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Gene Therapy

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LIVING
February 7, 2000 | By Faye Flam, INQUIRER STAFF WRITER
Perhaps nothing has become as synonymous with futuristic medicine as gene therapy. But, as brought home by the recent death of a young research subject at the University of Pennsylvania, the dangers are still not well understood. The investigation into the death of 18-year-old Jesse Gelsinger revealed that it probably wasn't the genes he was getting that caused his death. Rather it appears that the death was the result of the delivery vehicle for those genes - in this case a virus modified so it wouldn't cause an infection.
BUSINESS
October 12, 2015 | By David Sell, Inquirer Staff Writer
Philadelphia's gene therapy community last week marked another milestone in its resurgence when locally based Spark Therapeutics said its most advanced product helped restore some vision in patients suffering from a rare eye condition during a clinical trial. The company plans to apply for FDA approval next year. Philadelphia was in some minds the gene therapy hub in the late 1990s, until an 18-year-old man died during a clinical trial by University of Pennsylvania researcher James M. Wilson.
NEWS
January 7, 1996 | By Daniel S. Greenberg
We have entered a new era of super-hyped science, personal glory seeking, and overnight millionaires from biotech speculation - the sort of atmosphere that leads to the "snake-oil" overselling of medical treatments. Now comes a disturbing indictment of the most publicized and promising field of medical science, gene therapy. That's the hopeful term for treating cancer, cystic fibrosis and other diseases by replacing defective or missing genes. It comes from a committee of physicians and scientists appointed by the National Institutes of Health (NIH)
BUSINESS
March 9, 2016 | By Linda Loyd, Staff Writer
Philadelphia drugmaker Spark Therapeutics Inc. has acquired for $15.1 million a private, Ireland-based gene-therapy company. Spark, spun out of Children's Hospital of Philadelphia, said Monday that it bought Genable Technologies Ltd. for $6 million in cash and 265,000 shares of Spark stock, valued at $9.1 million based on Friday's closing price. Additional financial terms were not disclosed. Spark said Genable's potential treatment, RhoNova, will target a common form of a rare inherited retinal disease, which impacts about 30,000 people worldwide.
NEWS
May 10, 2012 | By Marie McCullough, Inquirer Staff Writer
For a quarter of a century, gene therapy has been stymied, largely because the patient's immune system attacks the treatment as a suspected rogue - or because it actually does turn rogue. Now, University of Pennsylvania researchers have convincingly shown that they can overcome these formidable obstacles. Cells that were genetically modified to fight HIV have persisted for up to 11 years - and counting - without bad side effects in 41 patients. In two other patients, the modified cells were safe but not as durable, according to the Penn study, published last week in Science Translational Medicine.
NEWS
January 1, 1999 | By Shankar Vedantam, INQUIRER STAFF WRITER
In a development that may overcome a major stumbling block to gene therapy, scientists at the University of Pennsylvania Medical Center have figured out how to turn the treatment gene on and off in mice and monkeys. Such control over therapy is crucial. Too much medicine can be as dangerous as the disease itself. "Ideally what you want to be able to do is to turn the gene on when you want to and switch it off when you don't want it," said French Anderson, director of the gene-therapy laboratories at the University of Southern California.
NEWS
March 28, 2000 | By Shankar Vedantam, INQUIRER STAFF WRITER
In another attempt to reopen its stalled gene-therapy clinical trials, the University of Pennsylvania again defended its conduct of a trial that caused the death of an Arizona teenager last year. In response to a March 3 warning letter from the federal Food and Drug Administration that detailed a number of problems with the trial, Penn and its Institute for Human Gene Therapy yesterday said numerous measures were underway to prevent the problems from reoccurring. In a 14-page letter to the FDA, the institute, led by scientist James M. Wilson, said it had contracted with an independent company, Parexel International Corp.
NEWS
April 19, 1991 | By Jim Detjen, Inquirer Staff Writer
Scientists have developed a genetic technique that may make it possible to treat people suffering from cystic fibrosis and other deadly lung diseases by spraying aerosols into their throats. The treatment represents a dramatic advance in the exploding field of gene therapy - the altering of genetic material in a patient's cells. In a study being published today in the journal Science, researchers said they used the treatment to transfer a human gene into the lung cells of rats.
NEWS
December 27, 1995 | By Donald C. Drake, INQUIRER STAFF WRITER
Donald appeared much more relaxed than the researchers watching from the edges of the operating room as nurses and anesthesiologists readied him for surgery. He was about to undergo a revolutionary cancer treatment, and the scientists had reason to be tense. They were trying a new form of gene therapy and didn't know how dangerous it was or whether it would work. Donald, 64, was a retired asbestos worker with less than two years to live. He was being killed by mesothelioma, a rare cancer that attacks the membranes covering the lungs and the opposing chest wall.
NEWS
March 3, 2011 | By Marie McCullough, Inquirer Staff Writer
In a feat that is renewing hopes for conquering AIDS, researchers have genetically engineered patients' vital immune cells to make them resistant to HIV infection. To confer this invulnerability, scientists took the immune cells from HIV-positive patients' own blood, then snipped out a single gene - the first time such a precise alteration has been achieved on a meaningful scale. When put back in the patient, the cells no longer make a receptor that HIV needs to enter the cell, effectively blocking the virus.
