Drug Profile

Mucopolysaccharidosis I therapeutic - Genetix Biotherapeutics

Alternative Names: HSC LVV-gene-modified stem cells - Genetix Biotherapeutics

Latest Information Update: 03 Oct 2025

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At a glance

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity No

18 Sep 2025 bluebird bio is now called Genetix Biotherapeutics
28 Jun 2023 No recent reports of development identified for preclinical development in Mucopolysaccharidosis-I in USA (Parenteral)
10 May 2019 Preclinical trials in Mucopolysaccharidosis I in USA (Parenteral)

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