Around the Helix: Cell and Gene Therapy Company Updates – November 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. PreBLA Meeting With FDA Sinks uniQure’s Hopes for Approval of Huntington Disease Gene Therapy AMT-130 Based Primarily on Phase 1/2 Data

uniQure has announced that based on feedback it received during a recent prebiologics license application (preBLA) meeting with the FDA, it no longer believes that the agency will find data from phase 1/2 studies compared to an external control sufficient to support a BLA for AMT-130, an adeno-associated virus vector-based gene therapy intended to treat Huntington disease.

2. Quarter Century Update: Thoughts on Reducing Costs of Goods

In this episode, Deborah Phippard, PhD, the chief scientific officer of Precision for Medicine, shared her thoughts with Renier Brentjens, MD, PhD, the chair of the department of medicine and the deputy director at Roswell Park Comprehensive Cancer Center, about ways the field might reduce costs and advance cell and gene therapy over the next 25 years.

3. FDA Activity Recap: October 2025 Features New Platform Technology Designation, RDEA Pilot Program Selection, and More

Last month the FDA granted platform technology designation to Krystal Biotech for the genetically modified, nonreplicating herpes simplex virus type 1 viral vector used in its gene therapy KB801, selected Abeona Therapeutics’ ABO-503 for its Rare Disease Endpoint Advancement (RDEA) Pilot Program, and took a number of other important actions.

4. Quarter Century Update: The Impact of Artificial Intelligence

In this final episode of the Special Report, Brentjens and Phippard discuss the emerging and hard to predict role of artificial intelligence in advancing cell and gene therapies.

5. Caribou's CAR-T CB-010 Vispa-Cel Shows 86% ORR in Patients Who Received it With Optimized Profile

New data from Caribou Biosciences' phase 1 ANTLER clinical trial (NCT04637763) has shown that vispacabtagene regedleucel (vispa-cel; formerly known as CB-010), and allogeneic chimeric antigen receptor T-cell (CAR-T) therapy, achieved an 86% overall response rate (ORR) in a cohort of 35 patients with relapsed/refractory B-cell nonHodgkin lymphoma who received the CAR-T with an optimized profile, which was generated from young donor-derived T cells. Caribou pointed out that the patients in this cohort matched at least 2 HLA alleles with the donor.

6. Sarepta's ESSENCE Trial Fails to Hit Statistical Significance on Primary End Point

Sarepta Therapeutics revealed that its phase 3 ESSENCE clinical trial (NCT02500381), which is evaluating its phosphorodiamidate morpholino oligomer therapies AMONDYS 45 and VYONDYS 53 for the treatment of a subset of Duchenne muscular dystrophy cases, showed a .05 steps/second in least square means difference on the 4-step ascend velocity test between treated patients and patients who received placebo (P = .309) at 96 weeks, which constituted the study's primary end point. Although the result was not statistically significant, Sarepta stated that "numerical trends favored treatment versus placebo."