The dark matter of long non-coding RNA (lncRNA) is shades brighter, after the signing of two major deals between biotech pioneers and big pharma in the past week. Haya Therapeutics SA announced Sept. 4 that it has sealed a multiyear agreement with Eli Lilly and Co. to apply its lncRNA platform technology to identify targets in obesity and related metabolic disorders. Read More
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. Read More
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. Read More
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. Read More
Nanjing Huanbo Biotechnology Co. Ltd. has disclosed proteolysis targeting chimeras (PROTACs) comprising an E3 ubiquitin ligase binding agent coupled to a β-adrenergic receptor kinase 1 (BARK1; GRK2) targeting moiety via a linker acting as GRK2 degradation inducers. Read More
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. Read More
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. Read More
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. Read More
Merck Sharp & Dohme LLC has identified plasma kallikrein (KLKB1) Inhibitors reported to be useful for the treatment of diabetic retinopathy, retinal vein occlusion, hereditary angioedema, diabetes, pancreatitis, intracerebral hemorrhage, nephropathy and inflammatory disorders. Read More
Mid-Atlantic Biotherapeutics Inc. and Accelero Biostructures have entered into a research collaboration to identify novel USP30 inhibitors for neurological disorders and other indications. Read More
Nanjing Mingde New Drug Research Co. Ltd. has divulged pyrimidinone derivatives acting as P2X purinoceptor 3 (P2RX3; P2X3) antagonists reported to be useful for the treatment of cough. Read More
Jiangsu Vcare Pharmatech Co. Ltd. has described proteolysis targeting chimera (PROTAC) compounds comprising an E3 ubiquitin ligase binding moiety covalently linked to a Bruton’s tyrosine kinase (BTK) targeting moiety via a linker reported to be useful for the treatment of cancer. Read More
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). Read More
