The first healthy volunteers have been dosed in a Phase 1 clinical trial evaluating MZE001, Maze Therapeutics’ experimental oral therapy for Pompe disease. “The initiation of this study is a significant milestone for both Maze and the Pompe community as we advance into the clinic with a potentially…
Experimental treatment
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…
Maze Therapeutics has announced its plans to begin clinical testing of MZE001, its lead candidate therapy for Pompe disease, in the first half of this year. The California-based precision medicine company raised $190 million in financing — led by Matrix Capital Management — to support the development of…
A new enzyme replacement therapy (ERT) targeting specific cell types boosts the delivery of acid alpha-glucosidase (GAA) — the enzyme missing or defective in Pompe disease patients — to muscle and heart cells, a study shows. Compared to standard ERT, the targeted approach by Regeneron Pharmaceuticals normalized the…
AT-GAA, an investigational therapy by Amicus Therapeutics for late-onset Pompe disease, will be available to select patients in the U.K. through an early access program before its potential regulatory approval. Eligible patients are adults who have received the enzyme replacement therapy (ERT) alglucosidase alfa — the…
Administration of 3,4-diaminopyridine phosphate (3,4-DAPP) eased problems at the neuromuscular junction (NMJ) — sites of nerve-muscle communication — in a zebrafish model of Pompe disease. Results suggest that the medication, also known as amifampridine and approved under the brand names …
Maze Therapeutics announced its first three lead therapeutic candidates, including an investigational oral medication designed to treat Pompe disease. Pompe disease is caused by genetic mutations that make the body unable to break down glycogen, a complex sugar molecule. As a result, glycogen builds to toxic levels in…
Miglustat — a component of AT-GAA, an investigational therapy for late-onset Pompe disease (PD) — enhances the activity of the other component in AT-GAA, cipaglucosidase alfa, researchers report. These findings were presented at the 2021 MDA Virtual Clinical & Scientific Conference, in the poster “Enhancing Delivery…
Treatment with AT-GAA, an investigational therapy for late-onset Pompe disease, led to improvements in measures of physical and lung function in the Phase 3 clinical trial PROPEL, top-line data show. “Data from the PROPEL study demonstrate the potential to further improve motor and respiratory functions in patients with Pompe…
Spark Therapeutics has dosed the first patient in the Phase 1/2 RESOLUTE clinical trial of SPK-3006, an experimental gene therapy for people with late-onset Pompe disease (LOPD). “Dosing the first participant in the Phase 1/2 RESOLUTE trial of investigational SPK-3006 for late-onset Pompe disease is an important milestone and…
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