The ALDH2 gene encodes mitochondrial aldehyde dehydrogenase 2 (ALDH2), a major acetaldehyde-metabolizing enzyme. Approximately 36% of East Asians (around 560 million people) carry an inactivating E504K missense mutation of the ALDH2 gene, which reduces ALDH2 enzymatic activity. This inactivating mutation has been correlated with several diseases and is strongly associated with type 2 diabetes, body mass index and serum lipids in East Asians.
Following an unexpected FDA complete response letter (CRL), Alnylam Pharmaceuticals Inc. said it will no longer pursue an expanded indication for Onpattro (patisiran) in the U.S. The RNAi therapeutic was approved in 2018 to treat polyneuropathy of hereditary transthyretin-mediated (ATTR) amyloidosis and seemed well on its way to snagging a second U.S. indication after the FDA’s Cardiovascular and Renal Drugs Advisory Committee voted 9-3 in September that the drug’s benefits outweighed its risks as a treatment for cardiomyopathy of ATTR amyloidosis. The FDA disagreed with the committee, saying in the CRL that patisiran’s clinical meaningfulness had not been established in the proposed indication.
Researchers from the Universities of Malaga and Cordoba (Spain) presented data from a study that aimed to identify matrisome regulator miRNAs in obesity and prediabetes that could represent novel noninvasive circulating biomarkers of adipose tissue (AT) fibrosis.
In the ultra-rare disease congenital hyperinsulinism, an overproduction of insulin leads to persistent hypoglycemia and can cause neurological complications due to high glucose needs of the brain. About half of children go on to develop seizures or intellectual problems, but current therapeutic options are limited and there are no approved drugs specifically for the condition.
Obesity is a chronic disease caused by the concurrence of genetics and environmental and individual behaviors that lead to an imbalance between caloric intake and expenditure and are linked to increased mortality and morbidity.
Researchers from East China Normal University and Shanghai Jiao Tong University presented the discovery and preclinical evaluation of new potent antiosteoporosis agents. Synthesis and optimization of a series of heterocyclic ring-fused derivatives of 20(S)-protopanaxadiol (PPD) led to the identification of SH-491 as the lead candidate with the most potent inhibitory effects on RANKL-induced osteoclastogenesis (IC50=11.8 nM).
Sab Biotherapeutics Inc. has announced a private placement of up to $130 to fund the company’s lead research program, SAB-142, a potential disease-modifying treatment for type 1 diabetes. SAB-142 is expected to advance into clinical trials in the fourth quarter of this year.
Three years after Alnylam Pharmaceuticals Inc. gained U.S. FDA approval of the first treatment for rare disease primary hyperoxaluria type 1 (PH1), Novo Nordisk A/S solidified its own marketing clearance for RNAi therapy Rivfloza (nedosiran).
Fudan University has patented compounds acting as microtubule-associated proteins 1A/1B light chain 3B (MAP1LC3B) inhibitors and proprotein convertase subtilisin/kexin-type 9 (PCSK9) degradation inducers reported to be useful for the treatment of alopecia, atherosclerosis, cancer, Alzheimer’s disease, pulmonary fibrosis, hyperlipidemia, keratosis and obesity, among others.
The FDA has cleared IND applications for UBT-251 injection, a long-acting triple-targeted glucagon-like peptide-1 (GLP-1)/glucose-dependent insulinotropic polypeptide (GIP)/glucagon receptor agonist developed by The United Bio-Technology (Hengqin) Co. Ltd., a wholly owned subsidiary of The United Laboratories International Holdings Ltd., for adult type 2 diabetes and for overweight or obese subjects.
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