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Vision Care Gene Therapy Acquired

Geographic atrophy affects 5M people globally.

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RARITAN, NJ – Janssen Pharmaceuticals Inc. has acquired rights to an investigational gene therapy from Hemera Biosciences LLC.

The therapy, called HMR59, is administered as a one-time, outpatient intravitreal injection to help preserve vision in patients with geographic atrophy, a late-stage and severe form of age-related macular degeneration.

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Financial terms of the deal were not disclosed.

Patients with AMD often have low levels of CD59, a protein that protects the retina from damage caused by an essential part of the body’s natural immune response called “complement,” according to a Janssen press release. In geographic atrophy, an overactivity of complement destroys cells in the macula, the central part of the retina responsible for central vision and seeing fine details, and results in a relentless progression to blindness. HMR59 is designed to increase the ability of retina cells to make a soluble form of CD59, helping to prevent further damage to the retina and preserve vision.

Geographic atrophy affects 5 million people globally, and is a leading cause of blindness in people over 50 years of age. The prevalence of geographic atrophy increases as the global population ages with roughly one in 29 people over age 75 affected, and nearly one in four people over age 90. There are currently no available therapies other than vitamins and low vision aids, Janssen notes.

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“Geographic atrophy is a devastating form of AMD that impacts the ability to accomplish everyday tasks, such as reading, driving, cooking, or even seeing faces,” said James F. List, MD, PhD, global therapeutic area head, cardiovascular and metabolism, Janssen Research & Development LLC. “Our aim with this novel, single-administration gene therapy is to use our development expertise and deep heritage in vision care to help improve patient outcomes by intervening early, halting the progression to blindness, and preserving more years of sight.”

The Phase 1 study of HMR59 for patients with geographic atrophy is complete. A second Phase 1 study exploring HMR59 in patients with wet-AMD is currently conducting follow-up visits to evaluate long-term safety.

Janssen Pharmaceuticals is one of the Janssen Pharmaceutical Cos. of Johnson & Johnson.

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