• The Usher Syndrome market is poised for growth with emerging therapies addressing the root cause of the disease, offering alternatives to supportive care.
• Ultevursen, an investigational RNA therapy, targets the USH2A gene mutation and is currently in Phase II/III trials, showing promise in restoring functional usherin protein.
• Nacuity Pharmaceuticals' NPI-001, a free radical scavenger, is in Phase I/II trials, aiming to reduce oxidative stress and preserve photoreceptor cells in Usher Syndrome patients.
• The US has the highest prevalent cases of Usher Syndrome, with approximately 21,000 cases in 2023, highlighting the significant unmet need for effective treatments.