Neurology

The chief medical advisor of the Muscular Dystrophy Association discussed research he is working on with the goal of increasing eligibility for gene therapies and opening the door to redosing.

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

The chief medical advisor of the Muscular Dystrophy Association discussed what seasoned clinicians and newcomers to the field alike can look forward to at the 2024 MDA Meeting.

Aruna Bio stated that it expects to begin a phase 1b/2a clinical trial within the first half of this year.

Sharon Hesterlee, PhD, the chief research officer of MDA, discussed highlights of 2023’s meeting and exciting new things to look forward to at the upcoming 2024 meeting.

PepGen’s ongoing multiple ascending dose trial is expected to read out initial data in mid-2024.

The president and head of R&D at Taysha Gene Therapies discussed his expectation that younger patients my see even better results with the company’s gene therapy than the adult patients treated so far.

Review top news and interview highlights from the week ending January 19, 2024.

The cofounder and CEO of Neurona Therapeutics discussed the company’s goals to evaluate the cell therapy in more types of epilepsy and potentially even in Alzheimer disease.

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

Among 3 patients in the study’s first cohort, a 3.6 to 6.6-fold increase in CSF PGRN over baseline was observed at 30 days posttreatment.

The cofounder and CEO of Neurona Therapeutics discussed results from a phase 1/2 clinical trial presented at AES’s annual meeting.

The trial is recruiting 5 more participants for the highest dose cohort following a positive DSMB review.

The president and head of R&D at Taysha Gene Therapies discussed positive early results from the first 2 patients treated with gene therapy TSHA-102.

The first pediatric patient was treated in the United States, but the company has now also received clearance to treat pediatric patients in the UK.

Review top news and interview highlights from the week ending January 12, 2024.

Atara plans to submit a BLA for tab-cel, approved in Europe, in the second quarter of 2024.

Among 11 patients with PD who were treated in the study, there no serious adverse events (AEs) attributed to AB-1005.

The cofounder and CEO of Neurona Therapeutics discussed NRTX-1001, the company’s allogeneic regenerative neural cell therapy.

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

In terms of safety, Descartes-08 was characterized as well-tolerated during the study and long-term follow-up period.

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

The clearance continues a recent trend of IND clearances for CAR-T therapies in autoimmune disease.

The chief research officer of the Muscular Dystrophy Association also went over the highlights from last year’s meeting.

The AAV-SLB101 capsid used in SGT-003 may yield increased transgene expression and improved safety compared with first-generation AAV gene therapies.

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

CGTLive spoke with Donald Wood, PhD, president and CEO of the MDA while looking ahead to its March conference.

These, among many others, are some of the key clinical trials of gene, cell, and regenerative therapies that the CGTLive staff will be following throughout 2024.

Take a look at the stories that stood out as pillars of progress and success in endocrinology gene and cell therapy development in 2023.

This year we reached out to numerous experts outside the context of the conference cycle to seek their insight on company announcements, pipeline therapies, and disease awareness days.