Neurology

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.

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

The company stated that based on the meeting, the trial’s primary end points will remain the same.

Sponsors who hope to participate will be able to apply from January 2 to March 1, 2024.

Both the second and first patient dosed in the study received the lower of the 2 doses of the gene therapy that Taysha Gene Therapies is evaluating in the phase 1/2 REVEAL trial.

Review top news and interview highlights from the week ending September 29, 2023.

Unforeseen complications may arise when treating older patients with gene therapy.

Deborah Phippard, PhD, the chief scientific officer of Precision for Medicine, discussed the history and current state of gene therapy research for neurological indications.

Atamyo has received $8.6 million in nondilutive financing from France 2030 for ATA-200's development.

Committee members were overwhelmingly against the available data supporting NurOwn as an effective treatment for mild to moderate ALS, with 17 no votes, 1 yes vote, and 1 abstention.

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

The chief scientific officer of Precision for Medicine discussed what lies on the horizon for gene therapies directed at neurological indications, such as Alzheimer disease and Parkinson disease.

Juan Francisco Cabello, MD, the head of the Pediatric Neurology Fellowship Program at the University of Valparaíso in Chile, discussed factors holding back more widespread adoption of newborn screening for indications treatable with gene therapy.

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.

Sebastian Michels, MD, a postdoctoral fellow and member of the La Spada Lab at University of California, Irvine, discussed how his lab’s findings may open the door to developing RNA therapeutics for ALS.

Review top news and interview highlights from the week ending September 22, 2023.

The company is now prioritizing its gene therapy program for Rett syndrome.

The chief scientific officer of Precision for Medicine discussed the previous and current delivery methods used for gene therapies and new innovations on the horizon.

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

The founding president and chief executive officer of Parent Project Muscular Dystrophy discussed recent and upcoming milestones in the field.

Juan Francisco Cabello, MD, the head of the Pediatric Neurology Fellowship Program at the University of Valparaíso in Chile, discussed the current state of newborn screening and what needs to change.