NRG Therapeutics Ltd. has been awarded a grant from Target ALS Foundation to support its discovery program for a treatment for amyotrophic lateral sclerosis (ALS).
Mid-Atlantic Biotherapeutics Inc. and Accelero Biostructures have entered into a research collaboration to identify novel USP30 inhibitors for neurological disorders and other indications.
Essential branched-chain amino acids (BCAA) such as leucine, isoleucine or valine, cannot be synthesized by mammals, making them an obligate part of the diet.
Adaptin Bio has obtained IND clearance from the FDA for APTN-101 in glioblastoma, enabling initiation of a first-in-human phase I trial in patients diagnosed with WHO grade IV malignant glioma.
At the European Federation for Medicinal chemistry and Chemical biology’s International Symposium on Medicinal Chemistry (EFMC-ISMC) held this week in Rome, Novartis Biomedical Research reported the discovery and evaluation of NVP-EVS-459, a low molecular weight folate receptor (FR)-targeting radioconjugate, for the potential treatment and diagnosis of cancer.
Vivet Therapeutics SAS has announced its gene therapy VTX-806 has been awarded European orphan drug designation for the treatment of cerebrotendinous xanthomatosis (CTX), a rare disorder of bile acid metabolism.
Researchers from Astrazeneca plc presented the structure and preclinical characterization of a novel PCSK9 inhibitor, AZD-0780, being developed for the treatment of cardiovascular disease. A new potentially druggable functional binding pocket was identified on the PCSK9 C-terminal domain (CTD), and multiple fragment screens generated a single CTD-binding hit.
A team of scientists from the Perelman School of Medicine at the University of Pennsylvania and Aum Biotech LLC have described the development of a novel cancer immunotherapy designed to target FOXP3-positive T regulatory cells (Tregs) with a next generation of antisense oligonucleotides (ASOs), termed FOXP3 Aumsilence ASO. In contrast to previous ASOs, FOXP3 Aumsilence ASOs do not require delivery agents, and are capable of highly specific RNA silencing of previously undruggable targets.
Artificial intelligence (AI) drug discovery company Noetik Inc. has closed on an oversubscribed $40 million series A financing round. The company plans to use the money to expand its atlas of human cancer biology with its in vivo CRISPR platform to advance a pipeline of cancer therapeutics to the clinic. In describing its approach, the company said that making a genuine impact on drug discovery requires computational capabilities to understand and simulate disease biology at the patient level, identifying the right targets and matching them with the right therapies.
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