Aradigm Corporation and geneRx+ have announced a joint collaboration to study the feasibility of pulmonary delivery of geneRx+''s two lead gene drug candidates via the AERx technology platform. These two compounds have been identified as potential treatments for hereditary emphysema and pulmonary hypertension.

The collaboration focuses on two proprietary geneRx+ products, Alphagen and Coxagen, that, when chronically administered, have the potential to induce a patient''s own cells to produce therapeutic proteins. Alphagen is being studied for the treatment of hereditary emphysema, beginning with alpha-1 antitrypsin (A1AT) deficiency and Coxagen for the treatment of pulmonary hypertension, beginning with the primary form of that disease (PPH). In a proof-of-concept Phase 1b study, geneRx+ successfully demonstrated the expression of the A1AT gene (Alphagen). Both of these products should qualify for orphan drug status by the U.S. Food and Drug Administration.

According to the agreement, the two companies will jointly work on formulation efforts, with Aradigm designing and conducting preclinical studies and assessing the potential of the compounds via the AERx pulmonary delivery platform. Further, the agreement provides Aradigm a licensing structure in which Aradigm could further develop and commercialize the two compounds. This work is being partly funded by a grant awarded to geneRx+ from the National Institutes of Health.

"The collaboration enhances our strategies of optimizing the potential of our partner''s pipeline and addressing unmet medical needs through innovative non-invasive approaches to drug delivery," said Richard Thompson, President and Chief Executive Officer of Aradigm. "Aradigm''s experience in the pulmonary delivery and formulation of gene-based medicines is a result of our existing work with the National Institutes of Health and makes geneRx+''s drug candidates a fundamental fit for the AERx technology platform."

A1AT deficiency is one of the most common and serious hereditary disorders and can result in life-threatening lung and/or liver disease. PPH is a rare and often fatal disease seen in the progressive narrowing of the blood vessels of the lungs resulting in elevated pressure in the pulmonary circulation and eventually heart failure. Often misdiagnosed, existing treatments for both diseases consist of injectable therapies or, in some cases, organ transplantation.

"We at geneRx+ are excited about coupling our proprietary formulations with Aradigm''s aerosol delivery technologies," said Ken Brigham, Chief Executive Officer of geneRx+. "We believe that pairing nucleic acid based therapeutics applicable to acquired diseases of the lungs with state of the art delivery technologies is a promising approach to development of novel therapies."