An advisory committee of the European Medicines Agency (EMA) has recommended that Biogen’s Qalsody (tofersen) be approved in the European Union under exceptional circumstances to treat adults with amyotrophic lateral sclerosis (ALS) associated with SOD1 gene mutations. The positive opinion from the Committee for Medicinal Products for Human Use,…
ProJenX has been given the green light to expand its ongoing Phase 1 clinical trial of prosetin for amyotrophic lateral sclerosis (ALS) across the Atlantic Ocean to testing sites in Europe. The PRO-101 trial (NCT05279755), launched in 2022, is a three-part study evaluating prosetin’s safety, tolerability, and pharmacological…
TPN-101, an investigational oral molecule developed by Transposon Therapeutics, reduced multiple biomarkers of neuroinflammation and neurodegeneration, and slowed respiratory decline in some people with amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD). That’s according to an interim analysis of a Phase 2a clinical trial (NCT04993755) that’s testing…
Myrobalan Therapeutics will use a $400,000 grant from the ALS Association — along with $24 million in new financing — to help advance the development of its oral CSF1 receptor (CSF-1R) inhibitor program, intended to reduce neuroinflammation and promote nerve repair in people with amyotrophic lateral sclerosis…
A research team at Ball State University has received a $20,000 donation from the Todd Siebert Memorial Foundation supporting work into better understanding and treating amyotrophic lateral sclerosis (ALS) caused by C9orf72 mutations. Awarded to Philip Smaldino, PhD, a cell biology professor at the Indiana school, and…
Enrollment is complete in a Phase 2 trial testing InFlectis BioScience’s oral candidate IFB-088 (icerguastat) in people with amyotrophic lateral sclerosis (ALS), the company has announced. The trial (NCT05508074) enrolled a total of 50 adults with bulbar-onset ALS, a form of the disease that first affects the…
An early-stage clinical trial evaluating CK0803, Cellenkos‘ regulatory T-cell-based therapy for people with amyotrophic lateral sclerosis (ALS), has completed dosing its first patient group, the company announced in a press release. The six patients, all adults, were treated at the Columbia University Irving Medical Center, in New…
Verge Genomics has launched a proof-of-concept trial to test whether VRG50635, its oral PIKfyve inhibitor therapy, can be used as a potential treatment for people with amyotrophic lateral sclerosis (ALS). The Phase 1b study will assess the safety and tolerability of escalating doses of VRG50635 — which works…
After two meetings with the U.S. Food and Drug Administration (FDA), Coya Therapeutics says it’s received constructive feedback — and has reached an alignment with the agency regarding development plans for COYA 302, its therapy candidate for people with amyotrophic lateral sclerosis (ALS). The company held the two…
Arbor Biotechnologies and 4DMT — two U.S. companies working in next-generation genetic medicines — have established a strategic partnership to develop and commercialize novel gene therapies for neurological diseases, including amyotrophic lateral sclerosis (ALS). The agreement includes the codevelopment of up to six product candidates for diseases…
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