Neurology

Ralph Laufer, PhD, chief scientific officer, Lysogene, discussed LYS-SAF302, its mechanism of action, trial data, and further research.

The professor from National Taiwan University Hospital discussed gene therapy studies in AADC deficiency, Parkinson disease, SMA, and Pompe disease.

Review top news and interview highlights from the week ending December 3, 2021.

The director of the Mellen Center for MS Treatment and Research at Cleveland Clinic discussed upcoming new therapies for relapsing MS.

ALSFRS-R and FVC values were stable 3 months after treatment but significantly decreased 6 months after treatment, warranting further investigation.

Dan Oliver, cofounder and chief executive officer, Rejuvenate Bio, discussed the advantages of developing gene therapies for both dogs and humans.

Review top news and interview highlights from the week ending November 26, 2021.

The chief executive and scientific officers of Lysogene discussed Lysogene’s patient-focused therapy development.

The chief executive officer of Rocket Pharmaceuticals, Gaurav Shah, MD, discussed the company’s ex-vivo and in-vivo gene therapy platforms.

The professor from National Taiwan University Hospital discussed the benefits of delivering gene therapy directly to the putamen in AADC deficiency.

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.