- |||||||||| Moss-aGal (recombinant human alpha-galactosidase A) / Greenovation Biotech
Preclinical, Journal: Comparison of efficacy between subcutaneous and intravenous application of moss-aGal in the mouse model of Fabry disease. (Pubmed Central) - Nov 6, 2023
Here, we tested the efficacy of subcutaneous administration of moss-aGal and compared it with the results observed following iv infusion in Fabry mice. The obtained findings demonstrate that subcutaneously applied moss-aGal is correctly transported to target organs and efficacious in degrading Gb3 deposits there and thus suggest the possibility of using this route of administration for therapy of Fabry disease.
- |||||||||| Review, Journal: Developments in the treatment of Fabry disease. (Pubmed Central) - Oct 7, 2021
Chaperone therapy (Migalastat; 1-deoxygalactonojirimycin) is the only other currently approved therapy for Fabry disease...Treatments currently under evaluation in (pre)clinical trials are second generation enzyme replacement therapies (Pegunigalsidase-alfa, Moss-aGal), substrate reduction therapies (Venglustat and Lucerastat), mRNA- and gene-based therapy. This review summarizes the knowledge on currently available and potential future options for the treatment of Fabry disease.
- |||||||||| Myozyme (alglucosidase alfa) / Sanofi
Preclinical, Journal: Uptake of moss-derived human recombinant GAA in Gaa mice. (Pubmed Central) - May 13, 2021
We investigated general effects as well as the uptake into different organs following short-term treatment. Our results do confirm that moss-GAA reaches the target disease organs and thus might have the potential to be an alternative or complementary ERT to the existing one.
- |||||||||| Moss-GAA / Greenovation Biotech, Myozyme (alglucosidase alfa) / Sanofi
Journal: Moss-Derived Human Recombinant GAA Provides an Optimized Enzyme Uptake in Differentiated Human Muscle Cells of Pompe Disease. (Pubmed Central) - Jan 7, 2021
Moreover, incubation of immortalized muscle cells of Gaa mice with moss-GAA GnGn led to similarly efficient clearance of accumulated glycogen as with alglucosidase alfa. These initial data suggest that M6P-residues might not always be necessary for the cellular uptake in enzyme replacement therapy (ERT) and indicate the potential of moss-GAA GnGn as novel alternative drug for targeting skeletal muscle in Pompe patients.
- |||||||||| Moss-aGal (recombinant human alpha-galactosidase A) / Greenovation Biotech
Trial completion: Pharmacokinetics, Pharmacodynamics, and Safety of Moss-aGalactosidase A in Patients With Fabry Disease (clinicaltrials.gov) - Dec 13, 2017
P1, N=6, Completed,
These initial data suggest that M6P-residues might not always be necessary for the cellular uptake in enzyme replacement therapy (ERT) and indicate the potential of moss-GAA GnGn as novel alternative drug for targeting skeletal muscle in Pompe patients. Recruiting --> Completed
|
