Around the Helix: Cell and Gene Therapy Company Updates – April 9, 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. Nanoscope Therapeutics’ Gene Therapy MCO-010 Restores Vision in Patients With Retinitis Pigmentosa

Nanoscope Therapeutics’ MCO-010 (sonpiretigene isteparvovec), an investigational ambient-light activatable multi-characteristic opsin (MCO) gene therapy, showed the ability to restore vision in patients with retinitis pigmentosa in clinical data published Molecular Therapy.

2. Alcon Scoops Up Aurion Biotech With Intent of Advancing Ophthalmology Cell Therapy AURN001

A majority interest in Aurion Biotech, clinical-stage company developing ophthalmology cell therapy products, has been acquired by Alcon. The news comes shortly after an announcement from Alcon that it intends to acquire LENSAR, a deal which is expected to close in mid-to-late 2025.

3. Allogene Therapeutics’ ALLO-329 Snags FDA Fast Track Designations for 3 Rheumatology Indications

Allogene Therapeutics’ ALLO-329, an investigational allogeneic chimeric antigen receptor T-cell (CAR-T) therapy, has been granted fast track designation from the FDA for active refractory moderate-to-severe systemic lupus erythematous; active severe/refractory idiopathic inflammatory myopathy, specifically including dermatomyositis, immune mediated necrotizing myopathy, and antisynthetase syndrome; and active refractory diffuse systemic sclerosis.

4. Independent DMC Concludes that Risk-Benefit Ratio for Sarepta’s DMD Gene Therapy Elevidys Remains Favorable

An independent data monitoring committee (DMC) concluded based on a “totality of evidence” that the risk-benefit-ratio for Sarepta Therapeutics’ delandistrogene moxeparvovec-rokl (marketed as Elevidys), a marketed adeno-associated virus (AAV) vector-based gene therapy intended to treat Duchenne muscular dystrophy (DMD), remains favorable for continued dosing in 3 clinical trials without changes to the protocols.

5. Spark Therapeutics Cuts Staff

The company, a gene therapy focused subsidiary of Roche, will be laying off 337 employees in a process that is anticipated to take place from May to December of this year, according to the Philadelphia Inquirer. It was noted that 310 employees remaining at the company will stay in Philadelphia and be integrated into Roche's ongoing activities.

6. Ferring's Bladder Cancer Gene Therapy Adstiladrin Contributes to Record Sales

In 2024, Ferring Pharmaceuticals' nadofaragene firadenovec (marketed as Adstiladrin), an AAV vector-based gene therapy currently approved for the treatment of adult patients with high-risk, Bacillus Calmette-Guerin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors, contributed to record sales for the company, which also sells reproductive medicine products. "In our core area of reproductive medicine, the demand for our products was greater than ever, reflecting worldwide concern about falling birthrates," Jean-Frédéric Paulsen, MS, the chairman of Ferring's board of directors, said in a statement. "At the same time, Adstiladrin, a highly innovative gene therapy, emerged as another main driver of growth for the company."

7. Neurona Rakes in $102 Million for Epilepsy Cell Therapy

Neurona Therapeutics has garnered $102 million from investors in an oversubscribed round of financing. The funding will be utilized to advance the company's pipeline of cell therapy products for neurological indications, including its lead program NRTX-1001, an investigational allogeneic regenerative neural cell therapy intended to treat drug-resistant mesial temporal lobe epilepsy.

8. GeneVentiv and Duke University Join Forces to Tackle Pompe Disease

GeneVentiv and Duke have signed a global licensing agreement for GENV-002, a universal gene editing therapy intended to treat infantile-onset and late-onset Pompe disease, which was developed by experts including Dwight Koeberl, MD, PhD, a professor in the Department of Pediatrics and a medical genetics specialist at Duke University. "...GENV-002 surpasses existing AAV gene therapies with the ability to universally treat infantile- and late-onset Pompe disease in infancy via gene editing," Paris Margaritis, DPhil, the CEO of GeneVentiv, said in a statement.