Around the Helix: Cell and Gene Therapy Company Updates – January 21, 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 Posts Positive Preliminary Phase 2 ArMaDa Trial Data of OCU410

Ocugen has reported encouraging preliminary 12‑month findings from its phase 2 ArMaDa clinical trial evaluating OCU410 (AAV5‑RORA) as a potential treatment for geographic atrophy (GA) associated with dry age-related macular degeneration (dAMD).

2. AskBio’s Late-Onset Pompe Disease Gene Therapy AB-1009 Cleared for Phase 1/2 Trial

AskBio’s investigational new drug (IND) application for AB-1009, a gene therapy candidate intended to treat late-onset Pompe disease (LOPD), has been cleared by the FDA. In light of the IND clearance, AskBio has launched the phase 1/2 AB-1009 PROGRESS-GT LOPD clinical trial (NCT07282847). The company expects that the first patient will be enrolled in “early 2026”.

3. Phase 1b Trial for Solid Biosciences’ Friedreich Ataxia Gene Therapy SGT-212 Doses First Patient

The first patient has been dosed in the first-in-human phase 1b FALCON clinical trial (NCT07180355), which is evaluating Solid Biosciences’ SGT-212, an investigational adeno-associated virus (AAV) vector-based gene therapy product, for the treatment of FA. The open-label, dose-finding study will include patients with FA aged 18 to 40 years.

4. The Key Role of Manufacturing in Cell Therapy Success

ImmunoLogic cohosts Janna Minehart, MD, and Joseph Fraietta, PhD, discussed lessons learned from 2025 and predictions for 2026 with regard to cell therapy manufacturing.

5. Scribe Therapeutics Announces Plans for First-in-Human Trial for Epigenetic Silencing Therapy STX-1150

The company stated that it expects it will launch a trial for STX-1150 in hypercholesterolemia by the middle of this year. STX-1150 is intended to treat the condition by repressing PCSK9, thus in turn lowering levels of low-density lipoprotein cholesterol (LDL-C), via an epigenetic mechanism that does not alter the DNA itself. We designed STX-1150 to overcome many of the limitations of today’s lipid-lowering therapies through powerful epigenetic silencing, and to meaningfully change how cardiovascular risk is managed for millions of patients,” Benjamin Oakes, PhD, the cofounder and CEO of Scribe, said in a statement.

6. Wiskott-Aldrich Syndrome Therapy Waskyra Approved by European Commission

Etuvetidigene autotemcel (Waskyra; Fondazione Telethon ETS) a cell-based gene therapy for the treatment of Wiskott-Aldrich syndrome, has been approved by the European Commission. The therapy, which was developed by AGC Biologics’ client Fondazione Telethon, was originally approved by the FDA in December 2025. "We are extremely proud of the FDA approval and the positive Committee for Medicinal Products for Human Use opinion for Waskyra—milestones made possible also thanks to the high-quality services provided by AGC Biologics,” Celeste Scotti, MD, PhD, the head of research and development at Fondazione Telethon, said in a statement. “Their expertise in producing lentiviral vectors and genetically modified cells has been instrumental in bringing this therapy to patients and in strengthening Fondazione Telethon's role as a leading player in the field of advanced therapies."