Gensight Biologics develops adeno‑associated virus (AAV) gene‑therapy vectors that deliver therapeutic genes to photoreceptors and retinal pigment epithelium via a single subretinal or intravitreal injection. The engineered capsids provide long‑term transgene expression and are manufactured in a GMP‑grade, scalable pipeline, enabling retinal specialists to treat inherited and progressive retinal diseases with a disease‑modifying approach.
Funding
$4.3M 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
Patients with chronic, progressive retinal diseases such as retinitis pigmentosa and age‑related macular degeneration have few or no disease‑modifying treatments, resulting in irreversible vision loss and limited quality of life. Existing pharmacologic options address symptoms rather than the underlying genetic or molecular causes, and delivery to the retina remains technically challenging.
Solution
Gensight Biologics applies a proprietary adeno‑associated virus (AAV) gene‑therapy platform to introduce therapeutic genes directly into retinal cells, enabling sustained production of disease‑modifying proteins. The approach is designed for a single subretinal or intravitreal administration, reducing the need for repeated invasive procedures. By targeting the root genetic or molecular drivers of degeneration, the therapy aims to halt or reverse photoreceptor loss and preserve visual function over the long term. The platform leverages optimized capsid engineering for efficient retinal transduction and incorporates regulatory‑compliant manufacturing processes to support scalable clinical development. Preclinical studies demonstrate durable expression, safety, and functional rescue in relevant animal models, positioning the pipeline for accelerated progression through clinical trials.
Target Audience
Primary customers are retinal specialists and ophthalmology clinics treating inherited retinal dystrophies and other progressive retinal disorders, as well as pharmaceutical partners seeking gene‑therapy candidates for co‑development or licensing.
Features
- Engineered AAV capsids with enhanced tropism for photoreceptors and retinal pigment epithelium
- Single‑dose subretinal or intravitreal delivery enabling long‑term transgene expression (≥12 months)
- Modular gene cassette design allowing rapid adaptation to multiple retinal disease targets
- GMP‑grade vector production pipeline with scalable bioreactor manufacturing and viral purification
- Integrated safety switches (e.g., inducible promoters) to modulate transgene activity if needed
- Comprehensive preclinical validation package including functional vision assays and histopathology
- Compatibility with existing ophthalmic surgical workflows and imaging systems for precise dosing