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Feb 16, 2017
 |  BC Innovations  |  Targets & Mechanisms

Gilding the Goldfinch

Why Third Rock is betting Goldfinch can bring innovation to kidney disease

In launching Goldfinch Biopharma Inc., Third Rock is extending its already heavy footprint in precision medicine into chronic kidney disease. The VC thinks the unfolding genetics in that field can yield disease-modifying therapies and a blueprint for finding the right patients.

Goldfinch was the fourth precision medicine newco in 2016 from Third Rock Ventures, and the 17th in its portfolio, but this is the firm’s first foray into kidney disease. Third Rock was the sole investor in the $55 million series A round in December that launched the company.

Partner Abbie Celniker, who is serving as Goldfinch’s interim CEO, told BioCentury part of the appeal is that nephrology is a relatively neglected therapeutic area where fast-moving basic science is starting to open up a range of translational avenues. She added that the platform fits well into Third Rock’s emphasis on “areas of medicine that are being better defined by genetics and deep biology then they had previously been.”

“There has not been an innovative therapy for chronic kidney disease developed in the last twenty-plus years. But the five-year mortality rate for dialysis is worse than for most cancers,” said Celniker.

Now, Celniker thinks the field is ready for a translational renaissance. “There’s been somewhat of an explosion in the understanding of the genetics behind forms of chronic kidney disease,” she said.

Many of those findings were made by Goldfinch’s five scientific founders, who have delineated genetic forms of chronic kidney disease (CKD), identified cellular pathways and homed in on drug targets within the pathways, said Celniker.

Goldfinch co-founder Peter Mundel, who left his academic position at Harvard Medical School to become the company’s SVP of biology, told BioCentury its approach is to tailor therapies to genetically defined populations. “It’s a little bit like bringing the oncology playbook to the kidney space.”

Goldfinch has selected focal segmental glomerulosclerosis (FSGS), an orphan renal disease, and polycystic kidney disease (PKD) as its lead indications because only one drug, the V2 receptor antagonist Jinarc tovalpatan from Otsuka Pharmaceutical Co. Ltd. and Abdi Ibrahim Pharmaceuticals Industry and Trade Co. Ltd., is approved for PKD. At least 19 others companies have compounds in development for PKD or FSGS that could be disease-modifying (see “Therapies in Flight”).

Table: Therapies in flight

CompaniesProductDescriptionIndicationStatus
Otsuka Pharmaceutical Co. Ltd. / Abdi Ibrahim Pharmaceuticals Industry and Trade Co. Ltd. Jinarc tolvaptanVasopressin 2 (V2) receptor antagonistPKD

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