Tofersen’s development is progressing in fits and starts. That was evident at the U.S. FDA’s March 22 meeting of the Peripheral and Central Nervous System Drugs Advisory Committee, which unanimously agreed Biogen Inc.’s failed phase III study predicted a clinical benefit in treating amyotophic lateral sclerosis (ALS) that includes the rare superoxide dismutase 1 component.

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.

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.