Neurology

AMT-130 has shown some evidence of dose-dependent clinical benefits in treated study participants.

For the 3 patients who were treated at the higher dose, immunohistochemistry showed that a mean of 54% of muscle fibers were expressing microdystrophin.

The chief medical officer of Encoded Therapeutics overviews the company’s timeline and strategy for its gene regulation program for Dravet syndrome.

NRTX-1001 consists of human interneurons that provide long-term secretion of gamma-aminobutyric acid, an inhibitory neurotransmitter, which is expected to repair neural networks.

Review top news and interview highlights from the week ending June 21, 2024.

The company anticipates that it will be able to submit a BLA in late 2024 or early 2025.

KYV-101 racks up another potential indication in addition to its primary autoimmune investigations.

The gene therapy is now indicated for ambulatory patients aged 4 years and older, and has been granted accelerated approval for nonambulatory patients.

The clinical professor of neurology and pediatrics at Keck School of Medicine of USC discussed her learning experience administering gene therapies for the first time.

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

The case study was recently published in the Proceedings of the National Academy of Sciences.

Treated participants have achieved some mild improvements on MDS-UPDRS Part III at 18 months posttreatment.

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.