Around the Helix: Cell and Gene Therapy Company Updates – October 8, 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. Taysha Snags FDA Breakthrough Therapy Designation for Rett Syndrome Gene Therapy TSHA-102

Taysha Gene Therapies’ TSHA-102, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat Rett syndrome, has been granted breakthrough therapy designation by the FDA.

2. Rocket Pharmaceuticals Pulls BLA for Fanconi Anemia Gene Therapy RP-L102

Rocket Pharmaceuticals’ has voluntarily withdrawn the biologics license application (BLA) it had previously submitted to the FDA for mozafancogene autotemcel (also known as fana-cel and RP-L102), an investigational gene therapy intended to treat Fanconi anemia.

3. Orca Bio’s BLA for Hematologic Malignancy Cell Therapy Orca-T Accepted by FDA With Priority Review

Orca Bio’s biologics license application (BLA) for Orca-T, an investigational allogeneic cell therapy productintended to treat various hematologic malignancies including acute myeloid leukemia, acute lymphoblastic leukemia, and myelodysplastic syndromes, has been accepted by the FDA with priority review.

4. Avobis Bio's Crohn Disease Cell Therapy AVB-114 Garners FDA RMAT Designation

Avobis Bio's AVB-114, an investigational implantable cell therapy product, has received regenerative medicine advanced therapy (RMAT) designation from the FDA for the treatment of Crohn disease perianal fistulas. AVB-114 has previously been evaluated in the phase 1 STOMP-I clinical trial (NCT01915927) and is currently being evaluated in the phase 2 STOMP-II clinical trial (NCT04847739). Notably, in STOMP-I, at 12 months posttreatment, patients with persistent perianal Crohn disease showed 76% drainage cessation.

5. Moonlight Bio to Leverage MaxCyte's Tech for Solid Tumor Cell Therapy

Under a new strategic platform license agreement, Moonlight Bio has gained nonexclusive rights to utilize MaxCyte’s Flow Electroporation technology and ExPERT platform for development and manufacturing purposes for its engineered T-cell therapy programs aimed at treating solid tumors. “We’re proud to partner with Moonlight Bio as they advance their gene-edited cell therapies through clinical development,” Maher Masoud, JD, the president and CEO of MaxCyte, said in a statement. “Our globally supported, good manufacturing practice-ready and regulatory-proven ExPERT platform is built to accelerate clinical and commercial manufacturing and enable robust, scalable cell engineering."

6. Verismo Therapeutics’ Trial for CAR-T SynKIR-310 Doses First Patient With Follicular Lymphoma

The first patient with follicular lymphoma (FL) has been dosed in Verismo Therapeutics’ phase 1 CELESTIAL-301 clinical trial (NCT06544265), which is evaluating SynKIR-310, a chimeric antigen receptor T-cell (CAR-T) therapy based on the company's killer immunoglobulin-like receptor (KIR)-CAR platform, in patients with various types of relapsed/refractory B-cell nonHodgkin lymphoma. Verismo emphasized that the milestone strengthens its relationship with the Institute for Follicular Lymphoma Innovation, which made a strategic investment in the the program earlier this year with the intent of expanding FL-focused trial sites.