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.
At the ongoing ACS meeting in New Orleans, Rezubio Pharmaceuticals Co. Ltd. detailed the discovery and preclinical evaluation of novel small-molecule gut-restricted GPR40 agonists using membrane-anchored drug (MADD) design.
The University of California, Berkeley has announced the launch of the UC Berkeley Molecular Therapeutics Initiative (MTI) designed to accelerate drug discovery. The aim is to bridge fundamental research in rare neurological and metabolic diseases with drug discovery to identify and accelerate novel therapeutic modalities into the clinic.
Cascade Pharmaceuticals Inc. has synthesized thyroid hormone receptor β (THRβ) agonists reported to be useful for the treatment of MASLD/NAFLD, hypothyroidism and more.
Fractyl Health Inc. has reported promising new preclinical findings supporting RJVA-001, the first clinical candidate in its Rejuva pancreatic gene therapy platform. The company’s GLP-1 gene therapy candidate has potential to treat metabolic diseases, including obesity and type 2 diabetes.
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