Category Archives: Gene Therapy

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 →

Juvenile Arthritis report gene therapy approach educational and entertainment channel in the field of orthopedic surgery and sometimes other surgical or medical fields. By: Adham Ortho Channel (AOC). … Continue reading →

January 15, 2015 1:50 pm by Meghana Keshavan | 0 Comments MedCity News Bluebird Bios work has beenatthe epicenter of gene therapy, working on a litany of fields that intersect with the work of the industrys top firms. This slide from its J.P. Morgan presentation this week illustrates that quite nicely: The companys stock has just killed it in the past year from $BLUE trading around $17 per share a year ago to hitting highs above $100 this year. Biopharma Dive wrote of the #JPM15 presentation: Its one of the hottest biotechsin the world, and its planning on presenting major clinical data by the end of the year. bluebird bio CEONick Leschly told JPM15 attendees that the company will unveil data for its ambitious sickle cell gene therapy platform LentiGlobinin 2015. Worth watching, if you havent, is CEO Nick Leschlys TEDxBoston talk from a couple years ago. Get our daily newsletter or follow us. Please enter your email below: Read more: Just a cool #JPM15 Bluebird Bio slide that shows its impressive scope … Continue reading →

Worlds First Gene Therapy Trial To Cure Deafness - Nov 2014 DENVER - A local man is a making medical history. Rob Gerk is the first person in the world to receive gene therapy to restore his hearing. "It was very inte... By: sixesfullofnines … Continue reading →

Gene therapy for cancer and other diseases The recent two decades have seen dramatic progress in gene therapy, both scientifically and socially. We are now able to treat several single gene disorders, decreasing disease and saving lives,... By: Oxford University Department for Continuing Education … Continue reading →

Gene Therapy To Restore Hearing Loss - Nov 2014 KANSAS CITY, Kan. Restoring hearing with a drug is the goal of a metro researcher. Now he's begun the first study in humans of a gene therapy that could help people hear again. Rob Gerk... By: sixesfullofnines … Continue reading →

4 Gene Therapy By: guj studyboard … Continue reading →

HOPE, HYPE REALITY: SCIENTIFIC RATIONALE FOR GENOME EDITING Why we consider gene therapy to be a viable means to control or cure HIV. By: defeatHIV … Continue reading →