Neurology

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.

An interim analysis of a phase 1/2 study suggest the REGENXBIO therapy also showed efficacy signals.

The McCaw Endowed Chair of Muscular Dystrophy at University of Washington discussed highlights from the meeting.

The director of the Manton Center for Orphan Disease Research at Boston Children’s Hospital discussed takeaways from an MDA session on rare diseases.

The deputy director, Division of Rare Diseases Research Innovation, NCATS, NIH, discussed the NIH’s genome editing program and more.

Review top news and interview highlights from the week ending April 12, 2024.

The Lichtenstein professor of neurology at University of Miami Miller School of Medicine discussed research his lab is pursuing and its applications.

The data comes from 2 phase 1/2a clinical trials and their respective open-label extension studies.

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