Lysosomal Disorders

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.

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 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.

The phase 1 study of NTLA-2001 previously showed positive interim data, including serum TTR reductions, which were reported in June 2021.

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

The FDA cleared the IND application for HMI-203 in October 2021.

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

Dabocemagene autoficel has previously received orphan drug, rare pediatric disease, fast track, and regenerative medicine advanced therapy designations.

Homology is also developing a gene therapy, HMI-102, being evaluated in the phase 1/2 pheNIX trial.

The phase 1/2 SUNRISE trial is now enrolling patients as young as 6 months old with methylmalonic acidemia after positive review under a DSMB.

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

Rocket Pharmaceuticals announced positive data on RP-L201 in leukocyte adhesion deficiency-I, as well as RP-A501 for Danon disease.

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

The director of the Powell Gene Therapy Center at the University of Florida discussed improving access to gene therapies for patients with rare diseases worldwide.

Review top news and interview highlights from the week ending October 15, 2021.

New human gene editing therapies, drug discoveries, targets, and CRISPR technology holds the potential to usher in a new age in medicine.

The chair of the International Collaborative Gaucher Group Registry discussed challenges in Gaucher research.

Review top news and interview highlights from the week ending October 8, 2021.

The director of the Powell Gene Therapy Center at the University of Florida discussed adverse events in gene therapies.