Scientists at Indiana University and Purdue Research Foundation have synthesized inositol polyphosphate-5-phosphatase D (SHIP-1; INPP5D) inhibitors reported to be useful for the treatment of mild cognitive impairment, Alzheimer’s disease, vascular cognitive impairment, Lewy body dementia and frontotemporal dementia.
A preclinical study presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, showed a new epigenetic editing technology that enables durable gene silencing using ELXRs, short for Epigenetic Long-Term X-Repressors. With this approach, scientists at Scribe Therapeutics Inc. successfully inhibited the expression of the PCSK9 gene, a key regulator of cholesterol metabolism, in human cells, mice and nonhuman primates.
Suzhou Genhouse Bio Co. Ltd. has described histone deacetylase 6 (HDAC6) inhibitors reported to be useful for the treatment of cancer, asthma, Alzheimer’s disease, diabetes, amyotrophic lateral sclerosis, multiple sclerosis, pulmonary fibrosis and psoriasis, among others.
Adlai Nortye Biopharma Co. Ltd. and Adlai Nortye Pte Ltd. have divulged cyclin-dependent kinase (CDK) inhibitors reported to be useful for the treatment of cancer, inflammatory disorders and autoimmune diseases.
Purdue Research Foundation has identified conjugates targeting asialoglycoprotein receptors (ASGR) that are comprised of a therapeutic or imaging agent. They are reported to be potentially useful for the diagnosis and/or treatment of nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH).
Sichuan Kelun-Biotech Biopharmaceutical Co. Ltd. has synthesized proteolysis targeting chimera (PROTAC) compounds comprising a Von Hippel-Lindau disease tumor suppressor (VHL)-binding moiety covalently linked to a GTPase KRAS G12D mutant-targeting moiety through a linker reported to be useful for the treatment of cancer.
Neuronal ceroid lipofuscinosis type 1 (CLN1) disease, also known as infantile neuronal ceroid lipofuscinosis, is a rare and fatal neurodegenerative condition. CLN1 disease is caused by a deficiency in the enzyme palmitoyl-protein thioesterase 1 (PPT1) due to biallelic loss-of-function mutations in the gene encoding the lysosomal enzyme, leading to widespread neurological dysfunction and premature death.
Tubulin inhibitors by themselves can limit tumor growth, and they can be particularly effective when conjugated to monoclonal antibodies that target them to tumors. Two such conjugates, trastuzumab emtansine and brentuximab vedotin, have been approved by the FDA. However, many cancers can develop resistance to such inhibitors, highlighting the need for next-generation compounds.
Nabla Bio Inc. has established a new multiyear research collaboration with Takeda Pharmaceutical Co. Ltd. to advance AI-driven design of protein therapeutics.
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