Neurology

Review top news and interview highlights from the week ending August 19, 2022.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The move comes after the EMA recommended an additional clinical trial of sepofarsen before applying for marketing authorization.

BrainStorm Cell Therapeutics has decided to submit a BLA for NurOwn in ALS following an erratum to a phase 3 trial.

Preclinical data showed hARSA activity levels in the brain that are predictive of functional improvements.

Review top news and interview highlights from the week ending August 12, 2022.

The 2 deaths, due to acute liver failure, occurred in patients treated in Kazakhstan and Russia.

AstroRx previously demonstrated clinically meaningful reductions in the rate of disease progression.

PBFT02, developed by Passage Bio, is delivered via intra cisterna magna administration to the brain.

A single patient will be dosed with CRD-THM-001 in an upcoming trial.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

MyoAAV was shown to deliver 25 to 50 times greater gene expression in multiple skeletal muscles compared with natural AAV serotypes.

No such adverse events have occurred in the lower-dose group.

Susan Ruediger, founder and chief mission officer, CMT Research Foundation, discussed the origins of the CMTRF.

Review top news and interview highlights from the week ending August 5, 2022.

The trial is being launched by the University of Florida Center for Regenerative Medicine in collaboration with RESTEM.

The principal investigator of the HOPE-2 trial discussed safety and efficacy of Capricor’s CAP-1002.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The professor of neurosurgery at Rush University Medical School discussed the potential of Lineage Cell’s LCTOPC1 cell therapy.

Seven participants have been dosed with AGT103-T, an autologous T-cell therapy currently being evaluated in a phase 1 trial.

The chief scientific officer and senior vice president of the Parkinson’s Foundation discussed remaining research and education needs to support the future of genetic medicines.

Review top news and interview highlights from the week ending July 29, 2022.

The announcement follows positive data updates from the ENDEAVOR and SRP-9001-101 studies.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Susan Ruediger, founder and chief mission officer, CMT Research Foundation, discussed a new partnership with Nationwide Children’s Hospital.