BioWorld - Saturday, January 10, 2026

Articles Tagged with ''ALS''

Neurology/Psychiatric

Blocking nuclear export of ALS/FTD troublemaker helps neurons survive

March 3, 2023
By Anette Breindl
Treatment with a cell-penetrating peptide that prevented nuclear export of unprocessed C9ORF72 RNA and its subsequent translation into neurotoxic dipeptide repeat proteins reduced motor neuron damage and death both in fruit fly models of amyotrophic lateral sclerosis (ALS), and in patient-derived induced neuronal precursor cells (iNPCs). The work suggests that targeting nuclear export could be a therapeutic option in ALS, and possibly also frontotemporal dementia (FTD), where C9ORF72 mutations also play a role.
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Brain and neural networks
Newco news

Newly public Coya takes on neurodegenerative disease with Tregs

Feb. 15, 2023
By Jennifer Boggs
Fresh off an end-of-year IPO, Coya Therapeutics Inc. is gearing up for clinical testing with its lead Treg-enhancing biologic in neurodegenerative disease, aiming to build on a wealth of academic-generated data highlighting the potential of Treg therapy to attack the neuroinflammation underlying diseases such as amyotrophic lateral sclerosis and Alzheimer’s disease.
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Illustration demonstrating muscle contraction in amyotrophic lateral sclerosis.
Neurology/Psychiatric

Cerevance expands series B financing to advance therapeutics for CNS diseases

Feb. 14, 2023
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.
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Conceptual 3D illustration showing degradation of motor neuron
Neurology/Psychiatric

SYF2 is novel target for neuronal survival in ALS

Feb. 6, 2023
By Mar de Miguel
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.
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Brain and DNA
Biomarkers

NUP50 gene variation tied to ALS risk, researchers find

Jan. 26, 2023
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.
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FDA Approved stamp

Amylyx prices newly approved ALS drug, Relyvrio

Sep. 30, 2022
By Lee Landenberger
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.
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