Acceptable safety is being seen to date in four adults with late-onset Pompe disease (LOPD) given the one-time gene therapy  AT845 in the FORTIS Phase 1/2 trial, its developer, Astellas Gene Therapies, announced. Interim study data in these four enrolled patients, as of the Dec. 3 cutoff date, shows…
Audentes Therapeutics
An investigational gene therapy called AT845 could effectively increase the activity of an enzyme that’s deficient in mouse and nonhuman primate models of Pompe disease, according to a study. The research also highlights potential complications that can occur when gene therapies designed for use in humans are tested in…
The first patient has been dosed in the FORTISÂ Phase 1/2 trial of AT845, a single-dose gene therapy for adults with late-onset Pompe disease (LOPD). AT845 is designed to deliver a healthy copy of the gene that encodes the enzyme acid alpha-glucosidase (GAA) directly to…
A Phase 1/2 trial is recruiting adults in the U.S. who have late-onset Pompe disease to test a gene therapy, AT845, being developed by Audentes Therapeutics, according to an update from the Muscular Dystrophy Association. The company-sponsored FORTIS trial (NCT04174105) will be evaluating the safety…
A Phase 1/2 clinical trial is recruiting adults with late-onset Pompe disease to test Audentes Therapeutics‘ investigational gene therapy AT845, the company has announced. The trial (NCT04174105) will be conducted at clinical sites in the U.S., Germany, and the U.K. It aims to recruit up to…
Gene therapies — after a tragedy kicked them back to the lab two decades ago — are beginning to come into their own, making possible the progress in gene-targeted and combination treatments for neuromuscular diseases once thought unimaginable. But many challenges remain — from the likelihood of no “one and…
The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have already registered to attend the World Orphan Drug Congress (WODC) USA 2019, set to take place at the Gaylord National Harbor…
Audentes Therapeutics plans to submit an investigational new drug (IND) application for its Pompe disease gene therapy AT845 this year. In a recent update, Audentes, a biotech company, discussed its portfolio of investigational gene therapies for rare diseases. “2018 was an outstanding year for Audentes,” Matthew…