The biotechnology company Revir Therapeutics has raised $30 million in funding to advance the development of oral genetic therapies for amyotrophic lateral sclerosis (ALS) and other neurodegenerative disorders. This Series A financing was led by the healthcare-focused venture capital firm Lapam Capital. Revir will use the proceeds to…
The investigational therapy BIIB078 was generally well tolerated in a Phase 1 clinical trial, but did not show evidence of slowing disease progression in people with amyotrophic lateral sclerosis (ALS) caused by mutations in the C9orf72Â gene, according to recently published data from the trial. Because the trial failed to…
Treatment with Qalsody (tofersen) substantially slowed disease progression and reduced markers of nerve damage in people with amyotrophic lateral sclerosis (ALS) associated with SOD1 mutations (SOD1-ALS) who took part in an expanded access program in Germany. Patient-reported outcome measures also suggested a favorable perception of Qalsody, with most…
Nearly a year of treatment with TPN-101, an investigational oral molecule from Transposon Therapeutics, safely slowed disease progression and lung function decline in people with amyotrophic lateral sclerosis (ALS) related to C9orf72 mutations. That’s according to final data from a Phase 2a study (NCT04993755) that tested TPN-101…
A computer analysis using real-world data from an amyotrophic lateral sclerosis (ALS) clinic identified four groups of patients with distinct rates of disease progression, a new study by researchers in Portugal reported. The findings continue to demonstrate the differences in ALS presentation and progression seen among people with the…
Treatment with a high dose of ILB, an experimental injection therapy being developed by Tikomed, was well tolerated and appeared to slow the progression of amyotrophic lateral sclerosis (ALS) in a small clinical trial in the U.K. That’s according to data from the exploratory Phase 2 study…
A new genetic mutation that may cause amyotrophic lateral sclerosis (ALS) has been discovered in families living in La Rioja, a region in Spain, according to a study. The gene mutation, found in 10 ALS patients from seven different families, affected the ARPP21 gene, which encodes a protein that…
Io Therapeutics is planning to launch a Phase 2 trial into its investigational oral therapy IRX4204 in people with amyotrophic lateral sclerosis (ALS). The decision follows recent tests showing that the candidate treatment dampened neuroinflammation in an animal model of neuronal autoimmunity, or diseases marked by self-directed…
Kadimastem has partnered with Pluri to produce its investigational cell-based therapy AstroRx for an upcoming Phase 2a clinical trial in amyotrophic lateral sclerosis (ALS). Under the agreement, Pluri will manufacture AstroRx at its 47,000-square-foot cell-therapy production facility under current good manufacturing practices (GMP), a set of…
Repeat infusions of naive immune B-cells were safe and delayed disease onset, reduced disease severity, and extended survival in animal models of amyotrophic lateral sclerosis (ALS), a study found. The approach was also deemed safe in the treatment’s first in-human case study, in which it eased disease severity and…
