Vertex Pharmaceuticals Inc. has described α1-antitrypsin (SERPINA1) (Z-mutant) polymerization inhibitors reported to be useful for the treatment of α1-antitrypsin (AAT) deficiency.
Repair Biotechnologies Inc. has reported new data showing that its lipid nanoparticle (LNP)/messenger RNA (mRNA) therapy rapidly reversed the progression of atherosclerosis in mouse models applicable to familial hypercholesterolemia as well as atherosclerosis in the broader population.
Hoth Therapeutics Inc.’s subsidiary, Merveille.ai, has used advanced artificial intelligence to identify a promising new therapeutic candidate for obesity.
Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is a congenital metabolic disorder that leads to the accumulation of partially degraded heparan sulfate, which triggers neurodegeneration.
Beam Therapeutics Inc. has received clearance of its clinical trial authorization (CTA) application by the U.K.’s Medicines and Healthcare Products Regulatory Agency for BEAM-302, an in vivo base editor, as a potential treatment for patients with α1-antitrypsin deficiency (AATD).
Cinfina Pharma Inc. has obtained FDA clearance of its IND application for CIN-110, a PYY3-36 analogue, allowing it to initiate a first in-human study in otherwise healthy subjects with obesity. CIN-110 is a stable and long-acting analogue of PYY3-36 being developed both as a monotherapy and for co-administration for obesity.
Recent genome-wide association studies identified an association between low bone mineral density (BMD) and a single-nucleotide polymorphism (SNP) at the MALAT1 locus, but there is no functional evidence on the role of MALAT1 alterations in BMD or osteoporosis. Hence, scientists at MD Anderson Cancer Center aimed to assess the functional role of MALAT1 alterations in low BMD and osteoporosis.
A metabolite that suppresses appetite and food intake after exercise could be the reason for the weight loss observed in patients treated with metformin to control blood glucose. A study conducted by a group of scientists at Stanford University showed how this antidiabetic drug induced the biosynthesis of N-lactoyl-phenylalanine (Lac-Phe), which has an effect reducing the body mass index.
Stealth Biotherapeutics Inc. has presented new data demonstrating cardioprotective effects of SBT-589 in preclinical models of Friedreich’s ataxia (FA). SBT-589 acts on mitochondrial pathways essential for cellular health and energy production that are impaired in FA cardiomyopathy.
RSS
