Novartis invests, partners with Y Combinator grad in lysosomal storage disorders

Novartis HQ cropped

Novartis ($NVS) teed up its second discovery deal of the day with an investment and partnership with Perlara PBC to discover and develop treatments for lysosomal storage disorders using CRISPR-engineered animal models. The San Francisco-based startup is a graduate of well-known startup accelerator Y Combinator, which has provided seed funding.

Interestingly, Perlara is structured as a public benefit corporation (PBC), a company structure in California that’s intended to facilitate a devotion to a charitable purpose rather than profit. Perlara has tasked itself with orphan disease drug development. Organizations such as hospitals and colleges are sometimes organized as PBCs.

Novartis made an undisclosed equity investment in Perlara as part of the deal. The pair will start with a lead program in Niemann-Pick disease type C. NGLY1 deficiency, a metabolic disease related to proteasome-mediated degradation, may be up next.


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"Our expertise in whole-animal phenotypic screens, model organism genetics and automation is complemented by Novartis' world-class pathway and chemical biology expertise, clinical experience and global leadership," said Perlara CEO Ethan Perlstein in a statement.

Perlara is based on a CRISPR-engineered animal platform that uses phenotypic screens to identify orphan drug candidates. The expectation is that this approach will yield faster, cheaper results.

The CRISPR-altered animal models include yeast, nematodes, fruit flies and zebrafish. The idea is to identify single-gene diseases that are caused by a gene that remains present in simple animals, of which the company said that it has identified at least 250. Perlara said that it has been able to take a novel compound that it started testing in worms into a mouse model in under a year.

The Novartis deal is the first major biopharma deal for Perlara, which was founded in February 2014 as Perlstein Lab and later changed its name. The company raised more than $7 million in seed funding. Beyond Y Combinator, other Perlara investors include the biotech Retrophin, Slow Ventures and Techammer.

Y Combinator is very tech-heavy, but its other biotech alums include organism developer Gingko Bioworks, drug discovery accelerator Atomwise, nanodiamond player Bikanta and consumer microbiome company uBiome.

"We are eager to begin this next chapter so that our platform technology and discovery programs can move faster to our goal of helping patients and families impacted by diseases with high unmet need," Perlstein concluded.

For its part, Novartis also just announced another discovery deal with microcap Cerulean Pharma ($CERU) in nanotech oncology drug delivery.

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