[ad_1]
HMR59 targets geographic atrophy: a severe form of advanced AMD. In geographic atrophy, complement overactivity destroys cells in the macula, the central part of the retina responsible for central vision and fine detail vision, progressing to blindness.
Patients with AMD often have low levels of the CD59 protein, which protects the retina from damage caused by an essential part of the body’s natural immune response called complement.
HMR59 – given as a one-time, outpatient intravitreal injection – is designed to increase the ability of the retinal cells to produce CD59: thus helping to preserve vision.
Therapy in Phase 1 studies
Hemera Biosciences, based in Massachusetts, founded in 2010 to focus on the use of gene therapy for patients with AMD, has studied HMR59 for both dry and wet forms of AMD.
He completed a Phase 1 study for HMR59 for patients with geographic atrophy. A second Phase 1 study exploring HMR59 in wet AMD patients is currently conducting follow-up visits to assess long-term safety.
Dr. Adam Rogers, chief executive officer and founder of Hemera, said: Dry AMD is a debilitating visual disease that affects millions of people with no currently available treatment options. We are very excited that Janssen recognizes the value of our HMR59 program.
“Janssen is a recognized leader in the pharmaceutical industry, with extensive manufacturing, clinical, regulatory and commercial experience. As part of Janssen’s organization, HMR59 will be best placed to reach its maximum potential and benefit patients in need. “
Geographical atrophy is estimated to affect five million people globally; and it is a leading cause of blindness in people over the age of 50. The prevalence of the disease increases with the aging of the world population: about one in four people over 90 suffer from it.
It affects the ability to perform daily activities such as reading, driving or cooking.
Focus on gene therapy for Janssen
Janssen of Johnson & Johnson is developing expertise in the manufacture, development and commercialization of gene therapies through a range of mechanisms of action for the eye. This experience will then be used to address other parts of the body.
He established his eye disease portfolio in 2018 and is working on both rare and common eye diseases: including achromatopsia and X-linked retinitis pigmentosa, age-related macular degeneration, diabetic retinopathy, and diabetic macular edema.
According to Mathai Mammen, MD, Ph.D., global head of research and development at Janssen, gene therapy for advanced eye disease is “largely unexplored.” Its acquisition of the HMR59 rights is part of its strategy to “pursue the best science to discover transformational treatments”.
“With this acquisition, we are leading the way to bring innovative solutions to patients who are losing their sight”, Mammen said.
In 2019, Janssen announced a worldwide collaboration and licensing agreement with MeiraGTx Holdings, a clinical stage gene therapy company, to develop, manufacture and commercialize its portfolio of hereditary clinical stage retinal diseases, including key candidate products for achromatopsia (ACHM) caused by mutations in CNGB3 or CNGA3 and X-linked retinitis pigmentosa (XLRP).
Last month it released 12-month data for phase 1/2 of the ongoing XLRP pathway, showing that low and intermediate doses of the experimental gene therapy were well tolerated and continued to demonstrate sustained or increased vision improvement in statistically significant way across multiple metrics and modalities.
The companies also formed a research collaboration to explore new targets for other inherited diseases of the retina and further develop adeno-associated virus (AAV) manufacturing technology.
.
[ad_2]
Source link
