Wave Life Sciences Ltd. and GSK plc have entered into a strategic collaboration to advance oligonucleotide therapeutics, including Wave's preclinical RNA editing program targeting α1-antitrypsin deficiency (AATD), WVE-006. The discovery collaboration has an initial 4-year research term. The first part of the arrangement is a discovery collaboration that enables GSK to advance up to eight programs and Wave to advance up to three programs, leveraging Wave's PRISM oligonucleotide platform and GSK's expertise in genetics and genomics.
The California Institute for Regenerative Medicine (CIRM) has awarded Calidi Biotherapeutics Inc. a US$3.1 million grant to support continued development of the company's Supernova-1 (SNV-1) preclinical program through IND application. The grant was awarded to Calidi to support IND-enabling studies, finalize manufacturing and the completion of Calidi's IND application for the SNV-1 program.
Programmable genome insertion of long DNA sequences, useful for both gene therapy and basic research, commonly relies on cellular responses to double-strand breaks (DSBs) using programmable nucleases, such as CRISPR-Cas9, for induction of repair pathways such as non-homologous end joining (NHEJ). To overcome the current limitations of gene integration approaches, scientists from the Massachusetts Institute of Technology and colleagues developed a new strategy based on advances in programmable CRISPR-based gene editing, such as prime editing, together with the application of precise site-specific integrases.
New and updated preclinical data presented at the American Society of Hematology Annual Meeting in New Orleans, by: Affimed, Asher Biotherapeutics, Disc Medicine, Keros Therapeutics, Kymera Therapeutics, Neoleukin Therapeutics, NGM Biopharmaceuticals, Oric Pharmaceuticals, Salarius Pharmaceuticals, Vincerx Pharma.
Jiangsu Chia Tai Tianqing Pharmaceutical Group Co. Ltd. has presented solute carrier family 22 member 12 (SLC22A12) inhibitors reported to be useful for the treatment of gout and hyperuricemia.
Chengdu Baiyu Pharmaceutical Co. Ltd. has identified piperazine derivatives acting as protein mono-ADP-ribosyltransferase TIPARP inhibitors reported to be useful for the treatment of cancer.
Aurigene Discovery Technologies Ltd. has divulged fused isoxazolyl compounds acting as histone acetyltransferase KAT6A (monocytic leukemia zinc finger protein; MOZ; MYST-3) inhibitors reported to be useful for the treatment of cancer.
Clearmind Medicine Inc. has completed IND-enabling studies with its 5-methoxy-2-aminoindane (MEAI)-based lead compound, CMND-100, aimed at treating alcohol use disorder (AUD). The company is preparing for an IND submission early next year to the FDA and the Israeli Ministry of Health to start first-in-human phase I/IIa studies in the U.S. and Israel.
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