Axonal degeneration is a key early pathogenic driver of amyotrophic lateral sclerosis and other neurodegenerative diseases. Activation of calpain-2 (CAPN2) is thought to be the critical effector behind axonal degeneration. Amylyx Pharmaceuticals Inc. has presented data on their CAPN2 inhibitor antisense oligonucleotide (ASO) AMX-0114 as a potential therapeutic for axonal degeneration.
Spirits were high at the 2023 Annual Meeting of the American Society of Hematology (ASH), buoyed by U.S. FDA approval of the first two gene therapies for sickle cell disease (SCD) the day before the conference kicked off in San Diego.
The Nobel Prize-winning modification that prevents the innate immune system from recognizing injected mRNA as foreign and blocking transcription of the protein it encodes has been found on some occasions to cause ribosomal frameshifting.
Additional early-stage research and drug discovery news in brief, from: Adolore Biotherapeutics, Alterity Therapeutics, Artelo Biosciences, Celcuity, Marengo Therapeutics, Mira Pharmaceuticals.
In accordance with the publishing schedule, BioWorld Science was not published on Wednesday, Dec. 6, 2023, and will not be published on Friday, Dec. 8, 2023.
Ractigen Therapeutics Co. Ltd. has submitted a clinical trial application in Australia seeking to conduct a phase I study of RAG-01, a small activating RNA (saRNA) drug candidate, in patients with non-muscle-invasive bladder cancer who have not responded to Bacillus Calmette-Guérin (BCG) therapy.
Janssen Pharmaceutica NV had disclosed new pyrrolidinone derivatives acting as NF-κB-inducing kinase (NIK; MAP3K14) inhibitors and reported to be useful for the treatment of cancer.
Kinesin-like protein KIF18A inhibitors reported to be useful for the treatment of cancer have been detailed in a recent GT Apeiron Therapeutics patent.
Research at Nanjing Gritpharma Co. Ltd. has led to the identification of stilbene derivatives acting as aryl hydrocarbon receptor (AhR) agonists. As such, they are reported to be useful for the treatment of allergy, asthma, atherosclerosis, diabetes type 2, infections, osteoporosis, transplant rejection and graft-vs.-host disease.
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