Neurology

From baseline, all participants showed improvements on CGI-Improvement scale scores while all but 1 patient demonstrated improvement on modified Hoehn and Yahr scores.

Review top news and interview highlights from the week ending November 19, 2021.

The gene-edited approach hopes to overcome with the shortcomings of autologous hematopoietic stem-cell transplantation.

Following updated safety data, Rocket Pharmaceuticals has decided to discontinue the high-dose cohort.

The phase 1 trial is the first in-human trial to assess a gene therapy for CMT.

The cell therapy is set to be evaluated in a 2-part, phase 1/2 trial.

Dan Oliver, cofounder and chief executive officer, Rejuvenate Bio, discussed the company’s ultimate goal of reversing aging.

The professor of medicine at Duke University School of Medicine previously served as commissioner in 2016.

Review top news and interview highlights from the week ending November 12, 2021.

Primary efficacy data released by Sangamo Therapeutics showed that above normal α-Gal A activity was maintained for up to a year after treatment.

The professor from National Taiwan University Hospital discussed the challenges of working with a rare disease.

The chief executive officer of Rocket Pharmaceuticals, Gaurav Shah, MD, discussed the company’s lead programs and future plans.

Review top news and interview highlights from the week ending November 5, 2021.

4D Molecular Therapeutics announced positive data from trials assessing 2 of their gene therapies.

The professor from National Taiwan University Hospital discussed further research he would like to do with the gene therapy PTC-AADC.

Partners in the Bespoke Gene Therapy Consortium include the NIH, FDA, private companies such as Pfizer and Biogen, and non-profits.

André Choulika, PhD, chief executive officer and cofounder, Cellectis, discussed the company’s expansion into gene therapies.

Review top news and interview highlights from the week ending October 29, 2021.

The professor from National Taiwan University Hospital discussed the mechanism of gene therapy in AADC deficiency.

VM202 was found to be particularly effective in treating neuroischemic ulcers.

bluebird bio is also planning to withdraw the marketing authorization for their β-thalassemia therapy, beti-cel, from the EU and UK.

Review top news and interview highlights from the week ending October 22, 2021.

The professor from National Taiwan University Hospital discussed 3 studies of PTC-AADC for AADC deficiency.

AVROBIO shared interim safety data from ongoing phase 1 and 2 trials.

Tofersen failed to meet its primary end point in change from baseline in ALSFRS-R but differences in total cerebrospinal fluid SOD1 protein and neurofilament light chain were observed.