Neurology

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.

Recent long-term data from the phase 2 extension has also demonstrated CAP-1002's efficacy in Duchenne.

Review top news and interview highlights from the week ending July 22, 2022.

Upstaza has been granted marketing authorization by the European Commission.

The principal investigator of the HOPE-2 trial discussed results of the trial in the non-ambulatory population of patients with Duchenne muscular dystrophy.

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 data seen in the phase 1/2 clinical trial of LCTOPC1.

Ultragenyx also exercised its option to acquire GeneTx, its partner in developing GTX-102.

Rajesh Pahwa, MD, FANA, FAAN, discussed balancing hope with current challenges with stem cell approaches in PD.

Review top news and interview highlights from the week ending July 15, 2022.