HEALTH March 23, 2015

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HEALTH • Spring 2015 17 (UMMS) in Worcester and a researcher at the medi- cal school's Gene Therapy Center who specializes in lung, liver and central nervous system disorders. Genetic research takes years, and sometimes decades, before any results are realized. Once a com- pany can demonstrate that a theory can be applied to real-world situations, researchers create a viral vec- tor, which is the tool that will deliver genetic mate- rial into the cell. This vector must be manufactured in large quanti- ties under government-approved conditions. During early clinical trials, researchers must provide data showing where the virus goes after it's injected into the animal or human subject, a process called bio- distribution. Toxicity studies must also be completed to ensure the virus is safe for use in humans. Mueller said it could take between six and 18 months for this process to be completed. The price tag to produce enough vector to treat 10 patients ranges between $1 million and $4 million, he added. Since these vectors address genetic condi- tions that affect a small percentage of the popula- tion, the demand is relatively small, which results in a difficult cost-benefit equation. Funding sources Federal sources have helped some in advancing clinical research. The National Heart, Lung and Blood Institute (NHLBI), under the auspices of the NIH, provides grants and awards, to help fund ani- mal studies, and sometimes the manufacture of the vector, according to Mueller. Foundations have also been instrumental in mov- ing gene therapy forward, Mueller said. Since 1997, the Angel Fund, a Wakefield-based nonprofit supporting amyotrophic lateral sclerosis (ALS) research, has enabled Dr. Robert H. Brown, Jr., chair of the department of neurology at UMass Memorial Medical School, to pursue research in ALS, known also as Lou Gehrig's disease. In the last three years, the foundation's financial support has fueled gene therapy exploration. Brown receives additional financial help from a private benefactor and federal sources. "The world of private venture funding sees a profit in this research. Otherwise they wouldn't invest in it," Brown said. The Europe connection Developments in Europe are helping to inject enthusiasm, energy and, most importantly, cash, into gene therapy exploration in the U.S. In 2012, the European Medicines Agency's Committee for Medicinal Products, a regulatory body that authorizes medicines before they can be marketed, gave Dutch biotech company UniQure approval to market Glybera, a gene therapy for a rare, inherited blood plasma disease. Such discoveries and developments in gene thera- py are helping to breathe life into the field, according to Mueller, of the UMMS Gene Therapy Center; he said the UniQure approval led to a "huge influx" of venture capital funding for the industry. "More than $700 million poured into genetic therapy in one year," Mueller said. Tabatadze, of Zata Pharmaceuticals, added that in 2004, he and Zamecnik led a research project at Massachusetts General Hospital that resulted in the restoration of a mutated cystic fibrosis gene. They licensed the intellectual property to ProQR Therapeutics B.V., a startup in The Netherlands. ProQR further developed the technology and the company intends to launch a Phase I clinical study, according to Tabatadze. ProQR recently launched a public stock offering and is said to be worth more than $480 million. "In other words, technology developed here in Massachusetts generated (a) half-a-billion dol- lar business in the European Union," Tabatadze said. This cash infusion has also spawned an explosion of new companies in the industry at home. In 2014, Dr. Guanping Gao, director of the UMMS Gene Therapy Center and Vector Core, and Dr. Phillip Zamore, co-director of the RNA Therapeutic Institute at UMMS, founded Voyager Therapeutics in Cambridge, which is developing gene therapies for CNS diseases. Last February, Boston-based Third Rock Ventures invested $45 million to help launch Voyager. Adeno- associated virus (AAV), one of Voyager's key tech- nologies, has recently emerged as a highly attractive approach to gene therapy for patients with central nervous system diseases. Gillis explained that Third Rock's Series A fund- ing will allow Voyager to focus on developing its platform and pipeline, not on raising capital. Once Voyager reaches certain milestones in its research efforts, Third Rock will consider an initial public offering or a merger and acquisition. Another development came in February, when Genzyme, which is based in Cambridge and has operations in Framingham and Westborough, part- nered with Voyager on developing gene therapy. Mark Barrett, vice president of strategy and business development for Genzyme's rare diseases business, indicated the partnership would enable both compa- nies to become industry leaders. According to Barrett, gene therapy is experienc- ing renewed interest due to research that has shown the safety and effectiveness of the AAV as a delivery tool "is makes gene therapy a more attractive approach for using vectors to correct genetic defects," Barrett said. David R. Tabatadze, managing partner of Worcester-based Zata Pharmaceuticals Inc., said his company has the technology to potentially help people with cystic fibrosis live full lives, but maintaing funding to bring it to market is a challenge. Christian Mueller, researcher at UMass Medical School's Gene Therapy Center "... technology developed here in Massachusetts generated half-a-billion dollars in the European Union." David R. Tabatadze, Zata Pharmaceuticals Inc.

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