Chronic kidney disease affects more than 10 percent of U.S. adults, and the risk of developing the condition increases with age. There are plenty of drugs to treat the disease’s symptoms, such as anemia, high blood pressure, and bone trouble.
While these drugs can manage the signs and symptoms of the disease, they don’t cure the underlying cause, the Mayo Clinic notes. As the condition worsens, patients enter end-stage renal failure, which requires kidney dialysis or a transplant.
But research has uncovered genetic signs that point to the cause of the disease, and a new biotech firm, Goldfinch Bio, wants to turn that research into new therapies.
Cambridge, MA-based Goldfinch has raised $55 million from Third Rock Ventures, where it incubated for more than a year. Goldfinch aims to build a database of genetic information from patients and their tissue samples, says interim CEO Abbie Celniker, who is also a Third Rock partner. The firm is counting on medical centers around the world to send in their data, which Goldfinch will analyze for mutations that line up with patterns of disease that also show up in the database—such as a patient’s level of proteinuria, the presence of protein in the urine.
There’s a growing body of evidence that genetics can cause kidney disease in children and adults, says Peter Mundel, the company’s co-founder and senior vice president of biology. By identifying key mutations, Goldfinch aims to develop targeted therapies, similar to the approach drug developers have taken to develop drugs for cancer patients who share underlying mutations. “Our idea is to take the oncology approach and bring it to the kidney space,” Mundel says.
The patient database—or registry, in medical speak—that Goldfinch is building will also help the company understand which patients to study in clinical trials, Celniker says. There’s precedent for building a patient registry to support drug discovery and development. South San Francisco-based MyoKardia (NASDAQ: MYOK), which like Goldfinch received early financial backing from Third Rock, built its patient registry to develop drugs for genetic heart disease.
Celniker says Goldfinch will also build biological laboratory tools for exploring the genetics of the disease and finding and testing drugs. For example, Mundel says the firm is developing an “organoid,” a kidney in a dish. The $55 million might also be enough to also prepare one or two drug candidates for clinical trials, Celniker says. She expects to advance a compound into the clinic by late 2018 or early 2019.
Goldfinch is turning its initial attention toward two types of kidney disease that have a known genetic component: focal segmental glomerulosclerosis (FSGS), damaging of the glomeruli, the tiny filtering units inside the kidney that clean the blood; and polycystic kidney disease (PKD), an inherited disorder characterized by cysts forming in the kidney. Both conditions have no FDA-approved therapies. From there, the company hopes to use its molecular understanding of how disease develops to find ways to treat more patients who have chronic kidney disease.