This is a single-center, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of CRG003 injection in participants with late onset Pompe disease (LOPD), with a long-term follow-up period of 5 years.
CRG003 (BBM-G102) injection is an adeno-associated virus (AAV) gene therapy product for treating Pompe disease to stably express active GAA enzyme in the liver on a long-term basis after the injection.
Participants voluntarily sign informed consent form;
Clinically diagnosed with LOPD;
Males or females aged ≥ 18 years;
Undergone enzyme replacement treatment (ERT) with recombinant human acid alpha-glucosidase (rhGAA) previously, and has been discontinued for at least four weeks before screening;
Severe cardiomyopathy was defined as left ventricular ejection fraction (LVEF) < 45% or New York Heart Association (NYHA) functional class 3 or above;
Require invasive mechanical ventilation, or rely on noninvasive ventilation during the day;
Intolerance to ERT, prior experience of serious infusion-associated reactions (IARs), prior experience of serious allergic reactions or investigator-assessed intolerance to ERT;
Have received any systemic immunosuppressants (except inhalation or topical use) other than glucocorticoids or investigator-recommended immunosuppressants 30 days prior to screening, and known intolerance to immunosuppressants such as glucocorticoids;