Amphotericin B cystetic for inhalation

CM001 (also known as amphotericin b cystetic for inhalation or ABCI) is an investigational small molecule therapy developed as a molecular prosthetic for the treatment of cystic fibrosis (CF), particularly targeting patients who do not benefit from standard CFTR modulator therapies. The drug is designed to self-assemble and form ion channels in airway epithelial cell membranes, effectively replacing the function of the defective or absent Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein that causes CF. This mechanism allows restoration of ion channel function in patients with mutations resulting in little to no production of functional CFTR protein, addressing a significant unmet need for approximately 10% of people with CF who are unresponsive to current modulators. CM001 is administered via a portable dry powder inhaler and has entered phase 1 clinical trials, including both healthy volunteers and people with CF[1][2][3][4][5][7].

Other names

ABCI