Pharmaceutical composition for the treatment of cystic fibrosis

TechnologyŠpanielskoTOES20210604002
Offers
Summary: 
Pharmaceutical composition for the preparation of a medicament for the treatment and /or prophylaxis of diseases derived from the abnormal transport of anions at the cellular level, is offered by a Spanish research group under license, manufacturing or research cooperation agreement.
Description: 
The Spanish research group is an interdisciplinary and interuniversity group in the areas of knowledge of biotechnology, pharmacy and chemistry. This Spanish research belongs to a medium-sized Spanish university founded in 1994, housing others 76 research groups more. The university has been very active in several European research and innovation programmes (H2020 and previous FPs, LIFE, Justice, ERASMUS+, Interreg, COST, LLP, IEE, Research Fund for Coal and Steel, D. G. for Competition Policy and Strategy, Science for Peace and Security Programme-OTAN…) with over 50 European projects; with the role of coordinator in 22 projects and over 8.4 M€ overall EU funding received. Currently, this university participates in 26 on-going projects (in 12 of them as coordinator) in 8 different EU programmes with an average of 1.7 M€ of EU funding in the last 4 years. There are a group of diseases related to abnormal function of channel proteins facilitating the transport of ions through biological membranes. These conditions, known as “channelopathies”, are very diverse and include cystic fibrosis (CF). Currently, approved drugs such as Kalydeco or Orkambi target pharmacological correction of cystic fibrosis transmembrane conductance regulator (CFTR protein). However, the therapeutic outcome of these depends dramatically on the specific mutations of the patients. More than 1,700 mutations have been identified and there is a cohort of patients lacking CFTR production for whom the current strategies are not effective. The compound of the invention (Figure I) has demonstrated to correct key functional parameters in model epithelial cultures derived from CF patients. Thus, air surface liquid, pH and volume as well as mucus rheology in CF epithelium (Figure II) are corrected to values comparable to those of normal tissue upon application of the compounds. Importantly the dose required to observe these effects did not induce significant cytotoxicity due to unspecific anion transport activity. Potential applications of the compound: - Active pharmaceutical ingredient (API), with anionophore capacity for the treatment of patients with cystic fibrosis, regardless of the detected mutation. - Synthesis of molecules with anionophore capacity as antimicrobial agents for the treatment of infections caused by resistant bacteria (patients with CF / without CF). - Application in studies of correlation of cytotoxicity with anion transport activities of anionophores. - Application in studies of anionophoreic activities that influence transmembrane pH gradients (cell membrane, intracellular compartments). - Biosensor cell lines and cell based assays for biomembrane research. - Nanoformulations of compounds and ion sensors assays for molecular biologyresearch. The research group is interested in international cooperation under license agreement, manufacturing agreement and/or research cooperation agreement, with other research organization or universities and any size companies.
Type (e.g. company, R&D institution…), field of industry and Role of Partner Sought: 
License agreement - Type: research organization and industries. - Activity: pharmaceutical and medical products, and R+D. - Role: drug development for anion transport, drug development for cystic fibrosis. Manufacturing agreement - Type: pharmaceutical and basic chemical companies. - Activity: pharmaceutical production and experience in chemical production. - Role: drug development for anion transport, drug development for cystic fibrosis. Research cooperation agreement - Type: research organization and industries. - Activity: pharmaceutical and medical products, R+D and chemical research experience. - Role: drug development for anion transport, drug development for cystic fibrosis.
Stage of Development: 
Field tested/evaluated
IPR Status: 
Patents granted
External code: 
TOES20210604002