Compounds capable of facilitating the transmembrane anion transport for the treatment of Cystic Fibrosis

New and innovative aspects

There are very limited studies in the literature concerning the possibility of using synthetic transport systems to replace the missing transmembrane anion transport activity characteristic of Cystic Fibrosis. Most of them referred to the use of peptides which degradability limited the potential scope of this approach.

The current invention provides a significant contribution related to the characterization, design and synthesis of small molecules with anion transport capacity, and their biological activity.

Main advantages of its use

The current standard therapies for Cystic Fibrosis are based mainly in palliative treatments of the illness symptoms. Recently it has been approved for use a drug that acts on the root of this problem, enhancing the transport of ions through natural channels, but only applicable to patients suffering from certain genetic mutations of the CFTR transmembrane protein.

These small molecules facilitate the transmembrane transport of anions independently of the genetic mutation, therefore they are therapy applicable to all Cystic Fibrosis patients, regardless of the type of mutation they harbor.

Specifications

Compounds bearing triazole-dipyrromethane moieties are capable of facilitating transport of anions such as chloride and of bicarbonate in phospholipid vesicles models and cells.

Applications

Use of the compound for preparing a medicament for the treatment and / or prophylaxis of conditions arising from abnormalities in the anion transport at the cellular level such as Cystic Fibrosis.

Intellectual property status

Protected by PCT/ES2016/070859

Current development status

Samples ready for testing

Desired business relationship

Trade Agreement, License Agreement, Technical cooperation: further development, Technical Cooperation: testing of new applications; Technical Cooperation: adaptation to specific needs.