The endocannabinoid system is an endogenous neuromodulatory system that is highly expressed in the central nervous system (CNS) and peripheral tissues and is known to play an important role in modulating learning and memory processes, where the cannabinoid CB1 receptor is the most widely expressed G protein-coupled receptor in the CNS, although its presence is also high in peripheral tissues.
Tribune Therapeutics AB has raised €37 million (US$40 million) in seed and series A funding to advance a portfolio of therapies targeting central drivers of scar tissue formation.
Precision Biosciences Inc. recently presented a new gene-editing approach, PBGENE-DMD, which could allow life-long benefits to patients with Duchenne muscular dystrophy (DMD).
Epicrispr Biotechnologies Inc. has secured $68 million in the first close of its series B financing to support the clinical development of EPI-321, a first-in-class epigenetic therapy for facioscapulohumeral muscular dystrophy (FSHD).
Decoy Therapeutics Inc. has announced that a series of antiviral drug candidates previously designed by its Imp3act platform to be broadly effective against viruses of the paramyxoviridae family have also shown promising in silico activity against measles and Nipah viruses. The candidates were previously shown to be active in vitro against respiratory syncytial virus and human parainfluenza virus 3.
Researchers from ADT Pharmaceuticals and affiliated organizations have published preclinical data for the pan-RAS inhibitor ADT-007, being developed as an anticancer agent.
The first disease modifying therapies for Alzheimer’s may have limited utility in some senses, but they will be a force for change, providing momentum and altering the way governments as payers, and health systems as carers, think about the disease.
Transplanting an animal organ into a human is now a closer reality following the successful xenotransplantation of a genetically modified pig liver into a patient diagnosed with brain death in China. The operation was intended to evaluate organ function over a 10-day period. This is a complex experimental trial that did not involve removing the patient's liver and still requires further study. However, the positive preclinical results suggest this strategy could save the lives of those waiting for a human organ, at least in certain cases.
Agios Pharmaceuticals Inc. has patented new 4-pyrazolo[1,5-a]pyridin-2-yl-4,5,6,7-tetrahydro-1h-imidazo[4,5-c]pyridine derivatives acting as phenylalanine hydroxylase (PAH) R408W mutant stabilizers reported to be useful for the treatment of phenylketonuria.