Neurology

Among 29 patients who were treated with nusinersen in the RESPOND trial and evaluable for efficacy, most demonstrated an increase in mean total HINE-2 score from baseline.

In terms of safety, Capricor Therapeutics’ CAP-1002 was reported to be well-tolerated.

The professor at Ohio State University and University of California – San Francisco discussed investigations with different delivery methods of GDNF gene therapy.

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

Review top news and interview highlights from the week ending June 30, 2023.

Taysha also provided updates on its gene therapy program for Rett syndrome.

Benitec Biopharma is planning to initiate a phase 1b/2a clinical trial to evaluate BB-301.

Detailed topline data will be presented at the 2023 International Congress on Parkinson’s Disease and Movement Disorders in August.

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

Review top news and interview highlights from the week ending June 23, 2023.

Delandistrogene moxaparvovec treatment will reportedly cost $3.2 million.

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

uniQure deemed the safety profile for AMT-130 to be manageable.

Data from a phase 1/2 clinical trial of NRTX-1001 were presented at the ISSCR 2023 Annual Meeting.

The advocacy groups are funding a 1-year study to be led by Barry Byrne, MD, PhD, at University of Florida.

Review top news and interview highlights from the week ending June 16, 2023.

CGTLive takes a look at challenges that have beset the first gene therapy coming close to market for treating Duchenne muscular dystrophy in 2023.

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

Review top news and interview highlights from the week ending June 9, 2023.

For International Batten Awareness Day, held annually on June 9, CGTLive reached out to several experts to inquire about the potential impact of gene therapy on the Batten disease treatment landscape.

The FDA’s Advisory Committee meeting is scheduled for September 27, 2023, and the PDUFA date for the company’s BLA review is set for December 8, 2023.

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

The REVEAL trial is being carried out in Canada under a CTA that was cleared by Health Canada in March 2022.

Review top news and interview highlights from the week ending June 2, 2023.

In more positive news, the company’s phase 2 trial for treating DMD will enroll patients in the second half of 2023.