Keros Therapeutics Inc. has presented data regarding their activin receptor ligand trap, RKER-065, for the inhibition of the activin/myostatin signaling axis.
Acurastem Inc. has received a two-year research grant from Target ALS to advance therapeutics targeting SYF2, a recently identified regulator of TDP-43 function. TDP-43 dysfunction is a central biological hallmark of amyotrophic lateral sclerosis (ALS).
The exact genetic and epigenetic cause of the sporadic form of amyotrophic lateral sclerosis (ALS), which affects approximately 90% of patients, are largely unknown. Previous work found that mitochondrial dysfunction and metabolic dysregulation are crucial to ALS pathophysiology.
Newco Vesalic Ltd. has formed to take forward research indicating extracellular vesicles secreted by skeletal muscle cells carry toxic payloads that are key drivers of motor neuron diseases, including amyotrophic lateral sclerosis. The discovery of this process, which is largely external to the brain and the central nervous system, has opened up new targeting possibilities, and Vesalic is now working on in vivo studies to demonstrate preclinical proof of concept.
Newco Vesalic Ltd. has formed to take forward research indicating extracellular vesicles secreted by skeletal muscle cells carry toxic payloads that are key drivers of motor neuron diseases, including amyotrophic lateral sclerosis. The discovery of this process, which is largely external to the brain and the central nervous system, has opened up new targeting possibilities, and Vesalic is now working on in vivo studies to demonstrate preclinical proof of concept.
Vesalic has characterized a systemic metabolic dysfunction that creates a toxic exosome cargo in amyotrophic lateral sclerosis (ALS) patients, which is carried to the CNS, where it binds to and damages neurons, yielding a novel druggable target against the disease.
About 10% of amyotrophic lateral sclerosis (ALS) cases result from inherited genetic mutations, with about 20% of them attributed to mutations in the gene encoding the ubiquitous cytoplasmic copper/zinc superoxide dismutase 1 (SOD1).
Aperture Therapeutics Inc. has advanced its matrix metalloproteinase-9 (MMP-9) antisense oligonucleotide (ASO) program, APRTX-003, for the treatment of amyotrophic lateral sclerosis (ALS). This first-in-class RNA-targeting approach program targets chronic neuroinflammation and neurodegeneration.
Superoxide dismutase 1 (SOD1) mutations were among the first genetic causes identified in familial amyotrophic lateral sclerosis (ALS) and confer a toxic gain-of-function that drives motor neuron degeneration via protein misfolding, oxidative stress, mitochondrial dysfunction and neuroinflammation.
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder marked by progressive degeneration of upper and lower motor neurons, resulting in paralysis and death typically within 3-5 years of symptom onset. Historically, treatment options have been extremely limited. However, the identification of genetic contributors to ALS pathogenesis has enabled the application of antisense oligonucleotides (ASOs) to selectively modify or reduce the expression of disease-associated genes at the RNA level.
