Neurology

Various improvements were seen in 2 adult patients and 2 pediatric patients treated in separate clinical trials.

Review top news and interview highlights from the week ending June 28, 2024.

The cofounder and chief executive officer of Kate Therapeutics discussed research that awarded him an Outstanding New Investigator at the ASGCT 2024 meeting.

The company has begun enrolling participants in a new cohort for younger patients in the phase 1/2 AFFINITY DUCHENNE clinical trial.

Three participants with ATTR amyloidosis have received follow-on doses of NTLA-2001 with no serious complications for up to 3 years of follow-up.

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 unique difficulties of designing clinical trials for PD gene therapy candidates.

One patient is considered disease-free at 1 year posttreatment, but another patient’s disease relapsed at 5 months.

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.