Research programme: mucopolysaccharidosis IIIA gene therapies - LYSOGENE

Drug Profile

Research programme: mucopolysaccharidosis IIIA gene therapies - LYSOGENE

Alternative Names: LYS-SAF-302; SAF-302

Latest Information Update: 11 Jul 2016

Price : $50

At a glance

  • Originator LYSOGENE
  • Class Gene therapies
  • Mechanism of Action Gene expression modulators
  • Orphan Drug Status

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

    Yes - Mucopolysaccharidosis III
  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

    No
  • New Molecular Entity Yes

Highest Development Phases

  • Research Mucopolysaccharidosis III

Most Recent Events

  • 11 Jul 2016 Lysogene plans a phase II/III trial for Mucopolysaccharidosis type IIIA
  • 24 Nov 2015 Research programme: mucopolysaccharidosis IIIA gene therapies - LYSOGENE receives Orphan Drug status for Mucopolysaccharidosis III in USA
  • 21 Jul 2015 Lysogene collaborated with Alcyone to evaluate the intraparenchymal delivery of Lysogene’s proprietary rAAVrh 10, to treat patients with mucopolysaccharidosis type IIIA (MPS IIIA)
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