Around the Helix: Cell and Gene Therapy Company Updates – March 11, 2026

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 Peels Back CRL on Capricor's DMD Cardiomyopathy Cell Therapy Deramiocel

The FDA has lifted the complete response letter (CRL) previously issued for Capricor Therapeutics’ biologics license application (BLA) seeking approval of deramiocel, an investigational allogeneic cardiosphere-derived cell therapy being developed to treat cardiomyopathy associated with Duchenne muscular dystrophy (DMD).

2. Phase 2 FORWARD-53 Trial Reports Sustained Dystrophin Expression With WVE-N531 in Duchenne Muscular Dystrophy

The investigational exon-skipping therapy WVE-N531 (Wave Life Sciences) produced sustained increases in dystrophin levels and early indications of functional improvement in boys with DMD who are amenable to exon 53 skipping, according to 48-week findings from the phase 2 FORWARD-53 study.

3. Where is Neuromuscular Medicine Heading in the Future?

At MDA’s 2026 conference, Biotechnology Innovation Organization (BIO) CEO John Crowley, JD, MBA, discussed the importance of genomic medicine approaches to gene therapy’s future.

4. Phase 1/2 VALOR Study to Assess ASP2957 Gene Therapy in Infants With X-Linked Myotubular Myopathy

A first-in-human clinical trial is underway to evaluate ASP2957 (MyoAAV3.8-MHCK7-hMTM1), an investigational adeno-associated virus (AAV)–based gene therapy designed to restore MTM1 expression in infants with X-linked myotubular myopathy (XLMTM), a rare congenital neuromuscular disorder associated with severe muscle weakness and respiratory failure. Otherwise known as the phase 1/2 VALOR trial, this study will assess the safety and preliminary efficacy of a single intravenous administration of the gene therapy in young children with advanced XLMTM who require substantial ventilatory support.

5. Tensions Heighten as Anonymous FDA Official Calls uniQure’s AMT-130 a “Failed Therapy”

According to CNBC, an anonymous FDA official confirmed on a call with the news outlet that the FDA is requesting a sham surgery-controlled phase 3 clinical trial for uniQure’s investigational Huntington disease gene therapy AMT-130 and additionally referred to AMT-130 as a “failed” therapy. “UniQure is the latest company to make a failed therapy for Huntington’s patients,” the official reportedly told CNBC on the call. “They likely acknowledge or understand at some deep level that their trial failed years ago, and instead of doing the right thing and running the correct clinical study, UniQure is performing a distorted or manipulated comparison in the mind of FDA.” In response, uniQure released a statement on social media claiming that "statements made by anonymous FDA sources to the press have been highly irregular, unprecedented, and are incomplete or entirely incorrect. We do not believe they reflect a fair and faithful reading of the documents we have submitted or those we have received from the agency."

6. Sangamo Adds Preclinical and Clinical Modules to Rolling BLA for Fabry Disease Gene Therapy Isa-Vec

Sangamo Therapeutics announced that it has submitted preclinical and clinical modules to the FDA for its rolling BLA for investigational Fabry disease gene therapy isaralgagene civaparvove (isa-vec, also known as ST-920). The company originally initiated submission of the rolling BLA in December 2025. Sangamo has previously noted that it expects the rolling BLA submission, which is taking place under the accelerated approval pathway, will be finished in the second quarter of 2026.