Molecular Prosthetics for Cystic Fibrosis
One Simple Therapy for people with
Including the Final 10%
10% of the cystic fibrosis community has been holding its breath for far too long.
Let’s change that.
Introducing our latest breakthrough:
A new and promising approach—molecular prosthetics—has already shown significant promise by changing nasal potential difference in people with CF not on modulators.
How it works
Cystic fibrosis, affecting more than 80,000 people worldwide, is caused by a missing or defective anion channel in the lungs, called CFTR. Although existing treatments called modulators can help many with cystic fibrosis, 10% of people with cystic fibrosis have mutations that produce little-to-no CFTR—meaning they can’t benefit from modulators—and still await a treatment that addresses the missing CFTR protein.
That’s where cystetic Medicines comes in. Molecular prosthetics serve as functional
replacements for the missing CFTR protein, thereby restoring critical airway function. With this concept, we aim to provide real and lasting benefit to the final 10%.
Who it helps
Progress in fighting cystic fibrosis has dramatically accelerated in recent years, but for thousands of people with cystic fibrosis—the 10% with little-to-no CFTR protein—hope and treatment have remained out of reach.
At cystetic Medicines, we’re inspired by the final 10%: their stories, their hope, and the resilience with which they’ve fought for their own path to treatment. It’s a community we’re incredibly proud to serve.
When it will be available
Proof of concept
The molecular prosthetics approach has already been tested in an investigator-initiated proof-of-concept study. Seven of eight people with CF not on modulators experienced decreases in nasal potential difference when a molecular prosthetic was added to their nose.
cystetic Medicines has launched its first clinical candidate, CM001, in phase 1 clinical studies. Subject to the results from the Phase 1 trial, Phase 2 clinical studies are expected to begin as soon as possible thereafter.
Who’s leading the charge
cystetic Medicines was founded by Dr. Martin D. Burke, May and Ving Lee Professor for Chemical Innovation at University of Illinois at Urbana Champaign, and Dr. Michael J. Welsh, Professor of Internal Medicine, Pulmonary; Critical Care & Occupational Medicine at the University of Iowa Carver College of Medicine. They are joined on the leadership team by Dr. Jeffry Weers, Chief Technology Officer and a leader in the field of aerosolized medicines.
Dr. Martin D. Burke
Co-founder of cystetic Medicines, May and Ving Lee Professor for Chemical Innovation at University of Illinois at Urbana Champaign
Dr. Michael J. Welsh
Co-founder of cystetic Medicines, Professor of Internal Medicine, Pulmonary; Critical Care & Occupational Medicine, University of Iowa Carver College of Medicine
Dr. Jeffry Weers
Chief Technology Officer, cystetic Medicines
What’s the hope?
Immediately, we’re looking to provide hope and treatment to the final 10%.
But we believe molecular prosthetics can help everyone with CF—and we’re committed to democratizing practical access to lifesaving CF care.