Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by failure of motor neurons that lead to paralysis. To date, no treatment exists for ALS that focuses on improving neuromuscular transmission, which would improve quality of life for ALS patients.

Quralis Corp. has launched a program that targets UNC13A mis-splicing, a critical genetic alteration...

Losing the tail to survive. In neurons, the lizard’s strategy, losing the axon to be safe, could prevent cell death. Scientists at Harvard Medical School have observed that certain toxins activated axon loss to prevent damage and survive. This mechanism was mediated by the Gasdermin-E (GSDME) protein, which destroyed the mitochondria in the axons and eliminated the affected nerve projection before the cell died. The inhibition of GSDME prevented the loss of neurons and delayed the progression of amyotrophic lateral sclerosis (ALS) in mice models.

Oryzon Genomics SA has nominated ORY-4001, a selective histone deacetylase 6 (HDAC6) inhibitor, as a clinical development candidate for the treatment of certain neurological diseases, including Charcot-Marie-Tooth (CMT), amyotrophic lateral sclerosis (ALS) and others.

Quralis Inc. raised $88 million in series B round to fund clinical development of its two lead programs in amyotrophic lateral sclerosis (ALS) and to take forward earlier-stage pipeline projects in ALS and frontotemporal dementia.

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.