Mammoth Biosciences Inc. has nominated its first clinical development candidate – MB-111 – a potential one-time treatment for patients with very high triglycerides, including familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia. IND-enabling studies are on track to begin this year.
Adult skeletal muscle tolerates a lack of the coenzyme nicotinamide adenine dinucleotide (NAD), according to a study led by scientists at the University of Copenhagen. Their results suggest that adverse effects previously associated with congenital NAD depletion in this tissue may be due to impaired muscle development rather than to a deficiency of this molecule.
Innorna Co. Ltd.’s investigational mRNA therapy, IN-013, has been awarded orphan drug and rare pediatric disease designations by the FDA for the treatment of Wilson disease.
Metabolic dysfunction-associated steatotic liver disease (MASLD) is a growing global health concern, with an estimated prevalence of around 25% worldwide. This chronic liver condition is characterized by lipid deposition in the liver, which can lead to inflammation, scarring and even liver cancer if left untreated.
A metabolic vulnerability of pancreatic ductal adenocarcinoma (PDAC) could be used to address this type of cancer that often resists treatments. Scientists at the University of Michigan have discovered that inhibiting the PIKfyve enzyme prevented tumor development and reduced tumor growth by altering the lipid synthesis these cells rely on. The KRAS-MAPK pathway is involved in this process, leading the researchers to suggest that dual inhibitors of PIKfyve and KRAS-MAPK could be an effective therapeutic strategy.
Oral peptide delivery specialist Cyprumed GmbH is about to find out if the high bioavailability of its tablet formulations seen in animal models will translate across to humans, after signing a $493 million license and option agreement with Merck & Co. Inc.
In a newly published study, researchers from the CSIR Institute of Genomics and Integrative Biology and collaborators further explored the potential of Cdk5 targeting as a therapeutic approach for type 2 diabetes and cognitive deterioration.
Northstrive Biosciences Inc., a subsidiary of PMGC Holdings Inc., has entered into a development and license agreement with Yuva Biosciences Inc. to discover and develop novel pharmaceutical treatments for obesity, type 2 diabetes and other cardiometabolic conditions using Yuva’s mitochondrial science-focused platform Mitonova, powered by AI.
Metachromatic leukodystrophy (MLD) is an autosomal recessive lysosomal storage disease originating from biallelic pathogenic variants in the ARSA gene, mainly affecting young children.
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