Around the Helix: Cell and Gene Therapy Company Updates – August 13, 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. Sensorion Finishes Enrolling Patients in Second Cohort of Its Phase 1/2 Trial for Congenital Deafness Gene Therapy SENS-501

Sensorion has completed patient enrollment for the second cohort of its phase 1/2 Audiogene clinical trial (NCT06370351) evaluating SENS-501 (OTOF-GT), a gene therapy candidate for otoferlin gene (OTOF)-related congenital deafness, one of the most common forms of congenital deafness.

2. Capricor Locks in Type A Meeting With the FDA to Discuss the Future of DMD Cardiomyopathy Cell Therapy Candidate Deramiocel

Capricor Therapeutics has scheduled a Type A meeting with the FDA to discuss future regulatory moves for Deramiocel (also known as CAP-1002), an investigational allogeneic cardiosphere-derived cell therapy intended for the treatment of Duchenne muscular dystrophy (DMD) cardiomyopathy.

3. FDA Updates Labeling for bluebird's Active Cerebral Adrenoleukodystrophy Gene Therapy Eli-Cel to Reflect Risk of Hematologic Malignancy

The FDA is requiring updated labeling for bluebird bio's elivaldogene autotemcel (eli-cel; marketed as Skysona), a gene therapy product comprised of engineered autologous CD34+ hematopoietic stem cells intended to treat early, active cerebral adrenoleukodystrophy (CALD), to reflect an increased risk of hematologic malignancy following treatment with the product.

4. AAVantgarde’s Stargardt Gene Therapy AAVB-039 Garners FDA Fast Track Designation

AAVantgarde Bio’s AAVB-039, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat Stargardt disease, has received fast track designation from the FDA.

5. NextRNA Therapeutics to Shutter its Doors

NextRNA, which was founded in 2021 with the intent of applying long noncoding RNAs (lncRNAs) to the treatment of disease, will be shutting down, according to a LinkedIn post made by Dominique Verhelle, PhD, the cofounder, president, and CEO of the company. "We made important steps toward understanding the role of lncRNAs in diseases like cancer and how to therapeutically target them to address significant unmet needs... I believe our efforts will help pave the way for others to explore this exciting space and its potential for patients."

6. EMA's CHMP Gives Ocugen the Green Light to Submit MAA for OCU410ST Based on GARDian3 Trial

The European Medicines Agency (EMA)'s Committee for Medicinal Products for Human Use (CHMP) has provided Ocugen with a positive opinion regarding the use of data from the phase 2/3 GARDian3 clinical trial (NCT05956626) to support a marketing authorization application (MAA) for OCU410ST, an investigational adeno-associated virus (AAV) vector-based gene therapy, for the treatment of Stargardt disease. “This positive opinion endorses a single trial as the basis for both biologics license application and MAA submissions and brings us closer to providing a one-time, modifier gene therapy to approximately 100,000 Stargardt patients in the United States and Europe combined,” Shankar Musunuri, PhD, the chairman, chief executive officer, and cofounder of Ocugen, said in a statement.