Around the Helix: Cell and Gene Therapy Company Updates – July 2, 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. Survival Outcomes Found to Be Comparable for Axi-Cel, Liso-Cel, and Tisa-Cel in Real World DLBCL Data

In a retrospective, observational study, patients with diffuse large B-cell lymphoma (DLBCL) who were treated with tisagenlecleucel (tisa-cel; Kymriah), axicabtagene ciloleucel (axi-cel; Yescarta), or lisocabtagene maraleucel (liso-cel; Breyanzi) across their FDA-approved indications did not show statistically significant differences in overall survival (OS) (log-rank test: χ², 0.773; degrees of freedom [df], 1; P = .379).¹ The data were presented in a poster at the European Hematology Association (EHA) 2025 Congress, held June 12 to 15, both virtually and in Milan, Italy.

2. Barriers to Access for Gene Therapy in β-Thalassemia

Tami John, MD, a clinical associate professor at Stanford Medicine, spoke about the current landscape of care for TDT, how gene therapy products fit into that landscape, and the ongoing issues with accessibility for these new products.

3. FDA Drops REMS Requirement for 6 Approved CAR T-Cell Therapies

The FDA is no longer requiring Risk Evaluation and Mitigation Strategies (REMS) for the 6 CD19/BCMA-directed chimeric antigen receptor T-cell (CAR-T) therapies, which include Bristol Myers Squibb (BMS) and 2seventybio’s idecabtagene vicleucel (ide-cel; marketed as Abecma); BMS’s lisocabtagene maraleucel (liso-cel; marketed as Breyanzi); Janssen and Legend Biotech’s ciltacabtagene autoleucel (cilta-cel; marketed as Carvykti); Novartis’s tisagenlecleucel (tisa-cel; marketed as Kymriah); Kite Pharma’s brexucabtagene autoleucel (brexu-cel; marketed as Tecartus); and Kite Pharma’s axicabtagene ciloleucel (axi-cel; marketed as Yescarta). The FDA made the move with the view that the REMS are no longer necessary to ensure a favorable risk-benefit-ratio for the products and that dropping the requirement may increase accessibility to these therapies.

4. Neurogene Aligns With FDA on Registrational Trial Design for Rett Syndrome Gene Therapy NGN-401

Neurogene has announced details regarding a registrational trial for NGN-401, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat Rett syndrome, key elements of the design of which were agreed upon with the FDA in the context of the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program.

5. Rocket Snags IND Clearance for BAG3-DCM Gene Therapy Trial

Rocket Pharmaceuticals has received clearance from the FDA of an investigational new drug application (IND) for RP-A701, an investigational AAV vector-based gene therapy intended to treat BAG3-associated dilated cardiomyopathy (BAG3-DCM). The IND clearance enables the company to start a first-in-human phase 1 clinical trial for the gene therapy. "Phase 1 trial start-up activities are currently underway for RP-A701, and we are working towards treating the first patient," Kinnari Patel, PharmD, MBA, the president, head of R&D, and chief operating officer at Rocket Pharma, said in a statement.

6. China's NMPA Accepts Satri-Cel NDA for Filing

CARsgen's new drug application (NDA) for 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 accepted for 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. "This marks the world's first CAR T-cell therapy product for solid tumors to reach the NDA stage—a major milestone for the CAR-T field," Zonghai Li, MD, PhD, the founder, chairman of the board, CEO, and chief scientific officer of CARsgen, said in a statement.

7. SineuGene Garners Orphan Drug Designation for ALS Gene Therapy

SineuGene's SNUG01, an AAV vector-based gene therapy being evaluated for the treatment of amyotrophic lateral sclerosis (ALS), has been granted orphan drug designation by the FDA. The gene therapy product previously received clearance of an IND application from the agency in March of this year, and SineuGene plans to launch a phase 1/2a clinical trial for the candidate that will take place at sites in the United States and China.

8. Beacon Therapeutics Licenses AAV Capsid From Abeona

Beacon Therapeutics has exercised an option provided by a 2024 agreement to license Abeona Therapeutics' AAV204 capsid. Beacon intends to use the capsid in gene therapy candidates for retinal diseases. “Beacon’s option exercise further validates AAV204’s potential to enable targeted delivery of gene therapies in rare and prevalent ophthalmic diseases,” Madhav Vasanthavada, PhD, MBA, the chief commercial officer and head of business development at Abeona Therapeutics, said in a statement. “Our nonexclusive agreement with Beacon enables us to fully explore the therapeutic value of AAV204 in additional ophthalmic diseases.”