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ARTICLES BY DATE
NEWS
May 29, 2016
The Gene An Intimate History By Siddhartha Mukherjee Scribner. 592 pp. $30 Reviewed by Jim Higgins 'Like Pythagoras' triangle, like the cave paintings at Lascaux, like the Pyramids in Giza, like the image of a fragile blue planet seen from outer space, the double helix of DNA is an iconic image, etched permanently into human history and memory," Siddhartha Mukherjee writes in The Gene: An Intimate History...
BUSINESS
May 18, 2016 | By Harold Brubaker, STAFF WRITER
Biogen Inc. announced a research collaboration on gene therapy and gene editing with the University of Pennsylvania that will pay the West Philadelphia institution $20 million upfront, with an additional $62.5 million committed over the next three to five years for research and development costs. Biogen, a biotechnology firm based in Cambridge, Mass., said the collaboration with Penn professors James Wilson and Jean Bennett, will focus primarily on therapies for diseases that target the eyes, skeletal muscles, and the central nervious system.
BUSINESS
March 14, 2016
Maybe long term, gene therapy might derail a business like the one Paul Perreault leads in King of Prussia and Australia. After all, gene therapy, pursued by Philadelphia-based Spark Therapeutics and others, involves curing chronic conditions such as the bleeding disorder hemophilia by discovering and replacing the defective gene that causes it. Perreault, 59, is chief executive officer and managing director of CSL Ltd., a $5.5 billion global...
BUSINESS
March 9, 2016 | By Linda Loyd, Staff Writer
Philadelphia drugmaker Spark Therapeutics Inc. has acquired for $15.1 million a private, Ireland-based gene-therapy company. Spark, spun out of Children's Hospital of Philadelphia, said Monday that it bought Genable Technologies Ltd. for $6 million in cash and 265,000 shares of Spark stock, valued at $9.1 million based on Friday's closing price. Additional financial terms were not disclosed. Spark said Genable's potential treatment, RhoNova, will target a common form of a rare inherited retinal disease, which impacts about 30,000 people worldwide.
BUSINESS
February 28, 2016
The photo is adorable. Little Madeline, age 3, smiles happily next to her family's tinseled Christmas tree, hand plunged into her Christmas stocking, clearly pleased with the white nurse's cap perched on her head. Now Madeline Bell, 54, with a nursing degree, heads one of the world's largest health enterprises, the Children's Hospital of Philadelphia, where she presides over a $2.5 billion budget and 11,552 employees. In July, Bell, after eight years as CHOP's chief operating officer, replaced longtime chief executive Steven Altschuler, a pediatrician and researcher.
NEWS
February 8, 2016 | By Bonnie L. Cook, Staff Writer
Dr. Robert J. Suhadolnik, 90, of Roslyn, a biochemist renowned for his research on AIDS and chronic fatigue syndrome, died Tuesday, Jan. 26, at Abington Memorial Hospital. Through much of his career, Dr. Suhadolnik was a professor in the biochemistry department at Temple University School of Medicine, now the Lewis Katz School of Medicine. From a laboratory there, he and a half-dozen colleagues spent decades analyzing irregularities in immune-system pathways in patients with those conditions.
NEWS
January 4, 2016 | By Sandy Bauers, For The Inquirer
What will 2016 bring in the way of medical advances? As president and CEO of Philadelphia's University City Science Center, an incubator of medical research, Stephen Tang has an uncommon vantage point on that question. He predicts gene therapy, an experimental technique that uses genes to treat or prevent disease, and health information technology will boom this year. He spoke to us recently about the center and what lies ahead.   Tell us more about the Science Center.
BUSINESS
October 12, 2015 | By David Sell, Inquirer Staff Writer
Philadelphia's gene therapy community last week marked another milestone in its resurgence when locally based Spark Therapeutics said its most advanced product helped restore some vision in patients suffering from a rare eye condition during a clinical trial. The company plans to apply for FDA approval next year. Philadelphia was in some minds the gene therapy hub in the late 1990s, until an 18-year-old man died during a clinical trial by University of Pennsylvania researcher James M. Wilson.
BUSINESS
September 19, 2015 | By David Sell, Inquirer Staff Writer
Hoping to ride the recent biotechnology stock boom, Regenxbio Inc., a gene-therapy firm with local ties, began selling shares publicly on the NASDAQ stock market Thursday. When the IPO was priced on Wednesday, the company sold 6.3 million shares at $22 through underwriters, thereby raising $138.6 million. Regenxbio was cofounded by University of Pennsylvania researcher James M. Wilson, whose 1999 clinical trial ended with the death of an 18-year-old patient. Besides anguish for his family, Jesse Gelsinger's death set back gene-therapy research for at least a decade.
BUSINESS
February 11, 2015 | By David Sell, Inquirer Staff Writer
Venture-capital funding of drug research and development has shifted in recent years to specialty and rare-disease medicines, according to a report released Monday by BIO, the biotechnology trade organization. Those medicines often carry high price tags that insurers have thus far fought less than some other drugs because there are no or few alternatives. "VCs will pull back from areas that are seen as having unfavorable or unpredictable regulatory and reimbursement hurdles," the BIO report said.
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