Opus Genetics is developing gene therapies targeting specific mutations in genes responsible for inherited retinal diseases, such as bestrophinopathy and retinitis pigmentosa. The company aims to restore vision for patients suffering from these genetic conditions by advancing its gene therapy pipeline.
Funding
$1.7M 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 (IRDs) are caused by mutations in over 280 different genes, leading to progressive vision loss and affecting over 2 million people worldwide. Many of these genetic conditions lack effective treatments, leaving a significant unmet need for targeted therapies.
Solution
Opus Genetics is a clinical-stage biopharmaceutical company focused on developing gene therapies for inherited retinal diseases. The company's pipeline addresses mutations in genes that cause various forms of IRDs, including bestrophinopathy, Leber congenital amaurosis (LCA), and retinitis pigmentosa (RP). Opus Genetics leverages validated science and expertise in retinal gene therapy to advance novel treatments towards clinical application, with the goal of preserving and restoring vision for patients with these conditions. Their approach includes a tailored manufacturing process designed to support a sustainable pipeline of gene therapies for a wide range of retinal diseases.
Target Audience
The primary target audience includes patients suffering from inherited retinal diseases, as well as ophthalmologists and retinal specialists seeking effective gene therapy treatments for these conditions.
Features
- Focus on adeno-associated virus (AAV)-based gene therapies for inherited retinal diseases.
- Programs targeting specific mutations in genes responsible for bestrophinopathy, Leber congenital amaurosis (LCA), and retinitis pigmentosa (RP).
- Utilizes well-characterized material and safe retinal transduction methods.
- Leverages insights from researchers with extensive experience in inherited retinal diseases.
- Aims to produce at least one Investigational New Drug (IND) application per year.
- Employs high-quality, pharmaceutical-grade manufacturing tailored for rare disease populations.