Around the Helix: Cell and Gene Therapy Company Updates – June 18, 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. Second Patient Dies Following Treatment With Sarepta’s DMD Gene Therapy Elevidys

A second patient has died following treatment with Sarepta Therapeutics’ delandistrogene moxeparvovec-rokl (Elevidys), a marketed adeno-associated virus (AAV) vector-based gene therapy intended to treat Duchenne muscular dystrophy (DMD), according to an announcement from the company.

2. Study Suggests β-Thalassemia Gene Therapy May Help Improve Growth in Pediatric Patients

A study suggested that gene therapy used to treat transfusion-dependent β-thalassemia may help improve growth by positively impacting height-for-age in pediatric patients. 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.

3. Beam’s Base-Editing HSC Therapy BEAM-101 Raises HbF Levels in Patients With Sickle Cell Disease

Beam Therapeutics presented new data from its phase 1/2 BEACON clinical trial (NCT05456880), which is evaluating the company’s investigational base-edited autologous hematopoietic stem cell (HSC) therapy BEAM-101 for the treatment of sickle cell disease (SCD). In an interview with CGTLive®, experts from Beam Therapeutics, including chief executive officer John Evans, MBA, weighed in on the updated results.

4. Dual-Target CD19/CD20 CAR T-Cell Therapy Achieves 63.6% Complete Remission Rate in R/R B-NHL

A dual-target mCD19/hCD20 chimeric antigen receptor T-cell (CAR-T) therapy has demonstrated a 63.6% complete remission (CR) rate in a study that treated 11 patients with relapsed/refractory (r/r) B-cell nonHodgkin lymphoma (B-NHL).

5. FDA Gives Green Light for Pivotal Trial for Ocugen's Stargardt Gene Therapy

Ocugen's OCU410ST, an investigational AAV vector-based gene therapy, has received clearance from the FDA for an investigational new drug (IND) application for a pivotal phase 2/3 clinical trial in Stargardt disease. OCU410ST is currently being evaluated for Stargardt disease in the phase 1/2 GARDian clinical trial (NCT05956626). In May 2025, the therapy was granted rare pediatric disease designation from the FDA for the treatment of ABCA4-associated retinopathies, including Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3.

6. Capsida Biotherapeutics Bringing Parkinson Disease Gene Therapy CAP-003 to Clinical Stage

Capsida Biotherapeutics' CAP-003, an intravenously delivered gene therapy candidate intended to treat Parkinson disease associated with GBA mutations (PD-GBA), has received clearance of an IND application from the FDA for a phase 1/2 first-in-human clinical trial (NCT07011771). “We recognize the urgency for new treatment approaches, so we are working diligently to initiate the phase 1/2 clinical trial for CAP-003 with the aim of dosing the first patient in the third quarter of this year," Swati Tole, MD, the chief medical officer of Capsida, said in a statement.

7. Second Edition of the EU CAR-T Handbook Hits the Web

EHA, the European Society for Blood and Marrow Transplantation, and the GoCART Coalition have released the second edition of the EU CAR-T Handbook, an open-access resource intended to provide information on recent developments in the field of CAR-T. The EU CAR-T Handbook is a key component of our educational efforts and will serve as the foundational material for the upcoming CAR-T Passport—a practical resource for clinicians, nurses, pharmacist and other healthcare professionals involved in CAR-T and other Immune Effector Cell (IECs) therapies," Christian Chabannon, MD, PhD, the editor-in-chief of the handbook, said in a statement.