Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
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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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.
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).
Notably, 1 patient treated in the trial died of respiratory failure, but it is not clear whether the death was related to LYL797.
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.
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.
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.
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).
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).
Bendamustine Is an Effective Alternative to Fludarabine-Based Lymphodepletion in LBCL
December 7th 2024In the wake of fludarabine shortages, lemphodepletion with bendamustine was found to be an effective alternative compared for patients with large B-cell lymphoma being treated with a CD19-directed CAR T-cell therapy.