Research Update on Treatment for Aged Macular Degeneration

Janssen Pharmaceutical, a Johnson & Johnson company, has bought the rights to Hemera Biosciences’ gene therapy HMR59. This therapy consists of a one-time, outpatient, intravitreal (into the eye) injection to preserve eyesight in patients with geographic atrophy (Dry MD). Geographic atrophy is a late-stage, severe type of age-related macular degeneration (AMD).

“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, Global Therapeutic Area Head, Cardiovascular & Metabolism, Janssen Research & Development. “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.”

AMD patients often have low levels of CD59, a protein that protects the retina from damage caused by the body’s natural complement immune response. In geographic atrophy, complement overreacts and destroys cells in the macula, the central part of the retina that handles central vision and fine details. This causes a “relentless progression to blindness.”

The gene therapy, HMR59, increases the ability of retina cells to manufacture a soluble form of CD59. This helps prevent more damage to the retina and preserves eyesight.

Geographic atrophy affects about five million people around the world. It is the leading cause of blindness in people over 50 years of age.

Eye diseases are something of low-hanging fruit when it comes to gene therapies, because they can be directly administered to the eye, not systemically. Systemic gene therapies typically require being processed by the liver, which becomes a much more complicated mechanism of action and delivery.

Read the full article* here

Further from MDNZ

This possible treatment is in the early days of research and if it becomes available in New Zealand it is likely to be at least 18 months to 2 years (it will of course also need to go through the official approval process).

HMR59 is currently under FDA approved clinical testing for use in both dry and wet macular degeneration.

More about this here

When we hear further updates on the trials, we will update you.

*Source: Biospace.com