Dewpoint Therapeutics has selected a novel small molecule designed to prevent the formation of abnormal TDP-43 protein clumps that drive nerve cell death in amyotrophic lateral sclerosis (ALS) and other related conditions. The candidate is specifically designed to target condensates, or membrane-less structures that concentrate specific molecules to…
small molecule
AUT00201, an experimental oral small molecule being developed by Autifony Therapeutics, reduced the loss of motor neurons — the nerve cells that control movement — and eased motor symptoms in a mouse model of amyotrophic lateral sclerosis (ALS), a new study showed. It was also found to improve…
Spinogenix will soon launch a Phase 1/2 clinical trial in the U.S. to test its investigational small molecule SPG302 in people with amyotrophic lateral sclerosis (ALS). The trial, which will assess the medication’s safety, tolerability, and pharmacological properties when given as a once-daily pill, comes after The U.S.
Enrollment is complete in a Phase 2 trial testing InFlectis BioScience’s oral candidate IFB-088 (icerguastat) in people with amyotrophic lateral sclerosis (ALS), the company has announced. The trial (NCT05508074) enrolled a total of 50 adults with bulbar-onset ALS, a form of the disease that first affects the…
Spinogenix has been awarded a research grant from the U.S. Department of Defense (DOD) to further test its lead compound as a potential treatment of amyotrophic lateral sclerosis (ALS), the company announced. “We are pleased that the DoD has recognized the potential of our novel drug candidate to…
News
Small-Molecule Compounds ID’d That May Lessen Protein Buildup in Cells Under Stress, an ALS Hallmark
Researchers identified small-molecule compounds that help to prevent the build-up of stress-induced clumps of TDP-43 protein, a hallmark of amyotrophic lateral sclerosis (ALS). These findings, although preliminary, may lead to new therapies for ALS. The work “Small-Molecule Modulation of TDP-43 Recruitment to Stress Granules Prevents Persistent TDP-43 Accumulation in…
Researchers have developed a new small molecule that targets the most common genetic defect behind amyotrophic lateral sclerosis (ALS) and frontotemporal dementia, a study reports. These findings suggest that therapies directed at the underlying cause of ALS and frontotemporal dementia may be achievable in the near future. The…
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