Ray Therapeutics, Forge Biologics join forces on gene therapy

By The Science Advisory Board staff writers

March 2, 2022 -- Ray Therapeutics and Forge Biologics have partnered for manufacturing that will advance Ray Therapeutics’ lead optogenetics gene therapy program, Ray-001, into clinical trials for patients with retinitis pigmentosa (RP).

Under the agreement, Forge will provide adeno-associated viral (AAV) process development, scale-up engineering, and current good manufacturing practice (cGMP) manufacturing services for Ray Therapeutics’ program, Ray-001. The program will utilize Forge’s platform process including its proprietary HEK 293 suspension Ingition Cells and pEMBR adenovirus helper plasmid. All development and cGMP manufacturing activities will occur at The Hearth, Forge’s gene therapy cGMP production facility in Columbus, Ohio.

Ray-001 is intended for use in treating patients with RP. Optogenetics is a promising approach that has the potential to restore useful vision to visually-impaired and blind individuals. Patients with RP have damaged photoreceptors, the primary cells required for vision, which are lost and cannot regenerate. However, inner retinal neurons downstream to photoreceptors, especially retinal ganglion cells (RGCs), persist in significant numbers through late-stage disease. Ray Therapeutics’ lead candidate Ray-001 uses intravitreal administration, from which the vector diffuses into the retina and transduces primarily the RGCs.

Financial details of the partnership were not disclosed.

Ray Therapeutics is developing novel optogenetics gene therapies for patients with blinding diseases. Forge Biologics is a hybrid gene therapy contract manufacturing and clinical-stage therapeutics development company.


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