Goldfinch Bio Presents Novel Process for Developing Human iPSC-Derived Organoids at Scale at the American Society of Nephrology Kidney Week 2019 Annual Meeting

Goldfinch Bio, a U.S.-based, clinical stage biotechnology company focused on discovering and developing precision medicines for the treatment of kidney diseases, presented a novel approach to producing human induced pluripotent stem cell (iPSC)-derived organoids at scale in an oral presentation on November 9^th at the American Society of Nephrology (ASN) Kidney Week 2019 Annual Meeting in Washington, D.C.

"Human iPSC-derived organoids are emerging models that complement traditional animal studies in drug target validation and preclinical assessment of therapeutic agents targeting the kidney. However, challenges exist in scaling kidney organoid production for routine drug testing," said Anthony Johnson, M.D., President and Chief Executive Officer of Goldfinch Bio. "The data presented at ASN demonstrate the robust, reproducible and scalable platform that Goldfinch Bio has developed for generating human kidney organoids. Our assembly line production process results in decreased variance, increased quality, lower costs, and enables greater accuracy and efficiency. We expect the integration of robotics into this process, currently underway, to facilitate even greater scale, enabling the use of organoids to validate treatments on a patient-by-patient basis."

To validate this approach, Goldfinch Bio researchers leveraged its human iPSC-derived kidney organoids to test cyclosporin A (CsA), a positive control, and the company's lead compound GFB-887, a sub-type selective, small molecule transient receptor potential canonical 5 (TRPC5) ion channel inhibitor, currently in clinical development for focal segmental glomerulosclerosis, treatment-resistant minimal change disease, and diabetic nephropathy. CsA works downstream of TRPC5 in the Rac1 pathway by blocking calcineurin. Using these organoids, researchers demonstrated the therapeutic effect of both CsA and GFB-887 on the TRPC5-Rac1 pathway, validating the utility of human kidney organoids for compound testing. Scientists also observed the functional connectivity of transplanted kidney organoids to host vasculature, a complement to current in vivo efficacy models that allows for assessment of potential therapeutic activity on human-derived tissue in an in vivo setting.

A copy of the abstract, entitled, An iPSC platform for Human Preclinical Evaluation of Kidney Disease Targeting Compounds, is available on the Goldfinch Bio corporate website at https://www.goldfinchbio.com/publications.

About GFB-887

GFB-887 is a sub-type selective, small molecule transient receptor potential canonical 5 (TRPC5) ion channel inhibitor in clinical development for the treatment of focal segmental glomerulosclerosis (FSGS), treatment-resistant minimal change disease (TR-MCD) and diabetic nephropathy (DN). The ongoing Phase 1 study (NCT03970122) is evaluating the safety, tolerability, and pharmacokinetic profile of GFB-887 in healthy volunteers.

TRPC5 is a calcium-permeable ion channel implicated in the pathogenesis of kidney disease. Recent evidence has demonstrated that TRPC5 and Rac1 form a vicious cycle that drives pathogenic remodeling of the actin cytoskeleton in podocytes. This causes podocyte loss and breach of the filtration barrier, leading to proteinuria, the hallmark of progressive kidney diseases such as FSGS, TR-MCD and DN. Inhibition of TRPC5 offers a potential point of therapeutic intervention to restore podocyte integrity and halt progression of these diseases.

About Goldfinch Bio

Goldfinch Bio, Inc. is a clinical stage biotechnology company that leverages a genomics-based, precision medicine approach to discovering and developing kidney disease treatments. Its Kidney Genome Atlas^TM is a proprietary biology platform that drives candidate discovery, patient selection and biomarker development. The Company's lead candidate, GFB-887, is a transient receptor potential canonical 5 (TRPC5) ion channel inhibitor being evaluated in a Phase 1 clinical trial for the treatment of kidney diseases. Goldfinch Bio is also developing GFB-024, a peripherally-restricted cannabinoid receptor 1 (CB1) inverse agonist monoclonal antibody, for the treatment of rare and metabolic kidney diseases, which it licensed from Takeda Pharmaceutical Company Limited in October 2019. The company expects to submit an IND for GFB-024 in 2H 2020. Goldfinch Bio, headquartered in Cambridge, Massachusetts, was launched in 2016 by Third Rock Ventures and has an established strategic collaboration with Gilead Sciences, Inc. For more information about Goldfinch Bio, visit www.goldfinchbio.com.