Around the Helix: Cell and Gene Therapy Company Updates – March 26, 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. FDA Approves Alnylam’s Vutrisiran for the Treatment of ATTR Amyloidosis With Cardiomyopathy

The FDA has approved Alnylam Pharmaceuticals' subcutaneous RNA interface therapy vutrisiran, marketed as Amvuttra, for the treatment of cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM). The therapy is approved for adult patients.

2. REGENXBIO’s DMD Gene Therapy RGX-202 Generates Microdystrophin Expression in Additional Patients Treated in Phase 1/2 Trial

REGENXBIO’s RGX-202, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat Duchenne muscular dystrophy (DMD), has continued to demonstrate the ability to produce microdystrophin at “consistent, robust” levels in data from 2 new patients treated in the phase 1/2 portion of the AFFINITY DUCHENNE clinical trial (NCT05693142).

3. Secretome Therapeutics Doses First Patient in Phase 1 Trial for Heart Failure Cell Therapy STM-01

Secretome Therapeutics has dosed the first patient in a phase 1 clinical trial (NCT06560762) evaluating STM-01, an investigational allogeneic neonatal cardiac progenitor cell (nCPC)-derived cell therapy, for the treatment of heart failure with preserved ejection fraction.

4. Genetic and Gender Factors Linked to Fuchs Endothelial Corneal Dystrophy in University College London Study

Genetic and demographic factors, including gender, contribute to Fuchs Endothelial Corneal Dystrophy (FECD), according to research led by the University College London (UCL) Institute of Ophthalmology and supported by National Institute for Health and Care Research (NIHR) Moorfields Biomedical Research Centre. The large-scale study was published in JAMA Ophthalmology.

5. AccurEdit's Amyloidosis Gene Editing Therapy Snags Orphan Drug Designation

AccurEdit has received orphan drug designation from the FDA for ART001, a lipid nanoparticle (LNP)-based in vivo gene editing therapy intended to treat transthyretin amyloidosis (ATTR). Notably, IND applications for ART001 have been cleared in both the United States and China; according to AccurEdit, this is the first LNP-based in vivo gene editing therapy to have attained that milestone.

6. Ophthalmology Gene Therapy Focused Company Hubble Therapeutics Rakes in $7.3 Million in Series A

The funding secured from the Series A round of financing will be used by the company to help bring HUB-101, a gene therapy product intended to treat Leber congenital amaurosis (LCA16) and snowflake vitreoretinal degeneration (SVD), into clinical trials. "[W]ith our new financing and the establishment of key partnerships, we hope to scale up HUB-101 manufacturing and conduct additional investigational new drug (IND)-enabling studies to enter the clinic within the next 2 years to realize our steadfast, unwavering promise to support families throughout the world impacted by this devastating genetic disease," Jeff Sabados, the founder and president of Hubble Therapeutics, said in a statement.

7. Benitec Biopharma Expects to Bring $30 Million in With Common Stock Offering

Benitec announced that 1,443,000 shares of its common stock will be priced at $13.00 per share, and that along with prefunded warrants to purchase common stock in lieu thereof and a concurrent registered direct offering of 900,000 shares with Suvretta Capital a total of approximately $30 million in gross proceeds are expected to find their way to the company. The funding will help support development of the company's therapeutics, such as BB-301, an investigational AAV vector-based silence and replace gene therapy for the treatment of oculopharyngeal muscular dystrophy-related dysphagia