Neurology

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 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.

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

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

uniQure plans to initiate regulatory discussions in the US and Europe in the first quarter of 2024.

The company characterized the meeting as “productive”.

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

Throughout 2023, interest in the application of CAR-T and other cell therapies to B-cell-driven autoimmune disease skyrocketed, with a multitude of clinical trials being initiated by various companies.

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

The president and head of R&D at Taysha Gene Therapies discussed the early clinical evaluation of TSHA-102.

Benetic anticipates an announcement of interim results sometime around the midpoint of 2024.

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

For This Year in Medicine, CGTLive looks back at a landmark first approval, the new treatment opportunities it has provided, and the unmet needs in the field it has brought to light.

Health Canada has greenlit a protocol amendment for Taysha Gene Therapies’ trial to include patients aged 12 years and older, rather than only adult patients.

uniQure’s AAV9-vector therapy carrying 2 small interfering RNAs targeting GRIK2 was well-tolerated, with a hopeful risk-benefit ratio. A phase 1/2 is set to begin recruitment in late 2023.

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