Around the Helix: Cell and Gene Therapy Company Updates – April 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. FDA Approves Sanofi’s siRNA Therapy Fitusiran for the Treatment of Hemophilia A and B

The FDA has approved Sanofi's fitusiran, an siRNA therapeutic intended to lower antithrombin, for use as routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients aged 12 years and older with hemophilia A or hemophilia B, with or without factor VIII or IX inhibitors. The therapy will be marketed under the name Qfitlia.

2. Sarepta’s DMD Gene Therapy Elevidys Demonstrates Manageable Safety Across 5-Year Timespan

Sarepta Therapeutics’ delandistrogene moxeparvovec-rokl (marketed as Elevidys), a marketed adeno-associated virus ( rAAVrh74) vector-based gene therapy intended to slow progression of Duchenne muscular dystrophy (DMD) through delivery of a microdystrophin transgene, showed consistent safety outcomes across a broad population of patients with the disease.

3. National Colorectal Cancer Awareness Month 2025: Looking Back at Progress for Cell Therapy

In honor of National Colorectal Cancer Awareness Month, observed annually in March by the patient and clinician communities, CGTLive took a look back at the progress that was made for cell therapy in colorectal cancer over the past year.

4. Urgency of Funding and Equity in Neuromuscular Research and Care

Recent federal funding cuts have significantly affected healthcare organizations, such as the National Institutes of Health (NIH) and the FDA. Concerns have been raised by several leaders of academic institutions that such funding cuts endanger ongoing health research and could hold back innovation in the field. CGTLive®'s sister site NeurologyLive^® sat down with Robert Califf, MD, MACC, a cardiologist and former FDA commissioner, to discuss the role of funding in neuromuscular disease research.

5. Nkarta Cuts Staff

Nkarta, which is focused on the development of natural killer cell therapies, announced that it would be undergoing a restructuring that will include cutting 53 of its staff positions, which constitutes 34% of its workforce. In addition, some future hiring is being put on hold by the company. “We believe that this decision is necessary in today’s challenging financial and competitive environment to fulfill Nkarta’s vision of bringing potentially life-saving cellular therapies to people with autoimmune disease," Paul J. Hastings, the CEO of Nkarta, said in a statement.

6. Dose-Limiting Toxicity Reported in Trial for Cabaletta's Rese-Cel

A grade 3 case of immune effector cell-associated neurotoxicity syndrome, which constituted a dose-limiting toxicity, occurred in a patient treated with Cabaletta Bio’s resecabtagene autoleucel (rese-cel, formerly referred to as CABA-201), an investigational CD19-directed chimeric antigen receptor T-cell (CAR-T) therapy. The event occurred in the context of the phase 1/2 RESET-SSc clinical trial (NCT06328777) evaluating the therapy for systemic sclerosis (SSc). It was noted that the patient experienced a fever 3 days prior to the administration of rese-cel, which was not communicated to the Cabaletta Medical Monitor before treatment took place.

7. Peter Marks, MD, PhD, Departs from the FDA

Peter Marks, MD, PhD, who had been serving as the director of the Center for Biologics Evaluation and Research at the FDA, has resigned from his position. Disagreements with Robert F. Kennedy, the secretary of Health and Human Services, were key in his decision, according to a letter he sent to the Acting FDA Commissioner. During his time with the FDA, Marks worked to advance the development of gene therapy as an accessible treatment modality for patients, among other efforts. “My keynote address really focused on how we can make gene therapy for rare disorders into a reality," Marks previously told CGTLive in a 2023 interview at the Muscular Dystrophy Association Clinical & Scientific Conference, held March 19-22 of that year, in Dallas, Texas.