Neurology

There were no dose-limiting toxicities, serious adverse events, nor infusion-related reactions among 6 patients treated with ENCell’s investigational EN001 therapy.

Review top news and interview highlights from the week ending May 26, 2023.

There were no serious adverse events reported and no patients discontinued from the study.

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

The CEO, founder, and president of Siren Biotechnology discussed the preclinical data she presented at ASGCT’s 2023 meeting on the use of AAV immuno-gene therapy for high-grade gliomas.

The investigational gene therapy, also known as SRP-9001, was recently backed by the agency’s advisory committee in a tight decision. Its new deadline for review is June 22, 2023.

The trial compared data from external controls including a recent domagrozumab null study and a propensity score weighted comparison.

The chief scientific officer of Sangamo Therapeutics discussed the company’s AAV capsid evolution research.

The findings, presented at ASGCT’s 2023 conference, included an analysis of biomarker data in relation to disease severity.

The neurologist at Massachusetts General Hospital discussed BBP-812, an investigational AAV vector-based gene therapy being evaluated for Canavan disease in a phase 1/2 trial.

The most robust transduction in nonhuman primates was seen with ICV delivery of pooled PM4.AAV.mRFP and AAV2.mNG gene therapy,

Review top news and interview highlights from the week ending May 12, 2023.

The chief scientific officer of Sangamo Therapeutics discussed data from 2 of the company’s programs that were presented at ASGCT’s 2023 conference.

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

The professor at Ohio State University and University of California – San Francisco discussed updated data from a phase 1 study of GDNF gene therapy in patients with PD.

Review top news and interview highlights from the week ending May 12, 2023.

Bloomsbury Genetic Therapies’ BGT-DTDS, a neuron-targeted and AAV2-mediated therapy, previously received orphan drug designation from the FDA and EC.

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

Biogen and Novartis terminated collaborations with the company that first initiated in 2020.

The cell therapy field applications staff scientist at ThermoFisher discussed advantages of the company’s DynaCellect system.

The single-administration allogeneic cell therapy has shown positive safety—and signs of efficacy—in preliminary data from the first 2 patients dosed with the Neurona Therapeutics’ product.

CK0803 is being evaluated for safety and tolerability in the REGALS study.

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

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

The investigators from Children’s Hospital of Philadelphia discussed the follow-up studies they are conducting in light of their recent findings regarding DNA virus replication.

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

The investigators from Children’s Hospital of Philadelphia discussed the potential implications of their research into human adenoviruses for the development of new gene delivery tools.

The professor for human genetics and neurology at the University of Miami Miller School of Medicine discussed the rapidly changing landscape of diagnosing and treating genetic disorders.

In observance of International Pompe Day, held annually on April 15, catch up on the past year’s news and expert insights related to gene therapies in development for this rare disease.

Review top news and interview highlights from the week ending April 14, 2023.