Pompe Disease Manufacturers, Suppliers & Companies
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Manufacturerbased in Cambridge, MASSACHUSETTS (USA)
At AVROBIO, we aim to halt or reverse disease with a single dose of gene therapy. ONE COMPANY with a vision to bring personalized gene therapy to the world. Our goal is simple and powerful: We aim to enable individuals with genetic disease to live ...
AVROBIO - Model AVR-RD-03 - Gene Therapy for Pompe Disease
AVROBIO has a preclinical research program for a gene therapy for Pompe disease. We have shown a favorable preclinical safety and efficacy profile in a mouse model of Pompe ...
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Manufacturerbased in San Francisco, CALIFORNIA (USA)
Tempo Bioscience, Inc (Tempo) develops human stem cells (human iPSC technology) based cell models and biosensor enabling technologies for preclinical drug discovery & development, biobanking, in vitro diagnostics, and biomarker development. Our ...
Model Tempo-iCardio - Human iPSC-derived Cardiomyocytes
in various in vitro or in vivo studies; transplantation into normal or diseased systems; cardiac toxicology studies; or cardiovascular developmental studies. Cardiomyocytes have a high mitochondrial density, which allows them to ...
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Technologybased in Gaithersburg, MASSACHUSETTS (USA)
In November 2023, Selecta Biosciences Inc. (NASDAQ: SELB) merged with Cartesian Therapeutics to create a publicly traded company pioneering RNA cell therapy to treat autoimmune disease. The combined company will focus on advancing Cartesian’s wholly ...
Model MMA-101 - ImmTORTM + Gene Therapy Candidate for Methylmalonic Acidemia (MMA)
Selecta’s first ImmTORTM + gene therapy candidate MMA-101 for methylmalonic acidemia (MMA) is expected to enter clinical trials the end of 2021. MMA is a rare metabolic disease that may lead to metabolic acidosis and ...
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