A Spanish research group is working and researching on the production of a drug for the treatment and/or prophylaxis of diseases due to an abnormal transport of anions at the cellular level, offering under a licensing, manufacturing or research collaboration agreement.
Details:
The Spanish research group is an interdisciplinary and inter-university group in the fields of knowledge of biotechnology, pharmacy and chemistry. There is a group of diseases related to abnormal function of channel proteins that facilitate the transport of ions across biological membranes. These diseases, known as "channelopathies," are very diverse and include cystic fibrosis (CF). Currently approved drugs such as Kalydeco or Orkambi are aimed at pharmacological correction of the CFTR protein. However, the therapeutic success of these drugs is highly dependent 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 current strategies are ineffective.
The compound of the invention has been shown to correct key functional parameters in model epithelial cultures derived from CF patients. Thus, air surface fluidity, pH and volume, and mucus rheology in CF epithelium are corrected to values comparable to those of normal tissue by application of the compounds. Importantly, the dose required to observe these effects does not induce significant cytotoxicity due to nonspecific anion transport activity.
Possible applications of the compound
- Pharmaceutical active pharmaceutical ingredient (API) with anionophore capacity for the treatment of patients with cystic fibrosis, regardless of the mutation detected
- Synthesis of molecules with anionophore capacity as antimicrobial agents for the treatment of infections, caused by resistant bacteria (patients with cystic fibrosis/without cystic fibrosis)
- Application in studies correlating cytotoxicity with anion transport activities of anionophores
- Application in studies on anionophoric activities, affecting transmembrane pH gradients (cell membrane, intracellular compartments)
- Biosensor cell lines and cell-based assays for biomembrane research
- Nanoformulations of compounds and ion sensor assays for molecular biology research.
Wanted partners/advice:
The research group is interested in international collaboration through licensing, manufacturing and/or research collaboration agreements with other research institutions or universities and companies of any size. https://www.technologiepartner.de/de/technologiedatenbank/details?ref=TOJP20210623001
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