Neurology

Alexandra Collin de l’Hortet, PhD, the head of therapeutics at Epic Bio, discussed EPI-321, an investigational treatment for facioscapulohumeral muscular dystrophy.

Review top news and interview highlights from the week ending August 16, 2024.

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

A patient with MG is also the first evaluated to reach 1 year of follow-up and has a continued durable immunomodulator-free response.

The president and chief executive officer of Passage Bio discussed feedback from a recent Type C meeting with the FDA.

The head of therapeutics at Epic Bio discussed EPI-321, an investigational treatment for facioscapulohumeral muscular dystrophy.

Review top news and interview highlights from the week ending August 9, 2024.

Catch up on any of the key FDA news stories you may have missed last month, with coverage highlights from the CGTLive® team.

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

The cofounder and chief science officer of Longeveron discussed working to show potential benefits in a follow-up phase 3 trial.

The trial evaluating the safety and tolerability of IDP-023 will be led by Stanford and UCSF.

Paul Y. Song, MD, the chairman and chief executive officer of NKGen, discussed discussed the new clinical study design for evaluating cell therapy SNK01 in PD.

The president and chief executive officer of Passage Bio discussed feedback from a recent Type C meeting with the FDA.

The company has filed a protocol amendment with the EMA and anticipates resuming recruitment imminently.

Review top news and interview highlights from the week ending August 2, 2024.

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

Paul Y. Song, MD, the chairman and chief executive officer of NKGen, discussed the mechanism behind the company’s NK cell therapy SNK01 and promising early results in patients with PD.

The chief medical officer and head of translational research at Ring Therapeutics discussed research presented at ASGCT 2024.

The program’s cancellation follows a previous announcement that its phase 3 trial had missed its primary end point.

Ractigen is also evaluating saRNA therapies for ALS and SMA.

The cofounder and chief science officer of Longeveron discussed updated data from the phase 2a CLEAR MIND study.

There were no serious adverse events related to the cell therapy and no evidence of ARIA.

Review top news and interview highlights from the week ending July 26, 2024.

The clearance is based on the agency’s acceptance of clinical trial protocols and a chemistry, manufacturing, and controls clinical development plan for the program.

IASO also received IND clearance for a trial in myasthenia gravis in April 2024.

The Don and Marilyn Anderson Professor of Oncology at University of Wisconsin – Madison discussed the state of cell and gene therapy development.

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

Both bluebird bio and Vertex Pharmaceuticals have received unfavorable opinions from the Office of the Inspector General on the matter.

The cofounder and chief scientific officer at Encoded Therapeutics shared preclinical research with the company’s AAV and miRNA platforms.

In honor of World Brain Day, observed annually on July 22 by patient and clinician communities, CGTLive is taking a closer look at this ongoing study.