About us

OcuSphere was founded and is led by patients with inherited retinal diseases, driven by the mission to restore vision for patients' future. We have never lost hope in the face of rare congenital genetic mutations. While no treatment exists today, we are on the verge of developing a breakthrough platform technology that will lead to everyone's therapies.

Bringing Vision Within ReachAffordable Gene Therapies for All.

Problem & Unmet Need

Millions Affected, No Cure

Inherited retinal diseases (IRDs) are a major cause of progressive vision loss, yet most IRDs lack any approved treatment. Patients face inevitable blindness, devastating their independence and quality of life.

High Costs, Limited Access

Where therapies do exist (e.g., a single gene therapy for RPE65 mutations), they often cost over $850K, restricting patient access. Rare disease economics lead to high prices and limited insurance coverage, leaving most IRD patients without viable options.

Complex Genes, Complex Delivery

IRD mutations often involve large genes (like USH2A) unsuitable for current viral vectors. Existing delivery methods face immune response risks, complex manufacturing, and payload size limitations.

Demand for Effective, Affordable Solutions

Patients urgently need breakthrough therapies that can address large genes, offer repeat dosing potential, and be produced cost-effectively. A scalable, non-viral gene therapy platform could finally bring hope to the millions waiting for a cure.

Our Parters

Technology

Synthetic Virion

Our Platform Technology

Modular Design

They can be engineered with separate modules (or "parts") that handle distinct functions such as cargo protection, cell targeting, endosomal escape, and nuclear entry. This modularity allows you to mix and match functionalities to optimize delivery for specific cell types, like photoreceptors.

Safety and Reduced Immunogenicity

As synthetic constructs, they can be designed to be non-replicative and lack pathogenic viral genes, minimizing risks such as insertional mutagenesis and undesirable immune responses. Their surfaces can also be tailored to evade pre-existing neutralizing antibodies.

Customizable Payload Capacity

Unlike many viral vectors limited by their natural genome sizes, synthetic virions can be optimized to encapsulate larger or multiple therapeutic payloads (e.g., full-length genes, gene editing components).

Scalable and Consistent Manufacturing

Being produced by synthetic methods, they offer the potential for more consistent, scalable manufacturing processes with reduced batch-to-batch variability and lower risk of contamination.

AI-Driven Promoter Prediction in Retinal Cells

Utilizes Evo 2, an advanced genomic AI model, to predict promoter regions in retinal cell DNA by analyzing nucleotide scores. Predictions are experimentally validated, and data from approximately 130 retinal cell types are used to train a custom machine learning model for improved precision. The initiative aims to enhance retinal genomics research through integrated AI analysis and empirical confirmation.