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Articles Tagged with ''ALS''

Illustration demonstrating muscle contraction in amyotrophic lateral sclerosis.

Neurology/psychiatric

Huidagene’s HG-303 improves neuromuscular function in preclinical ALS

May 21, 2025

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).

Conceptual 3D illustration showing degradation of motor neuron

Neurology/psychiatric

Alchemab and Lilly sign licensing agreement for ATLX-1282

May 6, 2025

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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.

Neurology/psychiatric

Xellsmart announces US IND clearances for iPSC-derived cell therapies for Parkinson’s disease and ALS

April 16, 2025

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.

Neurology/psychiatric

Sineugene’s TRIM72-targeted gene therapy for ALS gains IND clearance from FDA

March 25, 2025

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.

Neurology/psychiatric

Voyager to evaluate alternate payloads for ALS gene therapy program

Feb. 12, 2025

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.

Neurology/psychiatric

Lifearc and Neuropeutics collaborate to advance small molecule for ALS

Jan. 28, 2025

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.

Neurology/psychiatric

Bristol Myers Squibb selects novel ALS target under Insitro collaboration

Dec. 19, 2024

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.

Neurology/psychiatric

Equity investment in Eikonizo supports HDAC6 inhibitor work

Dec. 10, 2024

Eikonizo Therapeutics Inc. has secured an equity investment from Novo Nordisk A/S.

Neurology/psychiatric

Dewpoint and Mitsubishi Tanabe collaborate on small-molecule condensate modulator for ALS

Dec. 5, 2024

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).

Neurology/psychiatric

Ractigen Therapeutics’ RAG-21 designated orphan drug for ALS

Nov. 20, 2024

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).

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BioWorld
BioWorld briefs for Jan 30, 2025.
Today's news in brief
Briefs
BioWorld MedTech briefs for Jan. 30, 2026.
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