmology gene therapy program.