Pharmaust Ltd.’s monepantel met its primary safety endpoints and showed positive signals of potential efficacy in a phase I trial in patients with motor neuron disease (MND)/amyotrophic lateral sclerosis (ALS). With these results, the company will now progress to a pivotal phase II/III trial by midyear, Pharmaust CEO Michael Thurn told BioWorld.
Researchers from Macquarie University have detailed the discovery of a novel gene therapy vector targeting pathological TAR-binding protein 43 (TDP-43), CTx-1000, as a potential therapeutic candidate for the treatment of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) – two diseases characterized by cytoplasmic deposition of the nuclear TDP-43.
The discovery of a complex formed by two types of ion channels in neurons has allowed researchers from Heidelberg University to develop an inhibitor that stopped motor neuron degeneration in amyotrophic lateral sclerosis (ALS) in mouse models and human brain organoids.
Myrobalan Therapeutics Inc. has been awarded a $400,000 grant from the ALS Association to support the advancement of its colony-stimulating factor 1 receptor (CSF-1R) inhibitor for the treatment of amyotrophic lateral sclerosis (ALS).
Acurastem Inc. has raised nearly $7 million in grant funding from the National Institutes of Health (NIH) and the Department of Defense (DoD) to advance research in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia.
Coya Therapeutics Inc. intends to expand proposed indications for COYA-302 beyond amyotrophic lateral sclerosis (ALS) to include frontotemporal dementia (FTD) and Parkinson’s disease.
4D Molecular Therapeutics Inc. (4DMT) and Arbor Biotechnologies Inc. have established a strategic partnership focused on advancing new AAV-based gene-editing therapies for central nervous system (CNS) diseases with high unmet medical need in both rare and common disease populations.
Sudo Biosciences Inc. has closed a $116 million series B financing, with the funding raised to be used to advance two investigational TYK2 candidates into the clinic in 2024.
Researchers from West China Hospital of Sichuan University presented data from a study that aimed to investigate the associations between serum cystatin C (CysC) levels and the progression and survival of patients with amyotrophic lateral sclerosis (ALS).
