Cerevance Inc. has expanded its series B financing with an additional close of US$51 million, bringing the total series B financing to US$116 million. The proceeds will be used to advance Cerevance's therapeutics for central nervous system (CNS) diseases developed using its proprietary Nuclear Enriched Transcript Sort sequencing (Netsseq) platform.
The suppression of the SYF2 factor could be a new therapeutic strategy for the treatment of the different types of amyotrophic lateral sclerosis (ALS). According to a study from the University of Southern California, SYF2 acts on the TDP-43 protein, improving the survival of motor neurons affected by this disease. “We wanted to find something that would improve neuron survival across many different iPSC lines for ALS,” Justin Ichida told BioWorld.
Amyotrophic lateral sclerosis (ALS) is the most frequent adult-onset motor neuron disease, and it is pathologically related with frontotemporal dementia (FTD). Genetic studies have identified C9ORF72 as a major genetic cause of ALS/FTD. Further genetic analyses and validation studies have identified some other genes associated with ALS risk, highlighting among them the NUP50 gene, which encodes nuclear pore complex protein Nup50.
The hexanucleotide repeat expansion (HRE) GGGGCC in the noncoding region of the chromosome 9 open reading frame 72 (C9ORF72) gene is the most common cause of hereditary (40%) and apparently sporadic (5%-6%) amyotrophic lateral sclerosis (ALS).
Eikonoklastes Therapeutics Inc. and Forge Biologics Inc. have established a manufacturing partnership to advance Eikonoklastes' adeno-associated viral (AAV)-based gene therapy, ET-101, into clinical trials for the treatment of patients with amyotrophic lateral sclerosis (ALS).
The FDA has awarded orphan drug designation to Eikonoklastes Therapeutics Inc.'s ET-101 program for the treatment of amyotrophic lateral sclerosis (ALS).
The wholesale acquisition cost for Amylyx Pharmaceuticals Inc.'s new U.S. FDA-approved amyotrophic lateral sclerosis (ALS) treatment, Relyvrio, has been set at about $158,000 for the first year’s treatment. It jumps to about $163,000 in the second year, the company said, a change that would move it closer to the cost of competitor Mitsubishi Tanabe Pharma Corp.'s Radicava (edaravone), which costs about $165,000 annually. A 28-day prescription will cost $12,504.
Frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) are two devastating neurodegenerative diseases, with clinical, pathological and genetic overlap, without effective therapy.
Human endogenous retrovirus-K (HERV-K) belongs to the β-retrovirus-like supergroup of viruses, and it has been previously demonstrated that the expression of HERV-K subtype HML-2 envelope (Env) in human neuronal cultures or in transgenic mice resulted in neurotoxicity and neurodegeneration.
