Neurology

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.

AVB-101 was simultaneously granted fast track designation by the agency. The company plans initiate US activities in 2024.

The founding president and chief executive officer of Parent Project Muscular Dystrophy discussed unmet needs that remain, chiefly concerning widening the criteria for therapies and trials.

Review top news and interview highlights from the week ending November 10, 2023.

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

The chief scientific officer of CureDuchenne discussed research he is looking forward to seeing in 2024.

UoM was previously collaborating on the development of AVR-RD-05 with AVROBIO, but regained the license to the HSC gene therapy following the company’s cessation of development on all programs.

Review top news and interview highlights from the week ending Novmber 3, 2023.

Hearing function was found to be normal for 4 of the patients (50%) at their most recent follow-up.

The DSMB has cleared Atamyo to go forward with a planned dose of ATA-100 for the second cohort that is 3 times higher than the dose used for the first cohort.

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

Despite the announcement, CEO Doug Ingram shared that the company is seeking a wider approval for delandistrogene moxeparvovec-rokl regardless of age or ambulatory status.

Review top news and interview highlights from the week ending October 27, 2023.