Around the Helix: Cell and Gene Therapy Company Updates – July 23, 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. Sarepta Voluntarily Pauses All Shipments of DMD Gene Therapy Elevidys Several Days After Informal Request From FDA

Sarepta Therapeutics stated that it has voluntarily paused all shipments of delandistrogene moxeparvovec-rokl (Elevidys), its marketed adeno-associated virus (AAV) vector-based gene therapy for Duchenne muscular dystrophy (DMD), in the United States.

2. FDA Supports Pivotal Trial Expansion of ATSN-201 Gene Therapy for XLRS

Atsena Therapeutics has announced alignment with the FDA on the regulatory path for ATSN-201, an investigational gene therapy for X-linked retinoschisis (XLRS). The agreement enables the company’s ongoing phase 1/2 LIGHTHOUSE trial (NCT05878860) to expand into a phase 1/2/3 study, serving as a pivotal trial to support a future biologics license application (BLA).

3. UCSF Tests Lentiviral Vector-Based Gene Therapy for Artemis-Deficient SCID in Phase 1/2 Trial

University of California, San Francisco (UCSF) Children's Hospital is currently evaluating IND1711, an investigational autologous gene-corrected hematopoeitic stem cell therapy, for the treatment of Artemis-deficient severe combined immunodeficiency (ART-SCID) in a phase 1/2 clinical trial (NCT03538899). For this month's installment of Clinical Trials in Progress, CGTLive® decided to take a closer look at this active study.

4. FDA Revokes Sarepta’s Platform Technology Designation for Muscular Dystrophy Gene Therapy Vector Following Patient Deaths

Sarepta Therapeutics’ platform technology designation from the FDA for the AAVrh74 viral vector it uses in gene therapy products for multiple muscular dystrophy indications has been revoked by the agency. The FDA’s decision follows the recent death of a 51-year-old patient who was treated with Sarepta’s investigational gene therapySRP-9004 for nonambulant limb-girdle muscular dystrophy (LGMD) type 2D/R3, which uses the AAVrh74 serotype, in a phase 1 clinical trial (DISCOVERY; NCT01976091).

5. First Patient Dosed in Phase 2/3 GARDian3 Trial for Stargardt Gene Therapy OCU410ST

Ocugen has dosed the first patient in its phase 2/3 GARDian3 clinical trial (NCT05956626) evaluating OCU410ST (AAV5-hRORA), a novel modifier gene therapy candidate intended to treat Stargardt disease (ABCA4-associated retinopathies). “The adaptive design of this trial, including a masked interim analysis at 8 months on 24 subjects, enables us to efficiently evaluate early signals of efficacy and safety while optimizing study conduct," Huma Qamar, MD, MPH, the chief medical officer of Ocugen, said in a statement.

6. Sarepta Announces Plans to Let Go of 36% of Employees

In a July 16 announcement, Sarepta also revealed that it plans to reduce its workforce by 500 employees as part of a strategic restructuring. “Faced with environmental changes, we have decided to act decisively, implementing a focused strategy to ensure Sarepta remains a vibrant, financially enduring, patient-centric organization dedicated to improving the lives of those with rare genetic diseases,” Doug Ingram, JD, the president and chief executive officer of Sarepta, said in a statement.

7. Klotho Neurosciences Teams Up With AAVnerGene for Next-Generation Manufacturing

Klotho Neurosciences has entered a partnership with AAVnerGene Inc. with the goal of improving manufacturing for its gene therapy candidates, which includes KLTO-202, a secreted-Klotho (s-KL) promoter, gene, and delivery system (KLTO-202, or s-KL-AAV.myo) intended to treat amyotrophic lateral sclerosis (ALS), among other products. "...we announced previously that we have been looking for a more efficient, 'next-generation' method to manufacture several of our patented gene therapy candidates, and the 2 platform technologies offered by AAVnerGene will catapult our product candidates into the clinic at a faster pace, at a lower cost, a higher efficacy and purity compared to the current state-of-the-art AAV manufacturing processes," Joseph Sinkule, the CEO of Klotho, said in a statement.

8. European Commission Approves Obe-Cel for Adults With R/R B-Cell Precursor Acute Lymphoblastic Leukemia

The European Commission has granted marketing authorization to obecabtagene autoleucel (Aucatzyl; Autolus Inc), also known as obe-cel, a CD19-directed genetically modified autologous T-cell immunotherapy, for the treatment of adults aged 26 years and older with relapsed or refractory (r/r) B-cell precursor acute lymphoblastic leukemia. Notably, the therapy has been approved in the United States since late 2024.