Ray Therapeutics, a biotechnology company developing optogenetic gene therapies for patients with retinal degenerative conditions, and Forge Biologics, a gene therapy-focused contract development and manufacturing organization, announced a manufacturing partnership that will advance Ray Therapeutics’ lead optogenetics gene therapy program, Ray-001, into clinical trials for patients with retinitis pigmentosa.
Forge will provide adeno-associated viral (AAV) process development, scale-up engineering and 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 200,000 ft2 gene therapy cGMP production facility in Columbus, Ohio.
“We have set up Forge to provide end-to-end gene therapy manufacturing services to enable support for clients striving to make significant improvements in patients’ lives,” said Timothy J. Miller, Ph.D., CEO, President, and Co-Founder of Forge. “We look forward to helping Ray Therapeutics advance their AAV manufacturing with an eye towards restoring vision in patients with ophthalmology disorders.”
Ray-001 is intended for use in treating patients with retinitis pigmentosa (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.
“Ray-001 is a ground-breaking new approach to treat inherited retinal diseases using the power of optogenetics, and working together with Forge, we are one step closer to restoring vision in patients who are going blind,” said Paul Bresge, CEO of Ray Therapeutics.
About Retinitis Pigmentosa
Retinitis pigmentosa (RP), is a heterogeneous group of genetic diseases that cause retinal degeneration leading to near or complete blindness for most patients. The severe loss of photoreceptor cells that occurs in this genetic degenerative disease leads to partial or complete blindness. At present, no effective treatment is available to restore vision once the photoreceptor cells have been lost. Over 100 genetic mutations are known to cause RP and all types of inheritance patterns are recognized. Patients are typically diagnosed in their late teens, with symptoms including night blindness, reduced visual fields and eventual loss of visual acuity. As the disease progresses, retinal atrophy, and permanent loss of the light sensitive photoreceptors occur. The prevalence of RP is approximately 100,000 persons affected in the US.
About Ray Therapeutics
Ray Therapeutics is developing novel optogenetics gene therapies for patients with blinding diseases. The company is developing its lead candidate Ray-001 in retinitis pigmentosa, a degenerative retinal disease with significant unmet medical need. The company’s mission is to use optogenetics to restore vision, independent of genetic mutation for patients with inherited retinal diseases.
About Forge Biologics
Forge Biologics is a hybrid gene therapy contract manufacturing and clinical-stage therapeutics development company. Forge’s mission is to enable access to life changing gene therapies and help bring them from idea to reality. Forge has a 200,000+ square foot facility in Columbus, Ohio, The Hearth, to serve as its headquarters. The Hearth is a custom-designed cGMP facility dedicated to AAV vector manufacturing and will host end-to-end manufacturing services to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing. By taking a patients-first approach, Forge aims to accelerate the timelines of these transformative medicines for those who need them the most.
