Around the Helix: Cell and Gene Therapy Company Updates – February 4, 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. Ocugen’s Gene Therapy OCU410ST Continues to Show Efficacy in Phase 1 Stargardt Disease Trial

Ocugen’s investigational adeno-associated virus (AAV)–based gene therapy OCU410ST demonstrated a +6-letter improvement in best-corrected visual acuity (BCVA) among BCVA-evaluable patients without confounders in the phase 1 GARDian1 clinical trial (NCT05956626). These findings, along with additional study results, were published in Eye.

2. Ultragenyx Puts BLA for MPS IIIA Gene Therapy UX111 in Front of FDA Again

Ultragenyx has resubmitted a biologics license application (BLA) for UX111 (rebisufligene etisparvovec), an AAV9 vector-based gene therapy intended to treat mucopolysaccharidosis type IIIA (MPS IIIA, also known as Sanfilippo syndrome), to the FDA. The new BLA includes “substantial longer-term data on multiple measures of neurologic benefit”, and is aimed at obtaining an accelerated approval for the gene therapy product. A previous BLA submission for UX111 was met with a complete response letter from the FDA in July 2025.

3. Pivotal Trial for Taysha's Rett Syndrome Gene Therapy TSHA-102 Doses First Patient

Taysha Gene Therapies has dosed the first patient in the pivotal phase 3 portion (Part B) of the REVEAL clinical trial (NCT05606614), which is assessing the investigational AAV vector–based gene therapy TSHA-102 for the treatment of Rett syndrome. The company reported that the initial dosing occurred in the fourth quarter of 2025.

4. Spur’s Gaucher Disease Gene Therapy FLT201 Improves Lyso-Gb1 Levels in Phase 1/2 Data

Newly updated data from Spur Therapeutics’ phase 1/2 GALILEO-1 clinical trial (NCT05324943) and GALILEO-2 long-term follow-up study (NCT06545136), which are evaluating the adeno-associated virus (AAV) vector-based gene therapy avigbagene parvec (also known as FLT201) for the treatment of Gaucher disease, have shown that patients experienced “rapid and sustained” decreases in levels of glucosylsphingosine (lyso-Gb1), a biomarker of disease severity, at 2 years posttreatment.

5. Wave Reassumes the Reigns for AATD RNA Editing Program

Wave Life Sciences has regained full rights to WVE-006, an investigational RNA editing oligonucleotide intended to treat alpha-1 antitrypsin deficiency (AATD), from its collaborator GSK, after the 2 companies agreed that Wave is “well-placed” to advance the program. The therapy is currently being evaluated in the phase 1b/2a RestorAATion-2 clinical trial (NCT06405633), and Wave intends to discuss potential for an accelerated approval pathway for the product with the FDA.

6. Anixa’s CER-T Therapy Gets USAN Council Name

Anixa Biosciences’ novel follicle-stimulating hormone receptor–targeting chimeric antigen receptor (CAR)/chimeric endocrine receptor (CER) T-cell therapy, which is being evaluated in a phase 1 clinical trial (NCT05316129) for the treatment of ovarian cancer, has received approval for the nonproprietary name “liraltagene autoleucel (lira-cel)” from the United States Adopted Names (USAN) Council. "The assignment of the nonproprietary name in the US represents an important step in the development and potential future commercialization of our CAR-T therapy,” Amit Kumar, PhD, the chairman and CEO of Anixa, said in a statement.