Neurology

The latest data build on previous research that showed on-target effects analyzed across multiple measurements and a favorable safety profile.

Review top news and interview highlights from the week ending April 1, 2022.

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

Review top news and interview highlights from the week ending March 25, 2022.

The professors and researchers from The University of Texas at Austin discussed new findings of Cas9 structure and mechanisms.

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

The director of the Powell Gene Therapy Center at the University of Florida discussed unmet needs that remain in Pompe disease.

A sister study of AMT-130 in Europe dosed its first patients in February.

The company recently received IND clearance for a solid tumor cell therapy.

Viral vectors have become the most common method for delivering gene therapy; however, pre-existing humoral immunogenicity can potentially render a gene therapy ineffective.

The professor from National Taiwan University Hospital discussed his work in improving diagnoses in Pompe disease and AADC deficiency.

The chief scientific officer and senior vice president of the Parkinson’s Foundation discussed the importance of accessibility in clinical trials.

Review top news and interview highlights from the week ending March 18, 2022.

The draft guidance from the Center for Biologics Evaluation and Research outlines safety considerations throughout the clinical development program timeline.

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

Data presented at MDA 2022 show improvements in motor and pulmonary function and patient-reported outcomes.

Sarepta also presented updated data from Study 101 of SRP-9001 at MDA 2022.

The new designation follows an IND clearance and fast track review in February 2022.

Myrtelle’s gene therapy previously demonstrated initial efficacy in the disease in February 2022.

New data presented at MDA 2022 showcased positive motor and bulbar function data.

The global head of research and development at Atara Biotherapeutics discussed the future of MS research.

Review top news and interview highlights from the week ending March 11, 2022.

The therapy’s IND has been cleared and a first in-human trial is expected to begin later this year.

Patients demonstrated a median reduction of 63.5% in CSF NfL levels 6 months after treatment.

The senior vice president of clinical development at Passage Bio discussed biomarker activity seen in the IMAGINE-1 trial.