The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Apic Bio‘s APB-102, an investigational gene therapy aiming to treat familial amyotrophic lateral sclerosis (ALS) associated with mutations in the superoxide dismutase 1 (SOD1) gene. “This orphan drug designation represents an important…

The enzyme superoxide dismutase 1 (SOD1) forms clumps inside the cells of some people with sporadic amyotrophic lateral sclerosis (ALS), making them more vulnerable to DNA damage — which may contribute to the neurodegeneration that marks the disease, an early study suggests. In other patients, SOD1 is transported…

NPT520-34, an investigational small molecule for the treatment of Parkinson’s disease and amyotrophic lateral sclerosis (ALS), is being tested on healthy individuals in a new Phase 1 clinical trial, its developer Neuropore Therapies has announced. Brain inflammation is a common feature among many neurodegenerative disorders, including Parkinson’s,…

A human-derived antibody that specifically binds to abnormal superoxide dismutase 1 (SOD1) improved the symptoms and delayed disease progression in a mouse model for amyotrophic lateral sclerosis (ALS), a study shows. The antibody, alpha-miSOD1, could be further developed as a candidate treatment for ALS caused by SOD1 misfolding. The study titled…

An experimental gene therapy based on RNA interference (RNAi) shows potential to treat patients with familial amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene, according to results of a preclinical study in nonhuman primates. The study, “Safe and effective superoxide…

A gene therapy effectively and safely lowered the production of a key amyotrophic lateral sclerosis (ALS) protein called superoxide dismutase 1 (SOD1) in primates, according to a new study. “This level of silencing, coupled with the lack of adverse effects, suggests that this approach to treating ALS should be…

Voyager Therapeutics’ experimental therapy VY-SOD102 shows promise for treating patients with familial amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene, according to results in animal models. The preclinical data were presented at the Congress of the European Society of Gene and Cell Therapy…

Specific patterns of a misfolded protein — superoxide dismutase 1 (SOD1) — are found in the brain and spinal cord of patients with sporadic amyotrophic lateral sclerosis (ALS), according to a new study, supporting research suggesting that misfolded SOD1 plays a role in this ALS subtype. The research, “…

Voyager Therapeutics presented new data from a study in an animal model of  its clinical candidate VY-SOD101, showing the therapy is effective as it targets the most common cause of familial amyotrophic lateral sclerosis (ALS). The presentation, along with another preclinical program data presentation on a Huntington’s disease therapy…

Two French researchers, Martine Barkats and Maria-Grazia Biferi, are winners of the Avi Kremer ALS Treatment Prize, worth $1 million, for developing a gene therapy approach that shows promise in treating certain types of amyotrophic lateral sclerosis (ALS), Prize4Life announced in a recent press release. The approach targets one of the…