Adeno-Associated Viral Gene Therapeutic for Cystic Fibrosis, TR5/2-CBA-(capital alpha)264CFTR
Cystic fibrosis (CF) is caused by a defect in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The CFTR protein regulates the conductance of sodium and chloride across cell membranes and a defect can have a large impact on the salt concentration of organ fluids. The salt conductance defect has an especially devastating effect in the lungs but can also cause symptoms in the pancreas, liver, and sexual organs. The leading cause of death in CF patients is suffocation, which stems from the build up of mucus in the lung and fibrosis resulting from the innate immune systems attempt to eliminate chronic infection. There is currently no cure for CF, but gene therapy may be a promising avenue for this single gene defect. Researchers at JHU have invented a novel Adeno-Associated Viral (AAV) packaging system to deliver a unique, shortened, and functional copy of the CTFR gene, alongside other sequences that promote and enhance the protein production, into the lungs of affected individuals. The invention also uses non-traditional attachment proteins on the surface of type 5 capsids to increase the attachment and entry of the viral particles into airway cells. It is anticipated that the therapy will be administered through a nebulization or aerosolization system Description (Set) Proposed Use (Set) Cystic fibrosis is the most common life-limiting autosomal recessive disease among people of European heritage. In the United States, approximately 30,000 individuals have CF and their life expectancy hovers around 36.8 years, although death comes much sooner in under-developed countries. A therapeutic cure for CF would be of most benefit to patients that have not yet progressed to significant lung damage, although lifespan and quality of life will be improved significantly for all CF sufferers.Inventor(s): Guggino, William B.
Type of Offer: Licensing
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