Research programme: personalised orphan disease therapeutics - Perlara

Drug Profile

Research programme: personalised orphan disease therapeutics - Perlara

Alternative Names: Batten disease therapeutic - Perlara; Leigh syndrome therapeutic - Perlara; NGLY1 therapeutic - Perlara; NPC1 therapeutic - Perlara; PERL 101

Latest Information Update: 21 Oct 2016

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At a glance

  • Originator Perlstein Lab
  • Developer Perlara
  • Class Small molecules
  • Mechanism of Action Undefined mechanism
  • Orphan Drug Status

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

  • New Molecular Entity Yes
  • Available For Licensing Yes - Niemann-Pick disease type C

Highest Development Phases

  • Preclinical Niemann-Pick disease type C
  • Research Inborn genetic disorders; Leigh disease; Neuronal ceroid lipofuscinosis

Most Recent Events

  • 20 Oct 2016 Perlara collaborates with Novartis for the development of drugs for lysosomal storage disorders including Niemann-Pick Type C disease
  • 20 Jan 2016 Early research in Inborn genetic disorders in USA (unspecified route)
  • 20 Jan 2016 Early research in Leigh disease in USA (unspecified route)
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