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Drug Profile

SAF 301

Alternative Names: LYS-SAF301; SAF-301

Latest Information Update: 22 Jun 2021

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At a glance

Originator LYSOGENE
Class Gene therapies
Mechanism of Action Gene transference; N-sulfoglucosamine sulfohydrolase replacements

Orphan Drug Status

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

Yes - Mucopolysaccharidosis III
New Molecular Entity No

Highest Development Phases

Phase I/II Mucopolysaccharidosis III

Most Recent Events

22 Jun 2021 Alcyone Lifesciences is now called Alcyone Therapeutics
22 Dec 2017 Lysogene plans a pivotal phase II/III trial for Mucopolysaccharidosis III in USA and Europe in 2018
28 Jun 2017 SAF 301 is still in phase I/II development for Mucopolysaccharidosis III in France

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