Around the Helix: Cell and Gene Therapy Company Updates – April 30, 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. FDA Approves Abeona Therapeutics' Epidermolysis Bullosa Gene Therapy Pz-Cel

The FDA has approved Abeona Therapeutics' prademagene zamikeracel (pz-cel; EB-101), an autologous gene-corrected epidermal sheet therapy, for the treatment of patients with recessive dystrophic epidermolysis bullosa. The therapy will be marketed under the name Zevaskyn.

2. The Evolving Landscape of Gene and Cell Therapies in Parkinson Disease: Pipeline Highlights

Cell and gene therapies are rapidly transforming the treatment paradigm for Parkinson disease (PD), with several investigational agents targeting underlying disease mechanisms such as lysosomal dysfunction, α-synuclein pathology, and dopaminergic neuronal degeneration. These innovative strategies span a range of modalities—from viral vector-based gene therapies to stem cell transplants and immunotherapies—with the potential to not just treat symptoms but also slow or halt disease progression.

3. Evaluating Capricor’s Cardiomyopathy Cell Therapy Deramiocel for DMD

Capricor Therapeutics' Deramiocel, a potentially-adjuvant allogeneic cardiosphere-derived cell therapy intended to treat Duchenne muscular dystrophy (DMD), has previously been evaluated in the HOPE-2 clinical trial (NCT03406780) and is currently being evaluated in HOPE-2’s ongoing open-label extension (OLE) study (HOPE-2-OLE; NCT04428476). Notably, a biolgics license application for the product is currently under review by the FDA. CGTLive® interviewed Linda Marbán, PhD, the CEO of Capricor Therapeutics, to learn more about the cell therapy and the clinical results.

4. Exploring New Pathways in Muscular Dystrophy Research: From Fibrosis to Function

A series of FDA approvals and clinical advances reflecting a shift toward disease-modifying and potentially gene-correcting therapies have significantly expanded the treatment landscape for Duchenne muscular dystrophy (DMD) in recent years. The field is still trying to find new ways to treat muscular dystrophies despite the influx of newly approved therapeutics, however. Matthew Alexander, PhD, an associate professor of pediatric neurology and genetics at the University of Alabama Birmingham, sat down with CGTLive®'s sister site NeurologyLive® to discuss this topic.

5. Ray Therapeutics Snags CIRM Grant for Retinitis Pigmentosa Gene Therapy

Ray Therapeutics has received an $8 million grant from the California Institute for Regenerative Medicine (CIRM) to support clinical development of its optogenetic gene therapy, RTx-015. “Retinitis pigmentosa remains a devastating condition with no approved treatments for the vast majority of patients,” Paul Bresge, the CEO and cofounder of Ray, said in a statement. “We are deeply grateful to CIRM for their belief in our science and their continued support of our programs."

6. FDA Gives Green Light to Additional Manufacturing Facility for Ferring's Bladder Cancer Gene Therapy

The FDA has approved an additional manufacturing facility, located in in Parsippany, New Jersey, for the production of Ferring Pharmaceuticals' nadofaragene firadenovec (marketed as Adstiladrin), a gene therapy currently approved for the treatment of adult patients with high-risk, Bacillus Calmette-Guerin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. Notably, the decision activated a $200 million royalty payment to Ferring based on a previous agreement with Royalty Pharma.

7. Genprex and New York University Langone Health Pen Agreement for Patent Exclusivity

Genprex has secured an exclusive patent license from NYU Langone for quaratusugene ozeplasmid (Reqorsa), an investigational immunogene therapy being evaluated for the treatment of mesothelioma. “We are pleased to expand our robust oncology franchise, intellectual property portfolio and research program for Reqorsa to continue exploring how it may serve as a therapeutic treatment for some of the most difficult to treat cancers and diseases,” Thomas Gallagher, Esq, the senior vice president of intellectual property and licensing at Genprex, said in a statement. “We believe Reqorsa has the potential to treat mesothelioma, and this license agreement positions Genprex to expand its clinical development pipeline with future clinical studies that are within the scope of the licensed patent.”

8. Caribou to Make Layoffs, Drops Lupus CAR-T Trial

Caribou Biosciences will be cutting about 32% of its staff and discontinuing a clinical trial evaluating chimeric antigen receptor T-cell (CAR-T) therapy CB-010 in lupus and another trial evaluating CAR-T CB-012 in relapsed/refractory acute myeloid leukemia. The moves are part of the company's efforts to strategically prioritize its pipeline, with a focus on advancing CB-010 and and CAR-T therapy CB-011 in oncology indications. "Caribou’s 2 lead phase 1 clinical programs, CB-010 for large B-cell lymphoma and CB-011 for multiple myeloma, continue to demonstrate encouraging efficacy and have the potential to serve this critical unmet need for individuals living with hematologic malignancies,” Rachel Haurwitz, PhD, president and CEO of Caribou, said in a statement.