Neurology

Updated data including a sixth patient and longer follow-up was presented at ASGCT’s 2024 Meeting.

Review top news and interview highlights from the week ending May 10, 2024.

The gene therapy is up for priority review with a PDUFA date of June 21, 2024.

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

MELPIDA was well-tolerated in patients and nerve conduction was stable or improved after treatment.

The research scientist at Seattle Children's Research Institute discussed mouse model research he presented at ASGCT’s 2024 Meeting.

In response to the death, Pfizer is pausing dosing associated with the crossover portion of the phase 3 CIFFREO trial.

The pediatric neurologist at Nemours Children’s Health offered advice and discussed his experiences delivering pediatric gene therapy.

Review top news and interview highlights from the week ending May 3, 2024.

PJ Brooks, PhD, deputy director, Division of Rare Diseases Research Innovation, NCATS, NIH, discussed ongoing initiatives.

Based on these data, the phase 2 Regenerate PD trial of AB-1005 will begin enrolling later this year.

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

PGN-EDODM1 is being evaluated in the US and Canada in a phase 1 trial.

NKGen anticipates that it will dose the first patient in the study in the second half of this year.

The McCaw Endowed Chair of Muscular Dystrophy at University of Washington gave a background on ASGCT, its founding, and how far the field has come since.

Another recent program in the investigational landscape was Helixmith’s deprioritized Engensis diabetic neuropathy gene therapy.

The first-in-human, open-label, single-arm trial is expected to enroll 9 participants over the course of a year.

Review top news and interview highlights from the week ending April 26, 2024.

The company expects to open the first clinical site in the DOSED study in the second quarter of 2024.

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 a phase 2 blinded study of ATL1102 being conducted in the Europe and Australia.

At the 90 day timepoint, the patient showed improvements in swallowing compared to their own natural history data, as measured by videofluoroscopic swallowing studies.

Review top news and interview highlights from the week ending April 19, 2024.

The postdoctoral scholar at University of California – Irvine discussed research aiming to link genetic variants and neurodegeneration.

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