Around the Helix: Cell and Gene Therapy Company Updates – July 3, 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 Issues CRL for Rocket’s LAD-I Gene Therapy

The FDA has issued a complete response letter (CRL) to Rocket Pharmaceuticals’ marnetegragene autotemcel (RP-L201), to be marketed under the name Kresladi, for the potential treatment of leukocyte adhesion deficiency-I (LAD-I) due to a request for more Chemistry Manufacturing and Controls information to complete its review.

2. REGENXBIO Set to Evaluate Duchenne Muscular Dystrophy Gene Therapy RGX-202 in Patients Aged 1 to 3 Years Old

REGENXBIO has begun enrolling participants for a new cohort of younger patients in the phase 1/2 AFFINITY DUCHENNE clinical trial (NCT05693142), which is evaluating RGX-202, an investigational adeno-associated virus (AAV) vector-based gene therapy, for the treatment of Duchenne muscular dystrophy (DMD).

3. Lyell’s ROR1-Targeted CAR-T LYL797 Attains 60% Clinical Benefit Rate at High Dose Level in Phase 1 Solid Tumor Trial

Notably, 1 patient treated in the trial died of respiratory failure, but it is not clear whether the death was related to LYL797.

4. Cartesian Therapeutics’ mRNA CAR-T Descartes-08 Continues to Show Efficacy in Phase 2b Myasthenia Gravis Trial

Cartesian also announced that the first patient has been dosed in a separate clinical trial evaluating the chimeric antigen receptor T-cell (CAR-T) therapy in systematic lupus erythematosus.

5. Novo Nordisk Snags Hemophilia Program and Gene Editing Tech Rights From 2seventy bio

Novo Nordisk has agreed to purchase 2seventy bio’s hemophilia A program and rights to the company’s megaTAL in vivo gene editing technology, excepting the technology's use for oncology and gene editing of immune cells for the treatment of autoimmune diseases. 2seventy bio noted that the sale, potentially worth up to $40 million, will allow it to further focus its efforts on the development of its multiple myeloma CAR-T idecabtagene vicleucel.

6. uniQure Selling Viral Vector Manufacturing Facility to Genezen

UniQure has agreed to sell a viral vector manufacturing facility located in Lexington, Massachusetts, to the contract development and manufacturing organization Genezen. Under Genezen, the facility will continue to produce materials for uniQure under strategic supply agreements, in addition to serving Genezen’s other customers.

7. Senti Bio Secures $8 Million CIRM Grant for CAR-NK Program

The grant from the California Institute for Regenerative Medicine (CIRM) will help fund the continued development of Senti Bio’s SENTI-202, an investigational allogeneic CAR natural killer (CAR-NK) cell therapy candidate intended to treat acute myeloid leukemia and myelodysplastic syndrome. The therapy, which incorporates logic gating technology, is currently being assessed in a phase 1 study (NCT06325748).

8. Artiva Therapeutics Files for IPO

Artiva Therapeutics has filed with the United States Securities and Exchange Commission (SEC) for an initial public offering (IPO). Within the SEC filing, Artiva characterizes itself as a biotechnology company focused on the development of NK cell therapies for autoimmune disease and cancer indications. Notably, Artiva is currently evaluating AlloNK (AB-101), its investigational allogeneic NK cell therapy, in separate clinical trials for lupus (NCT06265220) and lymphoma indications (NCT04673617; NCT05883449).