Goldfinch Bio Presents Data Enabling the Advancement of Innovative Approaches to the Treatment of Kidney Disease at the International Society of Nephrology World Congress 2019

TRPC5 Inhibition with GFB-887 Protects Against Podocyte Injury and
Proteinuria Associated with Focal Segmental Glomerulosclerosis and
Treatment-resistant Minimal Change Disease

Integration of Longitudinal Clinical Data with Genetic and
Transcriptomic Data Enables Goldfinch Bio’s Kidney Genome Atlas™ to
Uncover Novel Targets Related to Kidney Disease Progression

Development of Podocyte Cellular Models and Organoids to Assess
Kidney Disease Mechanism and Treatment Response

CAMBRIDGE, Mass.–(BUSINESS WIRE)–lt;a href="https://twitter.com/hashtag/conference?src=hash" target="_blank"gt;#conferencelt;/agt;–Goldfinch Bio, a biotechnology company focused on discovering and
developing precision medicines for the treatment of genetically-driven
kidney diseases, today announced the presentation of preclinical data
enabling the advancement of innovative approaches to the treatment of
kidney disease. These data, which were presented at the International
Society of Nephrology (ISN) World Congress 2019, held April 12-15 in
Melbourne, Australia include:

• Transient receptor potential canonical 5 (TRPC5) inhibition with
  GFB-887 provides protection against podocyte injury and proteinuria in
  models of focal segmental glomerulosclerosis (FSGS)
  treatment-resistant minimal change disease (TR-MCD) and diabetic
  nephropathy.
• The integration of longitudinal clinical data with genetic and
  transcriptomic data enables the Goldfinch Kidney Genome Atlas™ (KGA)
  to uncover novel targets related to kidney disease progression.
• The development of podocyte cellular models and organoids facilitate
  assessment of kidney disease mechanisms and treatment response.

“The comprehensive body of data presented by the Goldfinch team at the
ISN World Congress 2019 clearly demonstrate the
significant advances that Goldfinch Bio is making towards the
development of novel therapeutics for kidney disease,” said Anthony
Johnson, M.D., President and Chief Executive Officer of Goldfinch Bio.
“The Goldfinch KGA, which is the backbone of our product engine, has
enabled the discovery of genetic variants associated with kidney
disease. These genetic variants, combined with the integration of
clinical and transcriptomic data, advance our understanding of the
molecular causes of kidney diseases and drive our development pipeline.
We remain on track to initiate clinical studies with GFB-887, our
selective, small molecule TRPC5 inhibitor, in the first half of 2019.”

In a poster titled ‘GFB-887, a small molecule inhibitor of TRPC5,
protects against podocyte injury and attenuates proteinuria in models of
FSGS,’ Goldfinch Bio researchers describe the identification of
potent and selective novel small molecule inhibitors of TRPC5, including
the development candidate, GFB-887, for the treatment of proteinuria in
FSGS, TR-MCD and diabetic kidney disease (DKD).

In a poster titled ‘The Kidney Genome Atlas: a comprehensive whole
genome and transcriptome landscape of proteinuric kidney diseases,’
Goldfinch Bio researchers describe the functionality of the KGA and its
role in advancing the understanding of the molecular mechanisms of
kidney diseases at a whole genome and transcriptome scale.

In a poster titled, ‘Comparative transcriptomic analysis of cultured
podocytes and human kidney organoids at single cell-resolution,’
Goldfinch Bio researchers describe the advancement of a model of mature
podocytes with the potential to assess both mechanisms of human kidney
disease and responses to treatment.

About Goldfinch Bio
Goldfinch Bio is a biotechnology company
that is singularly focused on discovering and developing precision
therapies for patients with kidney diseases. Just as the goldfinch has
long been a symbol of healing and renewal and was a prominent figure of
the Renaissance, Goldfinch Bio is leading a new age of therapeutic
discovery to transform the treatment paradigm for patients with kidney
diseases. Goldfinch was launched in 2016 by Third Rock Ventures, and is
headquartered in Cambridge, Mass. For more information, please visit www.goldfinchbio.com.

Contacts

The Yates Network
Gina Nugent 617-460-3579
[email protected]