Neurology

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

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.

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

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.

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.

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.

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.

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.

The postdoctoral scholar at University of California – Irvine discussed further questions he is continuing to investigate.

The 2-part, multicenter BCT-006-US trial will seek to enroll patients who are in the earlier stages of ALS.

Eque-cel was previously evaluated in antibody-mediated idiopathic inflammatory disorders of the nervous system in an investigator-initiated trial.

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