Around the Helix: Cell and Gene Therapy Company Updates – October 15, 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. BLA for Rocket’s LAD-I Gene Therapy Under Review by FDA Again

The FDA has accepted Rocket Pharmaceuticals’ resubmission of a biologics license application (BLA) for marnetegragene autotemcel (RP-L201, to be marketed as Kresladi), a genetically modified autologous hematopoietic stem cell therapy intended to treat leukocyte adhesion deficiency type 1 (LAD-I).

2. FDA Picks Abeona’s XLRS Gene Therapy ABO-503 for RDEA Pilot Program

Abeona Therapeutics’ ABO-503, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat X-linked retinoschisis (XLRS), has been chosen by the FDA for its Rare Disease Endpoint Advancement (RDEA) Pilot Program.

3. Regeneron’s Gene Therapy DB-OTO Improves Speech Perception in Children With Otoferlin-Related Hearing Loss

Regeneron’s DB-OTO, an investigational AAV dual-vector-based gene therapy intended to treat otoferlin (OTOF)-related hearing loss, has improved hearing and speech perception in pediatric patients treated in the phase 1/2 CHORD clinical trial (NCT05788536), according to updated data reported by thecompany at the annual American Academy of Otolaryngology-Head and Neck Surgery (AAO-HNSF) meeting, held October 11 to 14 in Indianapolis, Indiana, and simultaneously published in the New England Journal of Medicine (NEJM).

4. Krystal Biotech’s Neurotrophic Keratitis Gene Therapy Viral Vector Secures FDA Platform Technology Designation

Krystal Biotech has received a platform technology designation from the FDA for the genetically modified, nonreplicating herpes simplex virus type 1 viral vector used in KB801, an investigational gene therapy intended to treat neurotrophic keratitis.

5. Tr1X's Multiple Sclerosis Cell Therapy Cleared for US Trial

The FDA has cleared an investigational new drug (IND) application for Tr1X's TRX319, an allogeneic chimeric antigen receptor (CAR)-engineered type 1 regulatory (Tr1 Treg) cell therapy intended to treat progressive multiple sclerosis. Alongside the IND clearance announcement, Tr1X also announced that it has received $50 million in venture financing that will support its clinical trial activities.

6. Saudi Arabia's KFSHRC Speeds Up Local Cell Therapy Treatment

King Faisal Specialist Hospital & Research Centre (KFSHRC), located in Riyadh, Saudi Arabia, has made improvements to its local CAR T-cell production capabilities, and can now manufacture genetically modifed T-cells in about 14 days. Furthermore, local production is substantially cheaper per patient than it was to import product from out of the country.