Around the Helix: Cell and Gene Therapy Company Updates – September 3, 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. Dual-Vector AAV Gene Therapy for GM2 Gangliosidosis Shows Promising Early Outcomes

Primary data from a phase 1/2 clinical trial of an investigational dual-vector gene therapy for GM2 gangliosidosis, published in Nature Medicine,¹ suggest that the approach resulted in measurable enzymatic activity, substrate reduction, and encouraging neurologic outcomes in patients with infantile Tay–Sachs disease (TSD) and Sandhoff disease (SD).

2. CD19 CAR EngTregs: The Next Step in Autoimmune Disease Cell Therapy?

Annaiz Grimm, BS, a research scientist at Seattle Children's Research Institute, discussed the potential use of engineered regulatory T-cells (EngTregs) to treat autoimmune disease.

3. John Murphy, PhD, and Tia DiTommaso, PhD, on Preclinical Evidence for PH1 Gene Therapy

Tia DiTommaso, PhD, the senior director of preclinical pharmacology Arbor Biotechnologies, and John Murphy, PhD, the chief scientific officer of Arbor Biotechnologies, spoke about future plans for ABO-101, a lipid nanoparticle (LNP)-delivered Cas12i2-based gene editing therapy intended to treat primary hyperoxaluria type 1 (PH1). Notably, Arbor announced the dosing of the first patient in redePHine in July 2025.

4. VeonGen Snags FDA RMAT Designation for Stargardt Gene Therapy

VeonGen Therapeutics has received regenerative medicine advanced therapy (RMAT) designation from the FDA for VG801, an investigational dual adeno-associated virus (AAV) vector-based gene therapy intended to treat Stargardt disease and other retinal dystrophies associated with ABCA4 mutations. Notably, VG801 previously received FDA rare pediatric disease and orphan drug designations and the agency selected VeonGen to help develop a new functional end point for Stargardt via its Rare Disease Endpoint Advancement (RDEA) pilot program.

5. Vertex Teams Up With Enlaza to Improve Conditioning

Vertex Pharmaceuticals and Enlaza Therapeutics have entered a new collaboration that has among its aims the improvement of conditioning for Vertex Pharmaceuticals and CRISPR Therapeutics’ exagamglogene autotemcel (exa-cel; marketed as Casgevy), a CRISPR/Cas9 gene-editing cell therapy marketed for the treatment of sickle cell disease and transfusion-dependent β-thalassemia, via the use of Enlaza's War-Lock technology platform. “Vertex is committed to developing transformative therapies for serious diseases that fit within our unique R&D strategy including certain autoimmune diseases and gentler conditioning for Casgevy...” Mark Bunnage, D.Phil., the senior vice president of global research at Vertex, said in a statement. “We look forward to exploring the potential of Enlaza’s War-Lock platform to help us achieve these goals.”

6. Nanoscope Nabs RMAT Designation for Retinitis Pigmentosa Gene Therapy

Nanoscope Therapeutics has received FDA RMAT designation for MCO-010 (sonpiretigene isteparvovec), an investigational ambient-light activatable multi-characteristic opsin (MCO) gene therapy intended to treat various retinal diseases. The RMAT designation specifically applies to Stargardt disease. Notably, the European Medicines Agency has also granted MCO-010 orphan drug designation for 5 retinal dystrophy categories, including macular dystrophies and nonsyndromic and syndromic rod-dominant and cone-dominant dystrophies.