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BioWorld - Tuesday, May 19, 2026
Breaking News: Bundibugyo is harsh reminder of need for broad vaccine strategiesBreaking News: Bundibugyo is harsh reminder of need for broad vaccine strategiesBreaking News: Bundibugyo is harsh reminder of need for broad vaccine strategies
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Ocular

Ocugen’s OCU-410ST awarded US orphan drug designation for ABCA4-associated retinopathies

April 28, 2023
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.
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Lungs
Respiratory

MCEMP1, a KIT receptor adaptor promoting mast cell proliferation in chronic airway inflammation

April 28, 2023
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.
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Illustration demonstrating muscle contraction in amyotrophic lateral sclerosis.
Neurology/Psychiatric

ATH-1105 protects against disease progression and delays time to mortality in mouse model of ALS

April 28, 2023
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).
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Breast cancer tumor and its microenvironment obtained from a live mouse model.
Cancer

CDK4/6 bifunctional inhibitors show promise for breast cancer treatment

April 28, 2023
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.
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Ocular

OSK-driven epigenetic reprogramming restores visual function in nonhuman primates

April 28, 2023
By Xavier Bofill Bruna
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).
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Illustration of animals, DNA double helix
Genetic/Congenital

In Zoonomia project, evolutionary lens hones search for functional genomic variants

April 28, 2023
By Nuala Moran
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.
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Illustration of the vagus nerve with lungs, heart, stomach and digestive tract
Gastrointestinal

Electric pill can stimulate appetite-regulating hormones

April 27, 2023
By Anette Breindl
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.
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Other news to note for April 27, 2023

April 27, 2023
Additional early-stage research and drug discovery news in brief, from: Athira Pharma, J Ints Bio, Nicox, Seraxis.
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Neurology/Psychiatric

Gedeon Richter reports GABRA5 positive allosteric modulators

April 27, 2023
Gedeon Richter Ltd. has prepared and tested bicyclic amine derivatives acting as GABA-A receptor α5 subunit (GABRA5) positive allosteric modulators.
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Neurology/Psychiatric

Merck Sharp & Dohme patents LRRK2 inhibitors for Parkinson’s disease

April 27, 2023
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.
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