Neurology

Most participants had improved or stable cognitive function in a proof-of-concept study.

The chief medical officer of Forge Biologics discussed updated data from the REKLAIM clinical trial evaluating FBX-101.

Oncological trials of biologics also face particularly higher rates of clinical holds.

Review top news and interview highlights from the week ending May 17, 2024.

Omer A. Abdul Hamid, MD, pediatric neurologist at Nemours Children’s Health offered advice and discussed important practical considerations with administering gene therapies.

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

The cofounder, chairman, and CEO of Kriya Therapeutics discussed the company’s goal of bringing gene therapy to a much broader population of patients.

The trial is set to dose its second cohort following a positive DSMB recommendation in March.

The Howard Hughes Investigator at the University of Iowa discussed his mouse model research into the pathophysiology of muscular dystrophy and how it relates to gene therapy approaches.

Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.

Updated data including a sixth patient and longer follow-up was presented at ASGCT’s 2024 Meeting.

Review top news and interview highlights from the week ending May 10, 2024.

The gene therapy is up for priority review with a PDUFA date of June 21, 2024.

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

MELPIDA was well-tolerated in patients and nerve conduction was stable or improved after treatment.

The research scientist at Seattle Children's Research Institute discussed mouse model research he presented at ASGCT’s 2024 Meeting.

In response to the death, Pfizer is pausing dosing associated with the crossover portion of the phase 3 CIFFREO trial.

The pediatric neurologist at Nemours Children’s Health offered advice and discussed his experiences delivering pediatric gene therapy.

Review top news and interview highlights from the week ending May 3, 2024.

PJ Brooks, PhD, deputy director, Division of Rare Diseases Research Innovation, NCATS, NIH, discussed ongoing initiatives.

Based on these data, the phase 2 Regenerate PD trial of AB-1005 will begin enrolling later this year.

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

PGN-EDODM1 is being evaluated in the US and Canada in a phase 1 trial.

NKGen anticipates that it will dose the first patient in the study in the second half of this year.

The McCaw Endowed Chair of Muscular Dystrophy at University of Washington gave a background on ASGCT, its founding, and how far the field has come since.