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JR-441 is a recombinant fusion protein of antibody fragment against the human transferrin receptor and heparan N- sulfatase, the enzyme missing or malfunctioning in subjects with MPS IIIA.
JR-171 (lepunafusp alfa) is a recombinant fusion protein of an antibody against the human transferrin receptor and α-L-iduronidase, the enzyme that is missing or malfunctioning in subjects with MPS I.
AT845, a novel gene replacement therapy using an AAV8 vector, under a cardiac- and skeletal muscle-specific promotor, delivers a functional copy of the GAA gene, for the treatment of Late-Onset Pompe Disease (LOPD).
AT845, a novel gene replacement therapy using an AAV8 vector, under a cardiac- and skeletal muscle-specific promotor, delivers a functional copy of the GAA gene, for the treatment of Late-Onset Pompe Disease (LOPD).
AT845, a novel gene replacement therapy using an AAV8 vector under a muscle-specific promotor to deliver a functional copy of the GAA gene, for the treatment of LOPD.
Astellas is developing AT845, a novel gene replacement therapy using an AAV8 vector, under a cardiac- and skeletal muscle-specific promotor, to deliver a functional copy of the GAA gene, for the treatment of Late-Onset Pompe Disease (LOPD).
ASPIRO is a two-part, multinational, randomized, open-label ascending dose trial to evaluate the safety and preliminary efficacy of AT132 in XLMTM patients less than five years of age.
This decision follows the reporting of a recent serious adverse event (SAE) in a study participant due to abnormal liver function tests (LFTs) observed in the weeks following dosing of the AT132 investigational gene therapy product at a lower dose (1.3x1014 vg/kg).
JR-171 is a blood-brain-barrier (BBB)-penetrating form recombinant α-L-iduronidase that was developed using JCR’s proprietary J-Brain Cargo® BBB technology.
In this study, MPS I patients will be treated with JR-171 for at most 12 weeks to evaluate the safety of JR-171 as the primary endpoint and exploratory efficacy and pharmacokinetics as secondary endpoints.