The first healthy volunteer has been dosed with CM001, a molecular prosthetic for missing or dysfunctional cystic fibrosis transmembrane conductance regulator (CFTR) protein channels in patients with cystic fibrosis (CF), as part of phase 1 clinical trial, cystetic Medicines announced.

“The initiation of our phase 1 trial for CM001 represents an important step forward in our mission to find a treatment for everyone with CF, especially the final 10 percent of people with CF who cannot benefit from CFTR modulators,” said Martin Burke, MD, PhD, cofounder of cystetic Medicines.

“Based on encouraging pre-clinical results and initial biomarker studies in the noses of people with CF not on modulators, we are excited about the potential for this molecular prosthetic approach to restore anion channel function in a way that is independent of CFTR.”

This randomized, double-blind, placebo-controlled study is set to assess the safety, tolerability, and pharmacokinetics of single-ascending and then multiple-ascending doses of CM001 administered with a portable dry powder inhaler. It will be conducted in New Zealand.

Read more about CF experimental therapies

The experimental drug will first be evaluated in healthy volunteers. Patients with CF will be treated in the second half of 2023.

“The concept of molecular prosthetics has the potential to alter the treatment landscape for cystic fibrosis in a profound way; in theory, it should work for all people dealing with the disease, regardless of the type of mutation they present,” added Jeffry Weers, chief technology officer of cystetic Medicines. “Our team is particularly optimistic with regard to the proprietary inhaled formulation CM001, which allows for efficient targeted delivery directly to the lungs, thereby potentially avoiding side effects and enabling optimal dosing regimens.” 

According to recent studies, the active pharmaceutical ingredient in CM001 can create ion channels that resemble the CFTR protein in cultured primary airway epithelia from patients with CF irresponsive to modulators.

Moreover, an investigator-initiated clinical study found the active ingredient to modify nasal potential difference in people with CF not on modulators the same way responsive people would react to CFTR modulator therapy.

CF, caused by a mutation in the gene encoding the CFTR anion channel, is currently treated with CFTR modulators that can only partially restore the CFTR protein function. However, about 10% of patients with CF have mutations that cause little to no CFTR protein production and thus do not respond to standard CFTR modulators.  


cystetic Medicines initiates phase 1 clinical trial of a molecular prosthetic for cystic fibrosis. News release. cystetic Medicines, Inc.; June 26, 2023.