Around the Helix: Cell and Gene Therapy Company Updates – May 28, 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 Dies After Treatment With Rocket Pharmaceuticals’ Danon Disease Gene Therapy RP-A501 in Phase 2 Trial

A patient has passed away after treatment with Rocket Pharmaceuticals’ RP-A501, an investigational adeno-associated virus serotype 9 (AAV9) vector-based gene therapy intended to treat Danon disease, in the context of a pivotal phase 2 clinical trial (NCT06092034).

2. Ocugen’s Gene Therapy OCU410ST Nets Rare Pediatric Disease Designation for ABCA4-Associated Retinopathies

Ocugen has received rare pediatric disease designation from the FDA for OCU410ST, an investigational AAV vector-based gene therapy, for the treatment of ABCA4-associated retinopathies, including Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3.

3. Global Cell and Gene Therapy: The Next Wave of Biotechnology Innovation

In the United States and the United Kingdom (UK), cell and gene therapy (CGT) is reshaping treatment paradigms and setting the stage for the next wave of biotechnology innovation. As CGT continues to evolve, innovation is set to expand the reach of these therapies, offering new solutions for treating a broader range of complex diseases and changing how we approach global patient care.

4. National Cancer Institute Tests TCR-Transduced T-Cell Therapy for Solid Tumors in Phase 2 Trial

The National Cancer Institute (NCI) is currently evaluating T-cell receptor (TCR)-transduced T-cell therapy for the treatment of various kinds of solid tumors in a phase 2 clinical trial (NCT03412877). For this month's installment of Clinical Trials in Progress, CGTLive® has decided to take a closer look at this ongoing study.

5. Genflow and CER Groupe Elevate Partnership

Genflow Biosciences and CER Groupe have signed a Master Service Agreement (MSA), bring their collaboration to a higher level. Under the MSA, CER will engage in development, production, and characterization for Genflow gene therapy products, among other activities. "Our partnership with CER has been instrumental in supporting our R&D efforts to date," Eric Leire, MD, the CEO of Genflow, said in a statement. "Formalizing this collaboration through the MSA will enable greater operational alignment and strategic agility as we continue to accelerate our pipeline."

6. MHRA Gives Sarepta Go Ahead to Continue Dosing in Elevidys Trial

The UK's Medicines & Healthcare products Regulatory Agency (MHRA) has cleared Sarepta to continue dosing patients in the phase 3 ENVISION clinical trial evaluating delandistrogene moxeparvovec-rokl (marketed as Elevidys), a gene therapy intended to treat Duchenne muscular dystrophy. The trial had been paused after an announcement made on March 18, 2025, that a patient, identified as a young man, who had been treated with Elevidys in the United States, had died.

7. CARsgen Snags Priority Review for CAR-T Satri-Cel in China

CARsgen's Claudin18.2-directed chimeric antigen receptor T-cell (CAR-T) satricabtagene autoleucel (satri-cel), which is intended to treat gastric/gastroesophageal junction adenocarcinoma (G/GEJA), has been granted priority review by China's National Medical Products Administration (NMPA) for Claudin18.2-positive advanced G/GEJA in patients who have received at least 2 prior lines of unsuccessful therapy. The therapy is currently being evaluated in several trials in China and North America.

8. Results From Trial Evaluating AskBio's Parkinson Disease Gene Therapy Published in Movement Disorders

Complete results from a phase 1b clinical trial (NCT04167540) evaluating Asklepios BioPharmaceutical (AskBio)'s AB-1005 (AAV2-GDNF), an investigational AAV vector serotype 2 (AAV2)-based gene therapy intended to treat Parkinson disease (PD), have been published in the International Parkinson and Movement Disorder Society's peer-reviewed journal Movement Disorders. Among the findings were results showing improvement in clinical assessments of motor function at 18 months posttreatment in the study's "moderate" cohort.