Jnana Therapeutics Raises $50 Million to Advance Lead PKU Program and Pipeline

Jnana Therapeutics, a company utilizing its next generation chemo-proteomic platform to target SLC transporters and other well-validated but hard-to-drug targets, said it closed a $50 million series B financing.

Photo: Joanne Kotz, co-founder, CEO and president of Jnana Therapeutics

RA Capital Management led the financing, which included all existing investors Polaris Partners, Versant Ventures, Avalon Ventures, AbbVie Ventures, and Pfizer Ventures.

Proceeds from this financing will enable Jnana to progress its lead program for the treatment of phenylketonuria (PKU) into clinical development, while advancing a pipeline of additional programs and continuing to invest in its validated RAPID platform for the discovery of small molecule therapeutics for hard-to-drug targets.

PKU is a rare inherited metabolic disorder caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH). This enzyme is required for the breakdown of phenylalanine (Phe), an amino acid found in all protein-containing foods. When PAH is deficient or defective, Phe accumulates to abnormally high levels in the blood. If left untreated, these toxic levels of Phe in the blood can result in progressive and severe neurological impairment and neuropsychological complications. The SLC transporter SLC6A19 is responsible for kidney reabsorption of Phe back into the bloodstream, and the inhibition of SLC6A19 offers a novel approach for the treatment of PKU.

Jnana’s lead program is designed to provide a novel oral approach to address PKU by inhibiting the SLC transporter SLC6A19 that is responsible for kidney reabsorption of phenylalanine (Phe), thereby reducing the elevated blood Phe levels that drive PKU disease pathologies. Based on data from human biology and in vivo experiments in animal models, SLC6A19 inhibition offers a promising approach to provide a profound therapeutic benefit to PKU patients.

“This commitment and support from a strong syndicate of investors will enable the next stage of evolution for Jnana as we advance our broad pipeline of wholly-owned programs to the clinic and continue to expand the application of our platform,” said Joanne Kotz, co-founder, CEO and president of Jnana Therapeutics. “We believe that Jnana’s lead program offers a differentiated approach to PKU with an oral small molecule, identified using our RAPID platform, that can be used to treat any PKU patient, notwithstanding age or PAH mutation.”

Author: Rare Daily Staff