Around the Helix: Cell and Gene Therapy Company Updates – February 5, 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. uniQure Makes Progress in Trial for SOD1-ALS Gene Therapy AMT-162

An independent data monitoring committee (IDMC) has given uniQure the green light to begin enrolling patients in the second dose cohort of the phase 1/2 EPISOD1 clinical trial (NCT06100276), which is evaluating AMT-162, an investigational adeno-associated virus (AAV) vector-based gene therapy, for the treatment of amyotrophic lateral sclerosis caused by mutations in superoxide dismutase 1 (SOD1-ALS).

2. United Therapeutics’ Gene-Edited Kidney Xenotranspant UKidney Cleared for US Trial

United Therapeutics’ UKidney, an investigational gene-edited porcine kidney product intended to treat end-stage renal disease (ESRD) via xenotransplant, has received clearance of an investigational new drug (IND) application from the FDA.

3. Spur Therapeutics’ Gaucher Disease Gene Therapy FLT201 Continues to Show Safety and Efficacy in Phase 1/2 Trial

Spur Therapeutics’ (formerly known as Freeline Therapeutics) FLT201, an adeno-associated virus (AAV) vector-based gene therapy, has continued to show safety and efficacy in data from the phase 1/2 GALILEO-1 clinical trial (NCT05324943) evaluating the therapy for the treatment of Gaucher disease.

4. Top News in Oncology Cell Therapy for World Cancer Day 2025

For World Cancer Day, held annually on February 4, take a look at the stories that have stood out in oncology cell therapy in recent months.

5. Gradalis' Personalized Immunotherapy Gemogenovatucel-T Snags FDA RMAT Designation

Gradalis' Gemogenovatucel-T (also known as Vigil), an investigational personalized cellular immunotherapy being evaluated for the treatment of ovarian cancer, has received regenerative medicine advanced therapy (RMAT) designation from the FDA. The product is currently being evaluated in the phase 2b VITAL clinical trial (NCT02346747) in patients with newly diagnosed, advanced Stage IIIb/IV ovarian cancer. Results from the ongoing study informed the FDA's decision.

6. Adicet's T-Cell Therapy ADI-100 Fast Tracked by FDA for Refractory SLE

Adicet Bio’s ADI-100, its investigational allogeneic chimeric antigen receptor (CAR)-engineered gamma delta T-cell therapy being evaluated for the treatment of various autoimmune diseases, has received FDA fast track designation for refractory systemic lupus erythematosus (SLE) with extrarenal involvement. The therapy is currently being evaluated in a phase 1 clinical trial (NCT06375993) for patients with SLE, systemic sclerosis, antineutrophil cytoplasmic autoantibody associated vasculitis, idiopathic inflammatory myopathy, and stiff person syndrome.

7. BMS's Breyanzi Garners Positive CHMP Opinion for R/R Follicular Lymphoma

Bristol Myers Squibb (BMS)’s Type II variation application for lisocabtagene maraleucel (liso-cel, marketed as Breyanzi), an autologous CD19-directed CAR T-cell therapy, has received a recommendation for approval in relapsed/refractory (r/r) follicular lymphoma (FL) from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP). Specifically, the recommendation pertains to a potential indication for adults with r/r FL who have received 2 or more prior lines of systemic therapy. “This is another important step in our commitment to delivering Breyanzi to more patients across indications, as well as expanding into new regions, especially for diseases with continued unmet need such as relapsed or refractory FL, which is considered incurable," Anne Kerber, senior vice president, head of Late Clinical Development, Hematology, Oncology and Cell Therapy, at BMS, said in a statement.

8. CIRM Grants Tenaya $8 Million for Arrhythmogenic Right Ventricular Cardiomyopathy Gene Therapy

Tenaya Therapeutics has received a CLIN2 grant for $8.0 million from the California Institute for Regenerative Medicine (CIRM). The funding from the grant will go towards the company's phase 1b RIDGE-1 clinical trial (NCT06228924) evaluating TN-401, an investigational AAV vector-based gene therapy, for the treatment of PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC). "RIDGE-1 is actively enrolling patients at leading centers, and we look forward to sharing initial data from the low-dose cohort in the second half of this year," Faraz Ali, MBA, the chief executive officer of Tenaya, said in a statement.