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Ush Biologics
Alachua, Florida, United States
- Ownership
- Private
- Development stage
- Other
01
Overview
USH Biologics is a preclinical-stage biotechnology company focused on developing transformative gene therapies for Usher syndrome, a rare genetic disorder characterized by combined hearing and vision loss. The company utilizes a proprietary dual-AAV (Adeno-Associated Virus) vector platform designed to deliver large therapeutic genes that exceed the standard packaging capacity of a single AAV vector. Their lead programs target Usher Syndrome Type 1B, caused by mutations in the MYO7A gene, and Usher Syndrome Type 2A. By focusing on innovative delivery mechanisms and overcoming genetic cargo limitations, USH Biologics aims to address the significant unmet medical need in the inherited retinal disease space.
- Therapeutic areas
- NeurologyOphthalmologyRare Diseases
- Modalities
- Gene therapyAAV-based therapy
- Industry
- Biotech
02
Partnerships
University of FloridaAAVantgarde Bio
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