Category Archives: Gene Therapy

JESSEGELSINGER- was a victim of a Gene Therapy Treatment that went dreadfully wrong. For the entirety of his life, Gelsinger suffered from a rare metabolic disorder called OTC, or ornithine transcarbamylase deficiency. In order to keep the symptoms of his disorder under control, Gelsinger was kept on a diet of low protein and took 32 drugs everyday. It was Gelsinger's desire to help find a cure for OTC for future generations, so he volunteered to undergo experimental gene therapy treatment, knowing that it would probably not personally benefit him. However, knowing that this prospective treatment existed, Gelsinger was given hop that one day he could be cured. Ironically, before his treatment occured at Penn Hospital in Philedelphia, he said to a friend,"What's the worst that can happen to me?"(Citation 2). However, this treamtment proved fatal for Gelsinger. He died on September 17th, 1999, being the first documented death as a result of gene therapy. Gelsinger's gene therapy treatment was comprised of a combination of adenovirus (diluted cold virus), which served as a vector, and healthy replacement genes for his malfunctioning ones.As a result of his treatment, Gelsinger's he suffered symptoms that had never been seen in other clinical tests that … Continue reading →

Spark Therapeutics, which is developing gene therapy treatments for retinal dystrophies and hematologic disorders, filed on Tuesday with the SEC to raise up to $86 million in an initial public offering. The Philadelphia, PA-based company, 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. No pricing terms were disclosed. 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. See the original post here: Gene therapy biotech Spark Therapeutics files for an $86 million IPO … Continue reading →

Rhys Evans life could have been very different. He could have been a bubble boy, forced to walk around in a protective see-through plastic canopy. You see, he was born with an immune system that barely worked. The slightest infection could have proved fatal. But Rhys is now 14years old and doing fine. So how did Rhys avoid living in a bubble? The simple answer is that Rhys got lucky his condition was diagnosed when he was a baby. Even more fortunately, doctors at Great Ormond Street Hospital were able to do something about it. They understood that Rhyss condition was caused by a genetic flaw and they thought that if they could correct this flaw then they could restore his immune system. That is exactly what happened, and why Rhys is now no different to any other young teenager. Rhyss treatment is an example of gene therapy, which was the subject of a fascinating lecture that I attended last month. Leonard Seymour, professor of gene therapies in the Department of Oncology at Oxford University, gave four reasons why 2014 has been a breakthrough year for this revolutionary, but controversial, approach. Let me begin by describing these successful trials. Rhys … Continue reading →

AADC Gene Therapy Trial Natural History Study. Dr Toni Pearson Dr Toni Pearson, Mount Sinai Medical Center - USA Gene Therapy Trial Natural History Study Summary: AADC Gene Therapy Trial. An update on the planning of t... By: aadcresearch … Continue reading →

Surgical Technique for AADC Gene Therapy. Prof John Heiss Prof. John Heiss, National Institute of Health NINDS - USA Surgical Technique for AADC Gene Therapy Summary: The history of delivering gene therapy to the central nervous system will be... By: aadcresearch … Continue reading →

Bioethics on Gene Therapy Talks about gene therapy the costs and benefits. By: cp87782 … Continue reading →

Gene Therapy AN NM Biology 1233 By: Angelica Nino … Continue reading →

In an interesting move, Pfizer ( PFE ) has struck a deal with Spark Therapeutics to establish a gene therapy platform. King's College Professor Michael Linden, who is an expert in gene therapy research, will lead the effort. Under the agreement, Pfizer will make an upfront payment of $20 million to Spark Therapeutics and $260 million in additional milestone-based payments. In return, Pfizer will handle late stage clinical trials, approval and commercialization of the product. Spark is currently investigating the efficacy of gene therapy forhemophilia B, and its program will enter early phase trials next year. Gene therapy has been under scientific research for over 2 decades, but viable therapies have yet to gain commercial acceptance due to safety and delivery-related issues. However, Pfizer's move and some other recent developments in the industry suggest that the therapy may be coming off age. There is another implication of this move. Even if successful, Pfizer is still a long way away from a commercialized gene-derived therapy. It is thus just a single step in its program to reverse its revenue decline in coming years. Other actions are possible, or even likely, including an outright acquisition of a substantially larger company, considering that … Continue reading →

Gene Therapy and Micro-needle Based Protein Therapy for RDEB - 2014 PCC The 2014 Patient Care Conference was held at the Gaylord Opryland Resort in Nashville, TN. DebRA invited those with EB as well as their families, advocates, ... By: DebraofUS … Continue reading →

ANN ARBOR (PRWEB) December 15, 2014 Gene therapy pioneer and longtime Veterans Affairs researcher Dr. David Fink received the 2014 Paul B. Magnuson Award from VA in a ceremony at the VA Ann Arbor Healthcare System on Dec. 15, 2014. Dr. Fink is a staff neurologist and an investigator with the Geriatric Research, Education and Clinical Center at the Ann Arbor VA. He is also the Robert Brear Professor and Chair of Neurology at the University of Michigan. He has been with VA since 1982. A Harvard Medical School graduate, Fink has pioneered methods to introduce genes into the body to treat chronic pain and other nervous-system diseases. His team led the first human clinical trial of gene therapy for pain. The phase 1 trial, published in the Annals of Internal Medicine in 2011, involved 10 cancer patients with severe pain who had failed to respond even to high doses of morphine or other pain drugs. Finks group gave them skin injections of an inactive form of the herpes simplex virus as a means to deliver a gene known as PENK. The gene helps the body produce an opioid-like molecule called proenkephalin. The gene treatment, based on years of research, … Continue reading →