News

Partnership Targets Gene-editing Therapies for Familial ALS

CRISPR Therapeutics and Capsida Biotherapeutics have partnered to develop gene-editing therapies for familial amyotrophic lateral sclerosis (ALS) and Friedreich’s ataxia. Capsida specializes in designing virus-based means of delivering therapies to specific cells, while CRISPR’s expertise lies in gene-editing technology, particularly the CRISPR gene-editing system. “Bringing together…

Amylyx Seeks Approval in Canada of Lead Candidate AMX0035

Amylyx Pharmaceuticals is seeking approval, in Canada, of its lead candidate AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS). This regulatory submission “represents a significant milestone in our efforts to develop a new treatment option for people living with ALS who have no time to wait,” Joshua Cohen, co-founder, co-CEO,…

Investigational RNA Molecule May Slow ALS Progression

An investigational RNA molecule called an RNA aptamer halted the death of motor neurons and helped preserve motor function in a mouse model of amyotrophic lateral sclerosis (ALS). Further research is ongoing to confirm the findings in additional ALS models before testing it in humans, “but I’m optimistic that this…

Spinogenix’s SPG302 Oral Therapy Named Orphan Drug by FDA

The U.S. Food and Drug Administration (FDA) has awarded orphan drug designation to Spinogenix’s investigational oral molecule SPG302 for treating amyotrophic lateral sclerosis (ALS). Orphan drug status is granted by the FDA to encourage the development of therapies for conditions that affect fewer than 200,000 people in the…

$1M DOD Grant Supports AS-202 for Sporadic ALS

AcuraStem has been awarded a $1 million grant from the U.S. Department of Defense (DOD) to advance AS-202, a potentially disease-modifying therapy for sporadic amyotrophic lateral sclerosis (ALS), according to a press release. The candidate therapy is designed to increase the number of lysosomes — cell compartments…