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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.

Illustration demonstrating muscle contraction in amyotrophic lateral sclerosis.

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).

Neurology/psychiatric

Lilly to develop ALS therapeutics against targets identified under Verge collaboration

Nov. 20, 2024

Eli Lilly & Co. has elected to pursue the development of therapeutics against two validated drug targets for amyotrophic lateral sclerosis (ALS) from its collaboration with Verge Analytics Inc. (dba Verge Genomics).

Neurology/psychiatric

Sea Pharmaceuticals advances candidates for neurological disorders

Nov. 15, 2024

Sea Pharmaceuticals LLC has announced it is advancing two new orally administered neurotherapeutic molecules for neurological disorders. SPM-0404 and SPM-0606 are biologically active as dual AMPA receptor (AMPAR) and kainate receptor (KAINR) antagonists.

Articles Tagged with ''ALS''

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

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

Voyager to evaluate alternate payloads for ALS gene therapy program

Lifearc and Neuropeutics collaborate to advance small molecule for ALS

Bristol Myers Squibb selects novel ALS target under Insitro collaboration

Equity investment in Eikonizo supports HDAC6 inhibitor work

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

Ractigen Therapeutics’ RAG-21 designated orphan drug for ALS

Lilly to develop ALS therapeutics against targets identified under Verge collaboration

Sea Pharmaceuticals advances candidates for neurological disorders

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