Neurology

The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed challenges with clinical trials in neurocognitive diseases including MPS2.

In terms of safety, Taysha stated that TSHA-102 was well-tolerated and that at 6 weeks after dosing no treatment-emergent serious adverse events had occurred.

Aspen Neuroscience noted that the study will be the US’s first multicenter phase 1/2a clinical trial for an autologous iPSC-derived therapy.

Review top news and interview highlights from the week ending August 11, 2023.

LION-CS101, the study evaluating gene therapy AB-1003, is currently recruiting patients aged 18 years through 65 years with LGMD2I/R9 who have a confirmed mutation in FKRP.

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

The initiation of dosing the second cohort in Ultragenyx’s study was recommended by the Data Safety Monitoring Board after a review of safety results from the trial’s first cohort.

Taysha Gene Therapies previously reported the dosing of the first patient with TSHA-102 in June 2023.

Nicole Paulk, PhD, the CEO, founder, and president of Siren Biotechnology, discussed research she presented at ASGCT’s 2023 conference.

Review top news and interview highlights from the week ending July 28, 2023.

Maria Escolar, MD, the chief medical officer of Forge Biologics, discussed the atypical approach being used in the company’s trial for AAV gene therapy FBX-101.

With up to 20% of incidences of FTD driven by genetic mutations, multiple gene therapy companies have initiated clinical trials of AAV-mediated treatments for a patient population with little to no options.

The data suggest that for patients with SMA with 2 and 3 copies of the SMN2 gene, the treatment is safe and effective, indicating the success of earlier, presymptomatic treatment.

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

The codirector of the MDA Clinic and professor of neurology at Johns Hopkins discussed updated data from the NURTURE study.

The chief scientific officer of LocanaBio discussed preclinical research presented at ASGCT 2023.

The chief medical officer of Forge Biologics discussed updated data from the phase 1/2 RESKUE clinical trial that she presented at ASGCT’s 2023 conference.

Review top news and interview highlights from the week ending July 21, 2023.

Gary Owens, MS, the associate director for gene therapy discovery at Precision Biosciences, discussed data he presented at ASGCT’s 2023 conference.

The chief scientific officer at REGENXBIO discussed RGX-202 and the ongoing clinical trial.

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

The professor of neurosurgery at Rush University Medical School discussed challenges with trials for SCI and the 1st Annual SCIIS.

The pediatric gastroenterologist and professor in residence at University of California at San Francisco discussed biomarker findings from the phase 1/2 CAMPSIITE trial.

The associate director for gene therapy discovery at Precision Biosciences discussed preclinical research he presented at ASGCT’s 2023 conference.

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

The guidance outlines recommendations for managing and reporting CGT manufacturing changes and PMR noncompliance.

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

The senior investigator at the National Cancer Institute Center for Cancer Research discussed unmet needs that remain and further research to be done.

Wave Life Sciences is discontinuing WVE-004.

Reductions in NfL were also associated with changes on ALSFRS-R scores.