Pipeline Therapeutics Inc. has received FDA clearance to initiate a phase I trial of PIPE-791 in healthy volunteers, with dosing expected to begin in the second half of this year. PIPE-791 is advancing toward clinical development for remyelination and neuroinflammation, with a leading indication of multiple sclerosis (MS).
Dtx Pharma Inc.’s Dtx-1252, an investigational small interfering RNA (siRNA) therapeutic, has been awarded U.S. orphan drug designation for the treatment of Charcot-Marie-Tooth disease type 1A (CMT1A).
Sanofi SA has prepared and tested isoxazolidines acting as receptor-interacting serine/threonine-protein kinase 1 (RIPK1; RIP-1) inhibitors. As such, they are reported to be useful for the treatment of Alzheimer’s disease, multiple sclerosis, amyotrophic lateral sclerosis and incontinentia pigmenti.
Two Vigil Neuroscience Inc. patents describe triggering receptor expressed on myeloid cells 2 (TREM2) agonists reported to be useful for the treatment of frontotemporal dementia, multiple sclerosis, rheumatoid arthritis, stroke, prion infections, Parkinson’s, Alzheimer’s and Nasu-Hakola diseases.
“One of the many reasons we don’t have effective therapies for AD at the moment ... is that we don’t understand the beginnings of the disease,” Constanze Depp told BioWorld. Understanding those beginnings is likely to be a necessary prerequisite for truly turning the tide on Alzheimer’s disease (AD). “The brain is so bad at repairing itself, and once a neuron is lost, it will most likely not regenerate,” she elaborated. Now, Depp and her colleagues have reported on a contributor to those beginnings.
Researchers from Seelos Therapeutics Inc. presented the discovery and preclinical evaluation of a gene therapy candidate, SLS-009, for the treatment of Huntington’s disease (HD).
The University of Oxford and Selvita SA have established an integrated drug discovery collaboration in the area of Parkinson’s disease. Based on evidence implicating α-synuclein aggregates as the cause of Parkinson’s, the approach looks at the inability of cells to efficiently clear these aggregates, which may lead to the death of neurons and the emergence of characteristic symptoms of the disease. Promoting the ability of nerve cells to rid themselves of these aggregates therefore offers a possible therapeutic strategy for Parkinson’s disease.
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