Huidagene Therapeutics Co. Ltd. has presented data for HG-303, a new CRISPR-hfCas12Max-based therapeutic approach that knocks down ATXN2 expression for the treatment of amyotrophic lateral sclerosis (ALS).
Alchemab Therapeutics Ltd. has entered into a licensing agreement with Eli Lilly and Co. for ATLX-1282, Alchemab’s first-in-class IND-ready program targeting a novel receptor and mechanism for amyotrophic lateral sclerosis (ALS) and other neurodegenerative conditions.
Xellsmart Biopharmaceutical (Suzhou) Co. Ltd. has announced IND clearances by the FDA for its XS-411 and XS-228 cell therapies, for Parkinson’s disease and amyotrophic lateral sclerosis (ALS), respectively.
Sineugene Therapeutics Co. Ltd. has obtained IND clearance from the FDA for SNUG-01, a first-in-class tripartite motif protein 72 (TRIM72)-targeted gene therapy candidate for amyotrophic lateral sclerosis (ALS). A phase I/IIa trial will evaluate SNUG-01 in adults with ALS.
Voyager Therapeutics Inc. has announced its decision to assess alternate payloads related to its gene therapy program for superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). Emerging preclinical data indicate the siRNA payload component of VY-9323 does not meet its standards due to what appears to be an off-target effect resulting in a narrowed therapeutic window.
Lifearc has announced a collaboration with Neuropeutics Inc. to develop a new small molecule for the treatment of motor neuron disease (MND), or amyotrophic lateral sclerosis (ALS). Neuropeutics and Lifearc intend to develop a lead candidate therapeutic to prevent and reverse TDP-43 protein aggregation in MND/ALS.
Bristol Myers Squibb Co. has selected the first novel target for amyotrophic lateral sclerosis (ALS) that was identified and validated by Insitro Inc. under the companies’ 2020 collaboration agreement to discover new therapies for ALS. The selection triggers a milestone payment to Insitro.
Dewpoint Therapeutics Inc. has entered into a strategic research collaboration with Mitsubishi Tanabe Pharma Corp. to advance Dewpoint’s novel small-molecule condensate modulator targeting TDP-43 for amyotrophic lateral sclerosis (ALS).
Ractigen Therapeutics Co. Ltd.’s RAG-21, a novel siRNA therapy targeting the FUS gene, has been awarded orphan drug designation by the FDA for the treatment of amyotrophic lateral sclerosis (ALS).