Goldfinch Bio is a biotechnology company based in Cambridge, MA which focuses on advancing kidney precision medicine. Its mission is to deliver disease-modifying precision medicine that brings hope and renewed quality of life to people living with kidney diseases. We recently spoke with Goldfinch Bio’s Dr. Liron Walsh, Vice President Clinical and Translational Nephrology, who explains more about the company’s precision medicine approach, breaks down what podocytes are, and speaks more on its Phase 2 clinical research trial, TRACTION-2, which is currently enrolling patients.
What is precision medicine?
The causes of kidney disease are unique among groups of individuals. In diseases such as focal segmental glomerulosclerosis (FSGS), the causes of disease can vary from genetic to environmental. However, current treatments for FSGS are the same regardless of the cause.
With these “one-size-fits-all” approaches, some patients respond to treatment, while others do not. Many patients experience harmful side effects from these treatments with little to no benefits.
The goal of precision medicine is to develop treatments which are specifically designed to treat the underlying cause of an individual’s kidney disease. In this way, patients can receive the right medicine and avoid treatments which are not going to be helpful or may even be harmful.
Today, precision medicine is often used to treat cancer. By first understanding the genetic makeup of a patient’s tumor, a doctor can then choose the best medicine to specifically treat that patient’s cancer. This approach has greatly improved the lives of many people with cancer.
Precision medicine is now on the horizon for people with kidney diseases. Patients, nephrologists, and kidney researchers are working together to better understand the causes of kidney diseases. Through this deeper understanding, precision medicine will help develop treatments that target the specific causes of kidney diseases like FSGS and minimal change disease (MCD). Precision medicine holds promise to improve treatment outcomes and quality of life for many people living with kidney disease.
Goldfinch Bio and Precision Medicine
Goldfinch Bio is developing a novel experimental medicine, GFB-887, with the hope of preventing the progression of kidney disease in patients with FSGS, treatment-resistant MCD (TR-MCD), and diabetic nephropathy. While these kidney diseases may seem very different, they have something very important in common– very specialized cells of the kidneys called podocytes are damaged. GFB-887 is designed to specifically target these podocytes and prevent damage.
What are podocytes?
Podocytes are very specialized cells that wrap around the blood vessels of the kidneys. They form a network with other nearby podocytes to prevent the body from losing important proteins, such as albumin, while at the same time, filtering out waste. Normal functioning podocytes are very critical to our health.
When podocytes are damaged, gaps form in the barrier and important proteins are lost in the urine. This is commonly referred to as ‘spilling protein in the urine,’ and is an early sign of kidney disease. Patients with damaged podocytes experience swelling, weight gain, and fatigue. Unfortunately, damaged podocytes cannot heal, and they cannot be replaced by new, healthy podocytes. The kidney tries to repair the damage by creating a scar, much like the scar from a cut. Over time, more damage and more scarring will lead to kidney failure. By protecting the podocytes from being damaged in the first place and ensuring that the podocytes can function normally, it may be possible to prevent or delay kidney failure.
What makes the Goldfinch Bio team different than other pharmaceutical companies?
First, we are focused on advancing treatments for people with kidney diseases. Second, we are pioneering precision medicine for kidney diseases. While precision medicine is often used in cancer, and some rare genetic diseases as well, they are an entirely new area of drug research and development for kidney diseases. We are deeply committed to bringing innovative treatments to people with kidney diseases and working very closely with the patient community as partners.
Goldfinch Bio’s tagline is ‘Saving Kidneys, Ending Dialysis.’ How did you come up with this?
Our tagline– Saving Kidneys, Ending Dialysis– is a shortened version of our vision, which is “We aspire to save kidneys and end dialysis.” We know that for many patients, kidney transplant and dialysis are the only treatment options. Unfortunately, kidney transplant may not be an option for many patients and is associated with significant long-term complications, and the experience of dialysis is very difficult. Through our precision medicine approach, we want to bring new, effective treatment solutions to patients so that we can give them hope and a renewed quality of life. We finalized our vision by hosting a focus group with people living with kidney disease to really understand what was important to them. A resounding theme emerged: these individuals expressed to us that they would give anything to avoid dialysis. And, from there, our new vision emerged.
Can you tell us more about your ongoing clinical trial?
The TRACTION-2 clinical research trial is evaluating our investigational precision medicine, GFB-887, for the potential treatment of FSGS, TR-MCD, and diabetic nephropathy. The purpose of the trial is to determine if GFB-887 is safe and may help people who have high levels of protein in their urine due to kidney diseases caused by podocyte injury. For more information on the trial, you can visit tractionclinicaltrial.com or at Kidney Health Gateway.
What are you most looking forward to at Goldfinch Bio?
We are very excited about the recent creation of our FSGS Patient Advisory Board. We have created this board to foster a close working partnership with the patient community. For National Kidney Month, we held a virtual panel discussion with our board members. You can learn more about our Patient Advisory Board and watch the panel discussion here. We are very excited to continue to collaborate with this Board to ensure we have a patient-centric approach and mindset throughout our company.
To learn more about the TRACTION-2 clinical trial, click here. To see a full list of clinical research opportunities, and to find the trial best for you or your loved one, visit KidneyHealthGateway.com.
This article was developed in partnership with Goldfinch Bio, Inc.