Beam Therapeutics Inc. has offered a progress update on its genetic disease franchise. BEAM-302, the company’s priority genetic disease program, is a potential treatment for α1-antitrypsin deficiency (AATD).
Fractyl Health Inc. has nominated RJVA-001 as the first clinical type 2 diabetes candidate from its Rejuva gene therapy platform, which is designed to deliver locally administered genetic medicines to the pancreas.
Researchers from Guandong Pharmaceutical University reported on the synthesis and preclinical characterization of a series of novel long-acting peroxisome proliferator-activated receptor (PPAR) pan-agonists targeting PPARα, PPARδ, and PPARγ with lipid-regulating effects. PPARs are involved in lipid and lipoprotein metabolism, thus targeting the PPARs system is a promising strategy for the management of metabolic syndrome.
Diabetic retinopathy is the main diabetes complication affecting the eyes, and it leads to irreversible vision loss; oxidative stress and impairment of the retinal Muller cells are among the pathogenic mechanisms. Chinese researchers presented data on adiporon, an agonist of the adiponectin receptor protein 1 (ADPOR1) and ADIPOR2 that plays a crucial role in the metabolism of lipids and glucose, in preclinical mouse models of diabetes induced by streptozotocin.
Biocells (Beijing) Biotech Co. Ltd. has patented isoquinolinone derivatives acting aquaporin-4 (AQP4) inhibitors and thus reported to be useful for the treatment of hypervolemia (fluid overload) and cerebral ischemia.
Fauna Bio Inc. has signed a multi-year agreement with Eli Lilly and Co. to apply Fauna’s Convergence artificial intelligence (AI) platform to support preclinical drug discovery efforts in obesity.
In their year-end list of top scientific achievements and the people who made them, both Science and Nature have included the fight against “the obesity epidemic.” Science named GLP-1 drugs as its Breakthrough of the Year, while Nature included Svetlana Mojsov in its 2023 list of the year’s most important investigators. Mojsov is research associate professor at The Rockefeller University and was an early contributor to understanding the metabolic role of GLP.
Olix Pharmaceuticals Inc. has received approval from the Alfred Human Research Ethics Committee (HREC) in Australia to conduct a phase I clinical trial of drug candidate OLX-75016 for the treatment of nonalcoholic steatohepatitis (NASH).
Researchers at Cerevance Inc. and Cerevance Ltd. have identified heterobicyclic amides acting as ADP-ribosyl cyclase/cyclic ADP-ribose hydrolase 1 (CD38) inhibitors reported to be useful for the treatment of aging, diabetic nephropathy, glaucoma, inflammatory disorders, metabolic syndrome, obesity and rheumatoid arthritis, among others.
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