Ray Therapeutics Licenses Delivery Technology for Optogenetics Therapy

6 min read

Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases, announces an agreement granting Ray Therapeutics a non-exclusive, royalty bearing license of Adverum’s proprietary AAV.7m8 intravitreal (IVT) vector capsid to be used in conjunction with Ray’s optogenetics payload.

“This deal highlights the value of Adverum’s AAV.7m8 gene therapy platform, a capsid for IVT delivery, in a variety of ocular gene therapy indications,” commented Laurent Fischer, M.D., president and chief executive officer of Adverum Biotechnologies. “A single IVT injection of Ixo-vec in the Phase 1 OPTIC study for wet age-related macular degeneration has been well tolerated and has led to stable and persistent aflibercept protein levels through at least three years. Ixo-vec is currently being evaluated in the ongoing Phase 2 LUNA trial. This marks the sixth asset delivered via AAV.7m8, including our Ixo-vec candidate for the treatment of wet age-related macular degeneration, Gensight’s GS030, an optogenetic asset for retinitis pigmentosa, both of which have shown promising results in early clinical trials, as well as Adverum’s own investigational pre-clinical pipeline assets in dry AMD, optogenetics and blue cone monochromacy.”

“Visual optogenetics is a disruptive technology which affords the opportunity to restore life-transforming levels of vision to blind and visually impaired patients with conditions such as retinitis pigmentosa,” said Paul Bresge, chief executive officer of Ray Therapeutics. “We have developed a next generation bioengineered optogenetic payload which we believe is optimized for human vision. When delivered using previously studied low doses of AAV.7m8, we anticipate levels of target retinal ganglion cell transduction that could realize the full potential of our RTx-015 asset.”

Under the terms of the agreement, Adverum grants Ray Therapeutics a worldwide, non-exclusive license of AAV.7m8 for the prevention, treatment, diagnosis or amelioration of any ocular disorder utilizing Ray’s optogenetics approach.

About Adverum Biotechnologies

Adverum Biotechnologies (NASDAQ: ADVM) is a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases with the aspiration of developing functional cures to restore vision and prevent blindness. Leveraging the capabilities of its proprietary, intravitreal (IVT) platform, Adverum is developing durable, single-administration therapies, designed to be delivered in physicians’ offices, to eliminate the need for frequent ocular injections to treat these diseases. Adverum is evaluating its novel gene therapy candidate, ixoberogene soroparvovec (Ixo-vec, formerly referred to as ADVM-022), as a one-time, IVT injection for patients with neovascular or wet age-related macular degeneration. Additionally, by overcoming the challenges associated with current treatment paradigms for debilitating ocular diseases, Adverum aspires to transform the standard of care, preserve vision, and create a profound societal impact around the globe. For more information, please visit www.adverum.com.

About Ray Therapeutics

Ray Therapeutics is developing novel optogenetics gene therapies for patients with blinding diseases. The company is developing its lead candidate RTx-015 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. Ray Therapeutics is based in San Francisco, California. For additional information, visit www.raytherapeutics.com

Forward-looking Statements

Statements contained in this press release regarding events or results that may occur in the future are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include but are not limited to statements regarding the potential benefits of AAV.7m8 as a potent capsid. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, including risks inherent to, without limitation: Adverum’s novel technology, which makes it difficult to predict the outcome of clinical trials; regulatory uncertainties; the results of early clinical trials not always being predictive of future clinical trials and results; and the potential for future complications or side effects in connection with use of AAV.7m8. Additional risks and uncertainties facing Adverum are set forth under the caption “Risk Factors” and elsewhere in Adverum’s Securities and Exchange Commission (SEC) filings and reports, including Adverum’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2023 filed with the SEC on May 11, 2023. All forward-looking statements contained in this press release speak only as of the date on which they were made. Adverum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.