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).
Merck & Co. Inc. continued to broaden its reach in neurodegenerative diseases by paying, through a subsidiary, as much as $610 million to take over preclinical-stage Caraway Therapeutics Inc. The deal involves an undisclosed up-front payment along with contingent milestone rewards.
Amyotrophic lateral sclerosis (ALS) is a disorder that leads to progressive muscle weakness and loss of muscle control due to affectation of the motor neurons. Increasing evidence points to defects in the nuclear envelope, which leads to disease progression.
Fundamental Pharma GmbH has described TRPM4/NMDA interaction inhibitors reported to be useful for the treatment of 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).
Huntington’s disease (HD) is caused by the CAG trinucleotide repeat expansion in exon 1 of the huntingtin (HTT) gene, leading to polyglutamine-expanded stretch of mutant huntingtin (mHTT) protein. Previous research has demonstrated that knockdown of HTT could represent an effective strategy for the inhibition of the formation of mHTT protein, and a recent study conducted by researchers from Huidagene Therapeutics Co. Ltd. aimed to assess the potential of high-fidelity Cas12Max (hfCas12Max)-based gene editing therapy as a novel treatment for HD.
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.
Brainstorm Cell Therapeutics Inc. said it’s exploring all its options in the wake of a Sept. 27 U.S. FDA advisory committee vote, in which the committee overwhelmingly disagreed with the company that the data it presented supported the effectiveness of Nurown (debamestrocel) for the treatment of mild to moderate amyotrophic lateral sclerosis.
Brainstorm Cell Therapeutics Inc.’s Nurown got a thumbs down from the U.S. FDA’s Cellular, Tissue and Gene Therapies Advisory Committee Sept. 27, as the committee voted 1-17, with one abstention, that the data presented demonstrated substantial evidence of effectiveness for treatment of mild to moderate amyotrophic lateral sclerosis.
