Argenx SE’s ARGX-119 is a monoclonal antibody (MAb) targeting the muscle-specific kinase (MuSK) Frizzled (Fz)-like domain, and has entered early clinical development for the treatment of neuromuscular diseases.

It has been previously demonstrated that the activation of spleen tyrosine kinase (SYK) contributes to processes that are central to the pathogenesis of neurodegenerative diseases, such as Alzheimer’s disease (AD) and Parkinson’s disease (PD).

NRG Therapeutics Ltd. has been awarded a grant from Target ALS Foundation to support its discovery program for a treatment for amyotrophic lateral sclerosis (ALS).

Acurastem Inc. has secured $4 million in grant funding from the California Institute for Regenerative Medicine (CIRM) to facilitate the development of its UNC13A program toward clinical trials for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

The big advantage of cell culture to model diseases is its throughput. “You can play the disease over and over again in the dish,” Clive Svendsen told the audience at the International Society of Stem Cell Research (ISSCR) Annual Meeting held in Hamburg last week. That high throughput, however, is not particularly useful if the cell lines themselves do not accurately model the disease. Cancer cell lines are used in many cell culture experiments far beyond cancer for their ability to grow. But they are “highly abnormal,” Bill Skarnes told the audience at an innovation showcase, as well as quite unstable. “I don’t think the [HEK-293] cell line is the same in your lab as it is in the lab next door,” Skarnes said.

Many patients with amyotrophic lateral sclerosis (ALS) or frontotemporal dementia (FTD) harbor the (G4C2)n pathogenic repeat expansion in the C9orf72 gene, which leads to aggregating dipeptide proteins, mainly poly-glycine-alanine (poly-GA).

Two drugs were pushed back by the EMA last week, with a recommendation that Ocaliva, currently the only second line standard of care for treating primary biliary cholangitis, be withdrawn from the market, and a refusal to grant conditional approval for masitinib in the treatment of amyotrophic lateral sclerosis.

Nervgen Pharma Corp. has outlined its plans to develop its new drug candidate, NVG-300, with a focus on three initial indications: ischemic stroke, amyotrophic lateral sclerosis (ALS) and spinal cord injury (SCI).

Shanghai Henlius Biotech Inc. has announced preclinical data showing that its small-molecule drug HLX-99 exhibited antineurodegenerative activities in multiple models, warranting further development.