Around the Helix: Cell and Gene Therapy Company Updates – January 7, 2026

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. Ultragenyx Finishes Rolling Submission of BLA for Glycogen Storage Disease Gene Therapy DTX401

Ultragenyx has completed its submission of a rolling biologics license application (BLA) to the FDA for DTX401, an investigational adeno-associated virus serotype 8 (AAV8) vector gene therapy expressing the human G6PC gene that is intended to treat Glycogen Storage Disease Type Ia (GSDIa).

2. The Impact of Artificial Intelligence on Cell and Gene Therapy

Deborah Phippard, PhD, the chief scientific officer of Precision for Medicine, and Renier Brentjens, MD, PhD, the chair of the department of medicine and the deputy director at Roswell Park Comprehensive Cancer Center, discussed their thoughts on the potential impact of artificial intelligence (AI) on the field of cell and gene therapy.

3. What buzzwords shaped immunotherapy research and care in 2025?

ImmunoLogic cohosts Janna Minehart, MD, and Joseph Fraietta, PhD, shared their thoughts on some of the key words and terms in the field of oncology immunotherapy during the past year.

4. Sangamo Starts Putting BLA for Fabry Disease Gene Therapy Isa-Vec in Front of FDA

Sangamo Therapeutics has begun submission of a rolling BLA application to the FDA for investigational Fabry disease gene therapy isaralgagene civaparvove (isa-vec, ST-920).

5. TGFBI Corneal Dystrophy Genome-Editing Therapy Cleared for US Trial

GenEditBio’s GEB-101, an investigational in vivo genome-editing therapy intended to treat corneal dystrophy related to mutations in the TGFBI gene, has received clearance of an investigational new drug (IND) application from the FDA. In light of the IND clearance, GenEditBio plans to launch a phase 1/2 clinical trial entitled CLARITY, with enrollment activities expected to begin in the second quarter of 2026.

6. Autolus to test Manufacturing Obe-Cel With Cellares Platform

Autolus Therapeutics announced that it will be evaluating whether Cellares’ Cell Shuttle platform can provide automated manufacturing of obecabtagene autoleucel (Aucatzyl; Autolus), also known as obe-cel, a marketed CD19-directed genetically modified autologous T-cell immunotherapy for relapsed/refractory (R/R) B-cell precursor acute lymphoblastic leukemia (B-ALL). “If a commercial CAR T shows durable benefit, the next question is whether the industry can produce enough doses at a sustainable cost,” Fabian Gerlinghaus, the cofounder and CEO of Cellares, said in a statement. “Autolus has already built a strong manufacturing foundation. Our role is to extend that foundation with a global infrastructure that can reduce cost and process failure rates, while meeting total patient demand.”