Gene Therapy: Hope For The Blind? In medicine, doctors often talk about treatments, but rarely about cures. Now, gene therapy is offering the potential to actually cure certain diseases and an experimental breakthrough could... By: NewsChannel 5 … Continue reading →

Gene therapy is a promising strategy to correct hereditary disorders. The approach takes advantage of viral vectors to deliver a corrected version of the mutated gene. Adeno-associated virus (AAV) has many features that make it a favorable vector for gene therapy. In animal models, AAV-mediated gene delivery is generally regarded as safe and has demonstrated efficacy for some genetic diseases. However, a recent study reported an increase in liver cancer in mice after AAV gene therapy. A new publication in the Journal of Clinical Investigation reveals that AAV vector design influences the likelihood of developing cancer in the liver. Charles Venditti and colleagues at the National Institutes of Health looked for the development of hepatocellular carcinoma (HCC) in a large number of mice that had received AAV gene therapy. HCC was associated with the AAV vector integrating within a specific site in the genome and inducing expression of microRNAs and a retrotranposon. Moreover, AAV dose, the choice of enhancer/promoter, and timing of delivery all influenced the HCC incidence. The results of this study provide insight into features that should be considered when designing AAV vectors for gene therapy. ### TITLE: Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy … Continue reading →

Pre-clinical studies in mice reveal ways to reduce cancer risk with modified treatment National Institutes of Health researchers have uncovered a key factor in understanding the elevated cancer risk associated with gene therapy. They conducted research on mice with a rare disease similar to one in humans, hoping their findings may eventually help improve gene therapy for humans. Researchers at the National Human Genome Research Institute (NHGRI), part of NIH, published their research in the Jan. 20, 2015, online issue of the Journal of Clinical Investigation. "Effective and safe gene therapies have the potential to dramatically reverse diseases that are life-threatening for affected children," said NHGRI Scientific Director Dan Kastner, M.D., Ph.D. "This study is an important step in developing gene therapies that can be safely used to benefit patients." Toxic side effects actually are rarely observed by researchers who have designed gene therapies using an adeno-associated virus (AAV) as a vector to deliver the corrected gene to a specific point in the cell's DNA. AAVs are small viruses that infect humans but do not cause disease. A vector is a DNA molecule of AAV used as a vehicle to carry corrected genetic material into a cell. AAV viruses are … Continue reading →

Spark Therapeutics, which is developing gene therapy treatments for retinal dystrophies and hematologic disorders, announced terms for its IPO on Tuesday. The Philadelphia, PA-based company plans to raise $88 million by offering 5.5 million shares at a price range of $15 to $17. At the midpoint of the proposed range, Spark Therapeutics would command a fully diluted market value of $378 million. Spark Therapeutics, which was founded in 2013, plans to list on the NASDAQ under the symbol ONCE. J.P. Morgan and Credit Suisse are the joint bookrunners on the deal. It is expected to price during the week of January 26, 2015. Investment Disclosure: The information and opinions expressed herein were prepared by Renaissance Capital's research analysts and do not constitute an offer to buy or sell any security. Renaissance Capital, the Renaissance IPO ETF (symbol: IPO) or the Global IPO Fund (symbol: IPOSX) , may have investments in securities of companies mentioned. The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of The NASDAQ OMX Group, Inc. Originally posted here: Gene therapy biotech Spark Therapeutics sets terms for $88 million IPO … Continue reading →

Contact Information Available for logged-in reporters only Newswise PHILADELPHIA The University of Pennsylvania has announced an expanded relationship with Spark Therapeutics, a late-stage gene therapy company developing treatments for debilitating, genetic diseases. As part of the expanded relationship, which included both an exclusive license agreement to certain Penn-owned intellectual property rights and a clinical trial agreement, Penn will be one of the clinical sites for a clinical trial to evaluate the treatment of a rare genetic form of progressive blindness. This expanded strategic relationship between the two organizations is representative of Penns strong commitment towards direct engagement with the private sector to advance promising technologies, said John Swartley, Associate Vice Provost for Research and Executive Director, Penn Center for Innovation (PCI). When Penn researchers on the cutting edge of their fields are able to partner effectively with innovators in the private sector it has the potential to accelerate the advancement of exciting new approaches for the treatment of disease. PCI serves as the University of Pennsylvanias commercialization center and actively works with the private sector to foster research and development collaborations leveraging Penn technology and research. Spark today announced the start of Phase 1/2 clinical trial for patients with choroideremia … Continue reading →