Research programme: personalised orphan disease therapeutics - PerlaraAlternative Names: Batten disease therapeutic - Perlara; Leigh syndrome therapeutic - Perlara; NGLY1 therapeutic - Perlara; NPC1 therapeutic - Perlara; PERL 101
Latest Information Update: 21 Oct 2016
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.
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.
- 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