Scalable drug discovery platform

All animals sit on the same branch of the evolutionary tree and share thousands of genes. Many of these ancient genes are unchanged at the DNA level despite a billion years of evolution.

Single gene diseases are caused by a spectrum of mutations and result in cellular defects. Missense mutations prevent proteins from folding correctly, which causes partial loss of gene function. Nonsense mutations prevent protein production, which a causes complete loss of gene function.

Gene editing technology enables us to generate patient-mutation-matched Perlara disease models that leave no mutation behind.

The resulting disease models manifest cellular defects that can be reversed by novel chemical entities in drug screens. The first version of the Perlara Platform leverages multi-well plate technology and automation.

We partner with BioPharma and Patient advocacy Groups to advance precision drug candidates to the clinic. Our pipeline begins with childhood single-gene disease involving loss of an ancient cellular process: lipid storage diseases, congenital disorders of glycosylations, peroxisomal biogenesis disorders, mitochondrial diseases, ciliopathies and laminopathies.