Around the Helix: Cell and Gene Therapy Company Updates – September 24, 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. Phase 1/2 Trial for Immix Biopharma’s AL Amyloidosis CAR T NXC-201 Hits Over 50% Enrollment

The phase 1b/2 NEXICART-2 clinical trial (NCT06097832), which is evaluating Immix Biopharma and its subsidiary Nexcella's NXC-201, an investigational autologous BCMA-directed chimeric antigen receptor (CAR) T-cell therapy, for the treatment of relapsed/refractory (R/R) amyloid light chain (AL) amyloidosis, has reached over 50% of its planned enrollment.

2. Atsena Therapeutics’ Trial for X-Linked Retinoschisis Gene Therapy ATSN-201 Finishes Dosing Adult Patients in Part B

Atsena Therapeutics has finished dosing adult patients in Part B of its phase 1/2/3 LIGHTHOUSE clinical trial (NCT05878860) evaluating ATSN-201, an investigational adeno-associated virus (AAV) gene therapy, for the treatment of X-linked retinoschisis (XLRS) associated with mutations in the RS1 gene.

3. Capricor Therapeutics Testing DMD Cell Therapy Deramiocel’s Mettle in Phase 3 HOPE-3 Clinical Trial

Capricor Therapeutics is currently evaluating deramiocel (also known as CAP-1002), an investigational allogeneic cardiosphere-derived cell therapy intended for the treatment of Duchenne muscular dystrophy (DMD) cardiomyopathy, in the phase 3 HOPE-3 clinical trial (NCT05126758). In honor of Duchenne Action Month, observed annually in September by the patient and clinician communities, and following up on World Duchenne Day, held every year on September 7, CGTLive® took a closer look at this ongoing study.

4. Roger Hajjar, MD, on the Commercial Viability of Gene Therapy for Ultra Rare Diseases

Roger Hajjar, MD, the director of the Mass General Brigham’s Gene and Cell Therapy Institute, spoke about the trend of doctors and scientists working on models to make gene therapy products financially viable.

5. Sanofi's DM1 Gene Therapy SAR446268 Snags Fast Track Designation

The FDA has granted fast track designation to Sanofi’s SAR446268, an investigational AAV vector-based gene therapy intended to treat noncongenital (juvenile and adult onset) DM1 myotonic dystrophy type 1 (DM1). The therapy, which is currently being evaluated in a phase 1/2 clinical trial (NCT06844214), is given as a one-time treatment and is intended to silence expression of the DMPK gene through RNA interference.

6. Phase 1/2 Trial for uniQure's Huntington Disease Gene Therapy AMT-130 Hits Primary End Point

Findings from a phase 1/2 clinical trial evaluating uniQure's AMT-130, an AAV vector-based gene therapy intended to treat Huntington disease, have shown that at 36 months posttreatment, high-dose AMT-130 was able to significantly slow disease progression by 75% in comparison to a propensity score-matched external control, as assessed by the Unified Huntington’s Disease Rating Scale (cUHDRS). As such, the study has met its prespecified primary end point. "“I believe these groundbreaking data are the most convincing in the field to date and underscore potential disease-modifying effects in Huntington’s disease, where an urgent need persists," Sarah Tabrizi, MD, FRCP, FRS, FMedSci, PhD, a professor of clinical neurology, director of the University College London Huntington’s Disease Center, and joint head of the department of neurodegenerative disease, said in a statement.