Centyrin-siRNA Conjugates for the Treatment of Pompe Disease in a new Clinical Trial
Learn about a Phase 1b clinical trial for ABX1100, a substrate reduction therapy (SRT), which aims to slow disease progression.
Aro Biotherapeutics starts Phase 1b Clinical Trial for Substrate Reduction Therapy ABX1100
Aro Biotherapeutics has launched a Phase 1b clinical trial for ABX1100, a substrate reduction therapy (SRT).
Maze Therapeutics Starting Phase I Clinical Trial with MZE001, A Substrate Reduction Therapy
Recently, preclinical data were presented at the 18th Annual WORLDSymposium in San Diego, CA, supporting MZE001 as a treatment for Pompe disease.
Amicus Remains Hopeful about AT-GAA Despite Mixed Results from Phase 3 Study
Amicus Therapeutics recently announced topline results of its phase 3 PROPEL study of its combination therapy, AT-GAA, for the treatment of late onset Pompe disease (LOPD).
Cheryl Wong Po Foo
Cheryl Wong Po Foo, PhD, Director of Clinical Development, Astellas Gene Therapies, responsible for the advancement of the clinical development program for Pompe disease.
Spark Therapeutics Has Begun a Phase 1/2 Gene Therapy Study for Late-Onset Pompe Disease
The Spark Therapeutics phase 1/2 gene therapy trial testing SPK-3006 to treat late onset Pompe disease has officially started with the first person being given their first (and theoretically only) dose.
Amicus Therapeutics Has Begun the FDA Submission for Their Pompe Drug, AT-GAA
Amicus Therapeutics has announced they have begun a rolling Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for their drug combination, AT-GAA, to treat late onset Pompe disease (LOPD).
New Review Article Highlights Latest Developments in Pompe Disease Research
An excellent review article focused on the latest developments in Pompe disease research was recently published by Naresh K. Meena and Nina Raben from the NIH.
New ERT For Late-onset Pompe Disease Shows Promise
Results from the COMET study were recently reported showing neoGAA (avalglucosidase alfa), the newer enzyme replacement therapy (ERT) currently in development, to be as effective as the currently approved ERT, Myozyme (alglucosidase alfa), in persons with late-onset Pompe disease.
Sanofi Genzyme COVID-19 Update
At this time, Sanofi Genzyme does not anticipate shortages for patients resulting from the COVID-19 situation.