Ampersand Biomedicines Inc. has secured $65 million in series B funding from investors including Ampersand’s founder, Flagship Pioneering, to support advancement of two lead programs and a new discovery partnership in obesity.
Prime Medicine Inc. has announced its new preclinical program for the treatment of α1-antitrypsin deficiency (AATD). The program is advancing through its final stages of lead optimization, with an IND and/or clinical trial application (CTA) filing anticipated around the middle of next year.
Opko Health Inc. and Entera Bio Ltd. have entered into a collaboration and license agreement to advance an oral dual agonist GLP-1/glucagon peptide as a once-daily tablet into the clinic for patients with obesity, metabolic and fibrotic disorders.
Pluvia AS has synthesized pharmacological chaperones able to stabilize phenylalanine hydroxylase (PAH) reported to be useful for the treatment of hyperphenylalaninemia, particularly phenylketonuria (PKU).
The Ca2+ stored in the cellular endoplasmic reticulum (ER) plays a crucial role in protein folding and lipid transfer, and its impairment leads to cellular ER stress. When chronic cellular ER stress occurs in the liver, it triggers the pathogenesis of metabolic dysfunction-associated steatotic liver disease (MASLD) and metabolic dysfunction-associated steatohepatitis (MASH). Previous reports found that NACHT and WD repeat domain containing 1 (NWD1) localized in the ER and mitochondria in neural stem/progenitor cells, but the significance of NWD1 outside the brain is not well known.
Harbour Biomed Ltd. has announced the launch of Élancé Therapeutics with an aim to improve the treatment of obesity. Élancé seeks to address challenges in obesity treatment, by increasing overall body weight loss and fat mass loss, while preserving and even increasing muscle and lean mass.
Endocannabinoids are lipid mediators that interact with G protein-coupled receptors, including cannabinoid CB2 receptor (CB2R), which is mainly expressed in peripheral tissues with immune functions.
Previous research has demonstrated that activin receptor-like kinase 7 (ALK7) signaling suppresses lipolysis, resulting in increased adipocyte size and lipid content. Additionally, predicted loss of function variants in the ALK7 gene (ACVR1C) have been associated with reduced waist-to-hip ratio (WHR) adjusted for body mass index, protection from type 2 diabetes, as well as reduced risk of cardiovascular disease.
Jnana Therapeutics Inc. has described sodium-dependent neutral amino acid transporter B(0)AT1 (SLC6A19) inhibitors reported to be useful for the treatment of diabetes, chronic kidney disease, nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH), phenylketonuria, metabolic syndrome, obesity, neurodevelopmental disorders and autism spectrum disorders, among others.
Epigenetic editing is a promising method for gene regulation in vitro and in vivo, allowing precise control of gene expression without altering the DNA sequence, thereby minimizing genotoxic risks.
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