Atsena Therapeutics is a clinical-stage gene therapy company utilizing adeno-associated virus (AAV) technology to develop treatments for inherited retinal diseases, specifically targeting X-linked retinoschisis and GUCY2D-associated Leber congenital amaurosis. Their ongoing Phase I/II clinical trials aim to reverse or prevent vision loss in patients suffering from these genetic conditions.
Funding
$87.7M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Founders
Product
Problem
Inherited retinal diseases, such as X-linked retinoschisis and Leber congenital amaurosis, lead to progressive vision loss and blindness due to genetic defects. Traditional treatments offer limited efficacy, highlighting the need for targeted therapies that address the underlying genetic causes of these conditions.
Solution
Atsena Therapeutics is developing adeno-associated virus (AAV) gene therapies to treat inherited retinal diseases. Their approach uses AAV vectors to deliver functional genes to retinal cells, aiming to reverse or prevent vision loss in patients with genetic mutations. Atsena's pipeline includes clinical-stage programs for X-linked retinoschisis (XLRS) and GUCY2D-associated Leber congenital amaurosis (LCA1), as well as preclinical programs for Usher Syndrome 1B. The company leverages a tailored AAV technology platform designed to overcome the specific challenges presented by inherited retinal diseases.
Target Audience
The primary target audience includes patients suffering from inherited retinal diseases such as X-linked retinoschisis, Leber congenital amaurosis, and Usher Syndrome, as well as their families and ophthalmologists specializing in genetic eye disorders.
Features
- AAV-based gene therapy platform targeting inherited retinal diseases
- ATSN-101: AAV.SPR capsid for X-linked retinoschisis (XLRS) in Phase I/II clinical trials
- ATSN-201: Gene therapy for GUCY2D-associated Leber congenital amaurosis (LCA1) in Phase I/II clinical trials
- Dual AAV vector-based gene therapy program for MYO7A-associated Usher syndrome (USH1B) in preclinical development
- Novel spreading capsids designed for efficient gene delivery to retinal cells