Neurology

The deputy director, Division of Rare Diseases Research Innovation, NCATS, NIH, discussed areas that the agency is targeting.

The scientist from Krembil Research Institute shared takeaways from the session she chaired.

Review top news and interview highlights from the week ending June 14, 2024.

Pfizer additionally noted that the trial missed the mark on key secondary end points.

The pediatric neurologist at Nemours Children’s Health discussed next steps in the field to fully enable the benefits of gene therapy.

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

The lead scientist at Percheron Therapeutics discussed research on antisense oligonucleotide therapies in mouse models of DMD.

AOC 001 previously demonstrated benefit in patients in the phase 2 MARINA trial.

Ineka Whiteman, PhD, head of research and medical affairs, BDSRA Australia, discussed what has occurred in the field of Batten disease gene therapy in the past year.

The phase 1/2 trial is proceeding to its phase 2 trial after safety was demonstrated in its first phase.

The Don and Marilyn Anderson Professor of Oncology at University of Wisconsin – Madison shared highlights from the discussion.

Catch up on any of the key data updates you may have missed last month, with coverage highlights from the CGTLive™ team.

Review top news and interview highlights from the week ending June 7, 2024.

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

The McCaw Endowed Chair of Muscular Dystrophy at University of Washington shared his outlook on the trajectory of research in the field.

Review top news and interview highlights from the week ending May 31, 2024.

Current handling guidelines do not align between the laboratory and clinical settings.

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

An ASGCT poster focused on special ethical considerations of gene therapy human research and how to address them.

The investigator of neurology at Mass General Research Institute discussed research from her grad student, Lisa Nieland, presented at the ASGCT 2024 meeting.

The head of research at Mass General Brigham’s Cell and Gene Therapy Institute discussed research from the center presented at ASGCT 2024.

The chief medical officer of Encoded Therapeutics discussed the company’s strategy with gene therapy development for Dravet syndrome.

The CAR-T product is currently being evaluated in the phase 2b portion of a clinical trial (NCT04146051) for MG.

The associate professor of otolaryngology from Harvard Medical School also shared his excitement for the positive data on AAV1-hOTOF.

Review top news and interview highlights from the week ending May 24, 2024.