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...
May 18, 2016 |
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.
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...
March 9, 2016 |
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.
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.
February 8, 2016 |
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.
January 4, 2016 |
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.
October 12, 2015 |
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.
September 19, 2015 |
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.
February 11, 2015 |
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.