Neurology

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.

The research associate at the The Texas Heart Institute discussed preclinical research she presented at AHA’s 2023 Scientific Sessions on MSC-derived exosomes.

The chief research officer of the Muscular Dystrophy Association discussed the need for disease-modifying therapies in other muscular dystrophies and programs the association has announced to this end.

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

The codirector of the MDA Clinic and professor of neurology at Johns Hopkins discussed the importance of continuing to fight for progress with older patients with SMA.

Among 10 patients evaluated compared to their baseline at 1 week after receiving their final dose in the trial, 30% of patients demonstrated clinical improvement on the ADCOMS.

After a negative AdComm meeting and withdrawing its BLA, the company seeks guidance on a phase 3 trial.

Review top news and interview highlights from the week ending November 17, 2023.

The CAR-T previously was cleared for separate trials in systemic lupus erythematosus, myositis, and systemic sclerosis.

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

The therapy is already being evaluated in patients with lupus nephritis and diffuse cutaneous systemic sclerosis.