Voyager Therapeutics Inc. has selected a lead development candidate for its superoxide dismutase 1 (SOD1)-mutated amyotrophic lateral sclerosis (ALS) gene therapy program. The company anticipates filing an IND application for the candidate with the FDA in mid-2025.

Neurosense Therapeutics Ltd. failed to impress Wall Street with top-line data from its phase IIb study called Paradigm with PrimeC, a fixed-dose combination of ciprofloxacin and celecoxib, in amyotrophic lateral sclerosis.

Oryzon Genomics SA has been awarded a $498,690 grant by the ALS Association to support the regulatory preclinical development of ORY-4001, a highly selective histone deacetylase 6 (HDAC6) inhibitor, for amyotrophic lateral sclerosis (ALS).

A multi-institutional research team has suggested that aberrant TDP-43 processing of the pre-mRNA of a microtubule-associated protein, stathmin-2 (STMN2), may be the primary contributor to amyotrophic lateral sclerosis (ALS).

Vectory Therapeutics BV has closed a €129 million ($138 million) series A financing to advance its vectorized antibody programs in neurodegenerative diseases.

Pasithea Therapeutics Corp. has selected a lead therapeutic candidate for its PAS-003 program, a proprietary humanized monoclonal antibody that targets α5β1 integrin, a protein overexpressed in both humans and mice with amyotrophic lateral sclerosis (ALS).

The EMA is standing firm on its refusal to recommend approval of the amyotrophic lateral sclerosis (ALS) treatment Albrioza in Europe after re-examining Amylyx Pharmaceuticals Inc.’s marketing authorization application and remaining unconvinced that the main study demonstrated the drug effectively slows disease progression.