Ocugen Inc.’s OCU-410ST (AAV5-hRORA) has been awarded orphan drug designation by the FDA for the treatment of ABCA4-associated retinopathies, including Stargardt disease, retinitis pigmentosa 19 (RP19) and cone-rod dystrophy 3 (CORD3) diseases.
Mast cell-expressed membrane protein 1 (MCEMP1) is a type II transmembrane protein predominantly expressed in myeloid lineage immune cells, including lung-resident mast cells and alveolar macrophages. MCEMP1 is a critical factor in allergic and inflammatory lung diseases, with higher expression levels correlating with increased disease severity. However, the molecular mechanisms underlying the role of MCEMP1 in the pathogenesis of lung inflammatory diseases remain unclear.
Researchers from Athira Pharma Inc. presented preclinical efficacy data for ATH-1105, a positive modulator of hepatocyte growth factor (HGF)/MET, being evaluate for the treatment of amyotrophic lateral sclerosis (ALS).
Cyclin-dependent kinase 4 and 6 (CDK4 and CDK6) are validated targets for the treatment of ER+/HER2- breast cancer, but resistance to therapy is still a problem in these patients, with >20% of them developing intrinsic resistance and up to 70% developing acquired resistance within 3 years.
In research presented this week at the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting, investigators from Life Biosciences Inc. reported using epigenetic reprogramming in a nonarteritic anterior ischemic optic neuropathy (NAION) model in nonhuman primates (NHPs).
A base-by-base comparison of the genome sequences of 240 species of mammals has pinpointed sites in the human genome where mutations are likely to cause disease. The sites are all perfectly conserved across the mammalian family tree over 100 million years of evolution, indicating they underlie fundamental biological processes that do not tolerate diversity or change very well.
A pill that delivers electrical stimulation to the vagus nerve from inside the stomach was able to trigger the release of appetite-controlling neurohormones, specifically the “hunger hormone” ghrelin. The work, which was described in the April 26, 2023, issue of Science Robotics, could pave the way for treating “metabolic, [gastrointestinal], and neuropsychiatric disorders noninvasively with minimal off-target effects,” the authors wrote in their paper.
Leucine-rich repeat kinase 2 (LRRK2; Dardarin) inhibitors have been detailed in a recent Merck Sharp & Dohme Corp. patent and reported to be useful for the treatment of Parkinson’s disease, among others.