Aevum Zenth Health Sciences Receives FDA Approval for Aetherix™, a First-in-Class Gene Therapy for Inherited Retinal Degeneration

Neo Geneva, Switzerland — October 24, 2025

Aevum Zenth Health Sciences, a global biopharmaceutical division of Aevum Zenth Conglomerate, today announced that the U.S. Food and Drug Administration (FDA) has granted approval for Aetherix™ (aexadecagene neparvovec), a novel adeno-associated viral (AAV) gene therapy for the treatment of inherited retinal degeneration (IRD) in adults with specific mutations in the RPE65 gene.

Aetherix™ represents a landmark achievement in molecular ophthalmology, offering a potentially transformative, one-time intravitreal injection that restores functional vision in patients with progressive retinal photoreceptor degeneration. The approval is based on robust Phase II/III clinical data demonstrating sustained visual acuity improvement and functional vision gains over a 36-month follow-up period.

"Aetherix™ is more than a therapy; it is a new standard of care for patients who have faced irreversible vision loss for generations. Today’s FDA approval validates years of rigorous scientific innovation, clinical execution, and unwavering commitment to patient outcomes by our Zenth Health Sciences team."

— Dr. Elena Vasquez, Chief Scientific Officer, Aevum Zenth Health Sciences

Clinical Trial Results

In the pivotal AETHER-1 trial (NCT05884102), 48 patients with biallelic RPE65 mutations received a single intravitreal injection of Aetherix™. At 24 months, 83% of treated participants demonstrated a ≥15-letter improvement on the Early Treatment Diabetic Retinopathy Study (ETDRS) letter chart compared to baseline. Additionally, 91% showed significant gains in visual function testing, including mobility, contrast sensitivity, and light perception. The safety profile was favorable, with most adverse events classified as mild-to-moderate ocular inflammation managed with standard topical corticosteroids.

"Our mission at Aevum Zenth is to tackle humanity’s most complex medical challenges with precision and scale. The FDA’s approval of Aetherix™ underscores our division’s leadership in gene therapy and reinforces our broader strategy of advancing curative medicine across the genome."

— Marcus Thorne, President, Aevum Zenth Health Sciences

Commercialization & Global Access

Aetherix™ will be commercially launched in the United States, European Union, and United Kingdom during Q1 2026. Manufacturing will be supported by Zenth’s dedicated viral vector production facilities in Basel and Boston, ensuring reliable supply chain integrity and strict compliance with cGMP standards. Patient access programs, including compassionate use and global health initiatives, will be coordinated through Aevum Zenth’s Medical Affairs and Market Access divisions.

The company also announced ongoing Phase II studies evaluating Aetherix™ for USH2A and CEP290-associated retinal dystrophies, with data expected in 2027. Aevum Zenth Health Sciences continues to expand its gene therapy pipeline across neurology, oncology, and rare metabolic diseases.