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We work with families, advocacy groups and BioPharma to find cures for rare diseases

We work with families, advocacy groups and BioPharma to find cures for rare diseases

Perlstein Lab PBC (PLab) is the first orphan therapeutics Public Benefit Corporation accelerating drug discovery for the 5,000+ rare genetic diseases.

PLab uses a platform of CRISPR-engineered animals (yeast, nematodes, fruit flies and zebrafish) in phenotypic screens to identify orphan drug candidates that reverse disease much faster and cheaper than current approaches.

Our proof-of-concept diseases are Niemann-Pick Type C, a lysosomal storage disorder first described nearly a century ago, and NGLY1 Deficiency, a congenital disorder of glycosylation first diagnosed last year. In addition to these, we are incorporating two additional diseases in our pipeline, namely Batten’s disease and Leigh Syndrome.
Total raised

$2,500,000

Funded over

1 round

Latest round (Apr 2015)

Seed

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