The startup develops inhaled therapeutics that restore ion balance in the lungs of cystic fibrosis patients, improving lung function and overall health-related quality of life. Their approach also has potential applications in treating fungal infections, providing a new therapeutic avenue for affected individuals.
Funding
Funding not disclosed
Founders
Product
Problem
Cystic fibrosis (CF) results from a missing or defective CFTR anion channel in the lungs, leading to impaired bicarbonate and anion transport. While CFTR modulator treatments exist, they are ineffective for approximately 10% of CF patients who produce little to no CFTR protein. This "final 10%" lack a treatment that addresses the underlying protein deficiency.
Solution
Cystetic Medicines is developing molecular prosthetics as functional replacements for the missing CFTR protein, aiming to restore critical airway function for CF patients who do not benefit from existing modulator therapies. Their lead candidate, CM001, is an inhaled dry powder formulation of amphotericin B, a small molecule that forms ion channels in cells, mimicking the function of the CFTR protein. The drug is delivered directly to the lungs via a portable inhaler, optimizing its impact on airway function. This approach seeks to provide a simple, accessible therapy that improves lung function and quality of life for the final 10% of the CF community.
Target Audience
The primary target audience is the 10% of cystic fibrosis patients with little-to-no CFTR protein who do not respond to CFTR modulator therapies.
Features
- CM001: An inhaled dry powder formulation of amphotericin B
- Molecular prosthetic approach: Amphotericin B acts as a functional replacement for the missing CFTR protein
- Targeted delivery: Dry powder formulation engineered for optimal delivery to the bronchial airways
- Portable inhaler: Designed for ease of use and accessibility
- Clinical validation: Demonstrated improvement in key biomarkers in a proof-of-concept clinical study
- Commercial scalability: Utilizes dry powder technologies already used in existing CF treatments