Around the Helix: Cell and Gene Therapy Company Updates – March 5, 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. Patient With Frontotemporal Dementia Treated With NKGen’s Autologous NK Cell Therapy Troculeucel in Compassionate Use Context

A patient has received NKGen Biotech's troculeucel (also known as SNK01), an investigational autologous natural killer (NK) cell therapy, for the treatment of frontotemporal dementia (FTD) under a single-patient compassionate use investigational new drug (IND) cleared by the FDA.

2. FDA Accepts BLA for Capricor’s DMD Cardiomyopathy Cell Therapy Deramiocel With Priority Review

The FDA has accepted the biologics license application (BLA) that Capricor Therapeutics submitted for Deramiocel (also known as CAP-1002), an investigational allogeneic cardiosphere-derived cell therapy intended to treat Duchenne muscular dystrophy (DMD) cardiomyopathy, with priority review. The agency has set the Prescription Drug User Fee Act (PDUFA) action date for the BLA as August 31, 2025.

3. Ocugen’s Geographic Atrophy and Stargardt Gene Therapies Garner Positive ATMP Opinions from EMA

The European Medicines Agency (EMA) and its Committee for Advanced Therapies (CAT) has provided a positive opinion to 2 of Ocugen's investigational gene therapy products: OCU410 and OCU410ST. OCU410 is under evaluation for the treatment of vision loss due to geographic atrophy (GA) associated with dry age-related macular degeneration (AMD) and OCU410ST is intended to treat Stargardt disease, which is associated with ABCA4-related retinopathies.

4. Regeneron’s Gene Therapy DB-OTO Improves Hearing in Children With Otoferlin-Related Hearing Loss

Regeneron’s DB-OTO, an investigational adeno-associated virus (AAV) dual-vector-based gene therapy intended to treat otoferlin (OTOF)-related hearing loss, has improved hearing in pediatric patients treated in the phase 1/2 CHORD clinical trial (NCT05788536).

5. Bioheng Therapeutics' UCAR-T CTD402 Cleared for US Trial

Bioheng Therapeutics' CTD402, an investigational CD7-directed universal chimeric antigen receptor T-cell (UCAR-T) therapy, has received clearance from the FDA of an investigational new drug (IND) application for a phase 1b/2 clinical trial (NCT identifier pending) in relapsed/refractory T-cell acute lymphoblastic leukemia/lymphoma. “Investigator-initiated trial study results showed an impressive overall response rate, alongside a favorable safety profile," Jiangtao Ren, PhD, the president and chief scientific officer of Bioheng, said in a statement. "These results validate our ANSWER platform’s ability to deliver both rapid therapeutic impact and reduced patient risk, positioning CTD402 as a potential best-in-class therapy for T-cell malignancies."

6. IN8bio Shows Off Gamma-Delta T-Cell Engager Platform for Cancer and Autoimmune Disease

IN8bio has revealed a next generation gamma-delta T-cell engager (TCE) platform dubbed INB-600, which is intended to circumvent drawbacks that have held back other gamma-delta TCE products. "Most gamma-delta TCEs have failed because they can’t engage the limited number of effector cells to eliminate their targets," William Ho, MBA, the CEO and cofounder of IN8bio, said in a statement. "Our INB-600 platform is working to change the equation by not only targeting B cells, but also by actively expanding the gamma-delta T cell immune army needed for deep, lasting B cell depletion.

7. Vertex Backs Out of Liver Disease Gene Editing Collaboration With Verve

Vertex Pharmaceuticals made the decision to step away from a preclinical gene therapy program for liver disease that it had been collaborating on under an agreement with Verve Therapeutics. As a result, Verve has regained the full rights for the program, which it intends to continue developing independently. Vertex cited changing priorities in its own portfolio as the reason behind the move.

8. CARsgen Snags NMPA Breakthrough Therapy Designation for Gastric Cancer CAR-T Satri-Cel

CARsgen's satricabtagene autoleucel (satri-cel), a Claudin18.2-directed CAR-T therapy intended to treat gastric/pancreatic cancers, has received breakthrough therapy designation from China's National Medical Products Administration (NMPA) for the treatment of Claudin18.2-positive advanced gastric/gastroesophageal junction cancer in patients who have failed 2 or more previous lines of therapy. CARsgen noted that it plans to submit a new drug application to the NMPA for satri-cel, which is currently being evaluated in multiple clinical studies, before the midway point of this year.