Ray and Forge expand partnership to include plasmid DNA manufacturing
SAN FRANCISCO & COLUMBUS, Ohio, Oct 10: 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 further collaboration for their manufacturing partnership to include clinical stage plasmid DNA production to support Ray Therapeutics’ lead optogenetics gene therapy program, RTx-015, in clinical trials for patients with retinitis pigmentosa.
Forge will provide research-grade and GMP-Pathway plasmid manufacturing services, in addition to adenoassociated viral vector (AAV) process development, scale-up engineering, and cGMP manufacturing services for Ray Therapeutics’ program, RTx-015. The program will continue to utilize Forge’s platform manufacturing processes, including its proprietary HEK 293 suspension Ignition Cells™ and pEMBR™ adenovirus helper plasmid. All development and cGMP manufacturing activities will occur at the Hearth, Forge’s 200,000 square foot gene therapy cGMP production facility in Columbus, Ohio.
Streamline
“With the recent launch of research and clinical stage plasmid DNA manufacturing, we can better streamline the production of our clients’ AAV programs by vertically integrating plasmids into our manufacturing process,” said Timothy J. Miller, Ph.D., CEO, President, and Co-Founder of Forge. “Our partnership with Ray Therapeutics showcases how these new offerings can accelerate AAV gene therapies from idea into reality for patients in need, and we are excited to help Ray Therapeutics meet the needs of patients with retinitis pigmentosa.”
RTx-015 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 latestage disease. Ray Therapeutics’ lead candidate RTx-015 uses intravitreal administration, from which the vector diffuses into the retina and transduces primarily the RGCs.
“By adding clinical grade plasmid production to their existing suite of AAV manufacturing capabilities, Forge is easing the scope of production and accelerating the development of our lead therapeutic,” said Paul Bresge, CEO of Ray Therapeutics. “Offering everything we need under one roof integrates our entire process so that we can focus on our mission to restore vision in patients losing their sight as fast as possible.”
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.