Author Archives: Forest Ray PhD

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…

AccuraStem 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…

AB Science will suspend recruitment and treatment in all studies involving masitinib, a tyrosine kinase inhibitor being investigated for amyotrophic lateral sclerosis (ALS) and several other conditions. The voluntary suspension follows the identification of a potential risk of ischemic heart disease — a condition of recurring chest…

A committee with the European Medicines Agency favors designating Seelos Therapeutics‘ investigational therapy SLS-005 (trehalose) an orphan drug as a potential treatment of amyotrophic lateral sclerosis (ALS), the company announced in a press release. The opinion, issued by the Committee for Orphan Medicinal Products (COMP), will now go to the…

Speech and oral therapy were added to covered services available by telemedicine for people with amyotrophic lateral sclerosis (ALS), which Medicare previously recognized only as in-person visits, during the COVID-19 pandemic. Medicare will now reimburse — at least through December 2021 — evaluations and treatments given by telemedicine…

Ionis Pharmaceuticals is opening a Phase 3 safety and efficacy trial of ION363 (jacifusen) in amyotrophic lateral sclerosis (ALS) patients with confirmed mutations in the FUS gene, a known cause of juvenile-onset disease. The trial (NCT04768972) will enroll up to 64 people, ranging in age from 12 to 65,…

Retrotope has dosed the first patients in its recently-initiated Phase 2 clinical trial evaluating RT001 for the treatment of amyotrophic lateral sclerosis (ALS). Enrollment is open across four European sites for patients ages 20–75 whose disease symptoms began less than three years ago and do not need…

Under an agreement with the University of Kansas, PharmaTher has gained exclusive worldwide rights to develop and possibly commercialize ketamine as a therapy for amyotrophic lateral sclerosis (ALS). “We are pleased to have added the [ALS] program to our already impressive development pipeline…

The small difference in treatment responses among amyotrophic lateral sclerosis (ALS) patients receiving NurOwn versus a placebo in a recent clinical trial likely was due to chance, the U.S. Food and Drug Administration (FDA) announced in a press release. Along with slightly more deaths in the NurOwn…

PrimeC is safe and well-tolerated for the treatment of amyotrophic lateral sclerosis (ALS) and showed signs of slowing disease progression in patients, findings from a recently-completed Phase 2a study show. All 15 participants in the NST002 clinical trial (NCT04165850) have chosen to…