Gene therapy briefs.

Oxford BioMedica (www.oxfordbiomedica.co.uk) said May 1 it will manufacture clinical-grade material using its LentiVector gene delivery platform technology for Novartis (www.novartis.com). Oxford BioMedica said it will also provide certain process development services and expects to receive between £2.5 million (about $3.9 million) and £4 million (about $6.2 million) from Novartis over the next 12 months. Under the agreement, Oxford BioMedica will be responsible for manufacturing several batches of a lentiviral vector encoding CTL019 technology. This vector will be used to transduce patients’ T cells in an ex vivo process before they are reinfused. CTL019 targets a protein called CD19 that is associated with a number of B cell malignancies including chronic lymphocytic leukemia, B cell acute lymphocytic leukemia, and diffuse large B cell lymphoma. ‘‘The strategic investment in our specialist manufacturing capabilities is a pivotal step towards building a financially self-sustaining business and this collaboration is an example of how we can commercialize our expertise,’’ Oxford BioMedica CEO John Dawson said in a statement. LentiVector uses a stripped-down version of the equine infectious anemia virus (EIAV). The technology is designed for long-lasting delivery of genetic material into target cells without toxicity or adverse immune reaction; integration of genes into nondividing cells; and a capacity for multiple therapeutic genes. According to Edison Investment Research, Oxford BioMedica’s deal with Novartis is expected to raise the genebased therapy firm’s valuation from £58.5 million ($91 million) to £60.1 million ($93.6 million) in that same timeframe. Edison calls the transaction ‘‘an attractive deal for Oxford BioMedica,’’ adding that it could eventually lead to a longer term supply agreement. Imperial College London (www.imperial.ac.uk) is working with the British Heart Foundation (BHF; www.bhf.org .uk), the Royal Brompton Hospital (www.rbht.nhs.uk), and other hospitals in and outside the United Kingdom on a pair of recently launched gene therapy trials for heart failure—the first two such trials to be conducted in the United Kingdom. The therapy involves raising the levels of SERCA2a protein in heart muscle cells by inserting extra genes into the cells, using a harmless virus. Research by the BHF and Royal Brompton has identified SERCA2a as an important factor affecting the contraction of heart muscle cells in people with heart failure. Doctors plan to study the gene therapy in two separate clinical trials. CUPID2 will assess whether cardiac gene therapy to increase SERCA2a is safe and can improve both quality and length of life, and reduce emergency hospital admissions, for heart failure patients. The trial, to involve 200 patients with heart failure from Royal Brompton and other sites worldwide, will begin treating people with heart failure over the next few weeks at Royal Brompton’s Cardiovascular Biomedical Research Unit, and will be funded by Celladon (www.celladon.net/). The second trial, called SERCA-LVAD, is due to start recruitment this summer. Cofunded by the BHF, this trial will test SERCA2a gene therapy in 24 U.K. heart failure patients already fitted with mechanical heart pumps, known as left ventricular assist devices (LVADs). It will give vital information about the effectiveness of the therapy by measuring the amount of the SERCA2a gene and protein that has been introduced into heart muscle. ‘‘Our goal is to fight back against heart failure by targeting and reversing some of the critical molecular changes arising in the heart when it fails,’’ Alexander Lyon, Ph.D., BHF Senior Lecturer at Imperial, and consultant cardiologist at Brompton, the U.K. lead investigator for both studies. Trials will be led by cardiologists and scientists at Brompton and Imperial, in collaboration with doctors at several U.K. hospitals including Royal Brompton’s Harefield Hospital in London, Papworth Hospital in Cambridge, and Scotland’s Golden Jubilee National Hospital. Avian influenza A H7N9 and its spread in China was the focus of a pair of recently published studies. Weeks before Shanghai officials on May 11 declared the end of their emergency response program for H7N9, because no new human cases had been reported over the previous 20 days, a team of Chinese researchers summarized preliminary findings of their investigations of the first reported cases, and of follow-up monitoring of close contacts of people with confirmed cases of the virus. The team analyzed data from H7N9 cases reported to the Chinese Center for Disease Control and Prevention as of April 17, and then published its findings April 24 on the website of The New England Journal of Medicine (Li et al., 2013). The study found that among 82 persons with confirmed H7N9 virus infection, a total of 17 people (21%) died after a median illness duration of 11 days, while an additional 60 remained critically ill; 4 were discharged from the hospital after treatment for clinically mild cases of avian flu; and 1 pediatric patient was not admitted to the hospital. The median age of people with confirmed H7N9 infection was 63 years, within a range of ages from 2 to 89 years. Of the 82 people with the infection, 73% were male, and 84% were urban residents. Animal exposure appeared to be a more significant factor in the disease than age. Of 77 persons for whom data were available, 4 were poultry workers, and 59 (77%) had a HUMAN GENE THERAPY 24:565–567 (June 2013) a Mary Ann Liebert, Inc. DOI: 10.1089/hum.2013. 2507