Trial Profile

Phase I/II Study of JR‐171 in Patients With Mucopolysaccharidosis Type I

Status: Completed

Phase of Trial: Phase I/II

Latest Information Update: 24 Mar 2025

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At a glance

Drugs Lepunafusp alfa (Primary)
Indications Mucopolysaccharidosis I
Focus Adverse reactions; First in man
Sponsors JCR Pharmaceuticals

Most Recent Events

06 Mar 2024 Results of safety and PK, published in the Molecular Therapy
14 Feb 2024 Interim results published in a JCR Pharmaceuticals Media Release.
14 Feb 2024 According to a JCR Pharmaceuticals Media Release, 52-week safety and efficacy data from this trial were presented at the 20th Annual WORLDSymposium, 2024.

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