Corp America February 2017

22 CORP AMERICA / FEBRUARY 2017 , Errant Gene Therapeutics, LLC™ (EGT) is a privately held biopharmaceutical company specializing in the development of treatments for life-threatening diseases with a special focus on rare diseases. We explore the firmand the fight Patrick is having with an international corporation to regain rights to treatments which have a very personal meaning to him. Patrick Girondi: Fighting to Fix Errant Genes Founded in 2003, EGT has been dedicated since inception to developing its portfolio of pharmaceutical products to correct the erroneous gene expression associated with many of these diseases. The firm’s therapies are currently being developed for hemoglobinopathies and selected cancers. The company’s product portfolio is the result of license agreements to technology for exclusive world-wide rights and the acquisition of a library of novel pharmaceutical molecules. Patrick, who founded the firm based on personal experience as his son has a rare form of cancer. He has since also mounted a law suit against Sloan-Kettering’s cancer research institute for fraud and breach of a rights transfer agreement for a blood disorder gene therapy treatment. Originally the U.S. District Court for the Southern District of New York dismissed EGT’s claim to documents, clinical trial data and samples and denied standing to two clinical trial participants, however it has since agreed to hear a trial. The case originated when, ten years ago, at a time when few companies were interested in gene therapy, Sloan Kettering licensed the rights to an experimental treatment to EGT. But after being accused by the cancer center of not fulfilling its obligations to move the therapy toward the market, EGT ceded its rights in 2011. Now, because of technological progress, gene therapy is considered highly promising. A company called Bluebird Bio has a market valuation exceeding $3 billion, largely on the basis of a very similar gene therapy for beta thalassemia and sickle cell anemia, which has had strong results in early clinical trials. However, while the well-financed Bluebird races ahead, the project at Sloan Kettering appears to have languished. In the absence of other therapy, Patrick’s son gets blood transfusions every 18 days and takes 10 pills daily to reduce the toxic iron that builds up in his blood and organs. Looking ahead, two of EGT’s products are poised to enter into clinical development, offering a number of exciting opportunities for the firm to support patients around the world. With regards to the case, the court which previously dismissed the case has since allowed EGT’s fraud and breach of contract claims to survive, although the breach claims are not allowed to include EGT’s allegations that conduct by Sloan-Kettering violated the clinical trial protocol. This offers hope for the future for both EGT and Patrick’s son. K Company: Errant Gene Therapeutics Name: Patrick Girondi Address: 218 North Jefferson Street, Suite 300, Chicago, IL. 60661, USA Phone: +1 (312) 441-1800 Fax: +1 (312) 893-2200 Website: errantgene.com 1701CA40