Cystetic Medicines
Clinical-stage biotechnology company developing inhaled small-molecule pore-forming therapeutics (“molecular prosthetics”) to restore anion transport in airway epithelia for people with cystic fibrosis, with a focus on individuals who produce little-to-no CFTR protein. The company advances preclinical structural and functional studies, inhalation formulation engineering, and early-phase clinical trials toward scalable pulmonary delivery.
Industries
Nr. of Employees
small (1-50)
Patents
Ion channel prosthetic compositions comprising lipid-coated crystals of amphotericin B
US-12419899-B2
View Details
Ion channel prosthetic compositions comprising lipid-coated crystals of amphotericin B
US-12419899-B2
View DetailsProducts
CM001 (investigational inhaled small-molecule ion-channel therapeutic)
Investigational self-assembling small-molecule therapeutic formulated as a dry powder for inhalation that forms ion channels in airway epithelial membranes to restore anion transport independently of CFTR protein.
CM001 (investigational inhaled small-molecule ion-channel therapeutic)
Investigational self-assembling small-molecule therapeutic formulated as a dry powder for inhalation that forms ion channels in airway epithelial membranes to restore anion transport independently of CFTR protein.
Services
Provision of information and enrollment contacts for ongoing clinical studies of inhaled small-molecule therapeutics for cystic fibrosis.
Provision of information and enrollment contacts for ongoing clinical studies of inhaled small-molecule therapeutics for cystic fibrosis.
Expertise Areas
- Clinical trial management (Phase I SAD/MAD)
- Inhalation formulation and aerosol science
- Particle engineering and spray-dried dry powder development
- Preclinical translational pulmonary research
Key Technologies
- Dry powder inhalation
- Spray drying
- Lipid-coated particle formulation
- Nanoparticle/particle engineering
News & Updates
First healthy volunteer dosed in a randomized, double-blind, placebo-controlled Phase 1 study evaluating safety, tolerability and PK of an inhaled dry powder therapeutic; study includes SAD and MAD parts and biomarker evaluation.
Company reported dosing the first person with CF in an ongoing Phase 1 trial and presentation of single-ascending dose data at a major cystic fibrosis conference.
Investigator-initiated clinical study reported that an antifungal small molecule produced statistically significant changes in nasal potential difference in people with CF not on modulators.
Summary and commentary on Nature report showing that a pore-forming small molecule restored ion transport and antibacterial defenses in human CF airway cells and an animal model.
First healthy volunteer dosed in a randomized, double-blind, placebo-controlled Phase 1 study evaluating safety, tolerability and PK of an inhaled dry powder therapeutic; study includes SAD and MAD parts and biomarker evaluation.
Company reported dosing the first person with CF in an ongoing Phase 1 trial and presentation of single-ascending dose data at a major cystic fibrosis conference.
Investigator-initiated clinical study reported that an antifungal small molecule produced statistically significant changes in nasal potential difference in people with CF not on modulators.
Summary and commentary on Nature report showing that a pore-forming small molecule restored ion transport and antibacterial defenses in human CF airway cells and an animal model.