International Niemann–Pick Disease Alliance

Perlara & Novartis: Join Fight Against Niemann-Pick Disease

perlara-logo-header-1650x621px-9-15-16

Perlara and Novartis join the fight against Niemann-Pick Disease:

Perlara was founded in February 2014 and is the brainchild of molecular biologist Ethan Perlstein.  Perlara is a biotech startup based in the USA, whose mission is to discover personalized cures for diseases rare and common.

The Perlara Drug Discovery Platform is based on creating disease models using simple animals that share genetic similarity with humans, allowing them to screen massive numbers of disease models and drug candidates quickly and at low cost.

Perlara uses the gene-editing tool CRISPR to generate model organisms, such as fruit flies, and worms, that feature the mutation driving a particular rare disease. They have recently focussed on Niemann-Pick type C (NP-C) disease, achieving promising results and gaining the support of Novartis in further developing the lead NP-C compound, which is currently being tested in mice.

To learn more about Perlara, visit https://www.perlara.com/

Read their latest press release: https://cen.acs.org/articles/94/i42/Perlara-signs-rare-disease-pact.html

Join the Registry

Calling all patients and carers! We've created a global registry of all NPD patients - if you're not enrolled yet, join us now...

Think Again. Think NP-C

Join our international campaign to improve diagnosis of NP-C!