Neurology

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.

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

AviadoBio announced the initiation of the phase 1/2 ASPIRE-FTD clinical trial earlier in October.

Neurona Therapeutics also reported that this first patient cohort has completed enrollment in addition to the early efficacy data from the first 2 patients treated.

Review top news and interview highlights from the week ending October 20, 2023.

The company will meet with the FDA again to discuss the future of the investigational cell therapy.

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

Among 15 tested patients, who received at least 2 IT injections of the MSC therapy, improvements of between 5% and 18% in 25 feet walking were observed.

The FDA previously placed the trial on clinical hold for undisclosed reasons.

Review top news and interview highlights from the week ending October 13, 2023.

The chief research officer of the Muscular Dystrophy Association discussed research needs that remain in the field in light of recent progress and approvals.

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

The open-label, multicenter, ascending dose trial is expected to treat approximately 9 patients in total across 2 dose cohorts.

Deborah Phippard, PhD, the chief scientific officer of Precision for Medicine, discussed what may be next for the field of gene therapy after early successes in single-mutation disorders.

Deborah Phippard, PhD, the chief scientific officer of Precision for Medicine, discussed the unique aspects of clinical trials for gene therapies that need to be considered before initiation.

Review top news and interview highlights from the week ending October 6, 2023.

The AFFINITY trial is set to dose patients at dose level 2 by the end of 2023.

The move was carried out in accordance with a recommendation for dose-escalation made by the trial’s independent Data Safety Monitoring Committee.

The codirector of the MDA Clinic and professor of neurology at Johns Hopkins discussed the updated analyses of the NURTURE study that affected the big picture of the data.