2013-01-28 05:34:54 - Professor M. Ian Phillips, Norris Life Professor and Director of the Center for Rare Disease Therapies at the Keck Graduate Institute, will present about gene and stem cell therapies at the Inaugural Orphan Drugs Research and Commercialization Conference, held May 8-10, 2013 in Boston, MA by GTC.
Nearly 400 new orphan drugs have been approved for marketing since the Orphan Drug Act of 1983. Despite this, currently only 5% of the approximately 7,000 rare diseases have therapies. The advanced platforms of gene therapy and stem cell therapy, or a combination of both, offer new approaches to cell therapies for rare intractable diseases that are currently untreatable. Gene therapy is for repairing dysfunctional cells, while stem cell therapy is for regenerating functional cells.
Gene therapies involve the correction of defective genes by the use of viral vectors. Adeno-associated virus and lentivirus are dominating the field. They are being tested for the treatment of diseases caused by the failure of a single gene, in cystic fibrosis, various forms of blindness,
hemophilia, lipase protein deficiency, adrenoleukodystrophy and other autoimmune diseases. In theory, if the dysfunctional gene is replaced with the correct healthy gene, a cure can be achieved. This raises questions of how much to charge for a one shot treatment that can cure a disease, and who will pay?
In November 2012, The European company UniQure, made headlines when its medication Glybera became the first gene therapy approved by regulatory authorities in the Western world. Alexion has developed a post gene translation therapy of providing chaperone molecules to prevent misfolding of gene products. How do these developments affect the research and commercialization of gene and stem cell treatments? What is the real promise of such therapies in treating rare diseases, and what is mere talk?
Dr. Phillips is a Norris Professor of Applied Life Sciences, Faculty Director for the PreMed Program and the Founding Director of the Center for Rare Disease Therapies at the Keck Graduate Institute in Southern California. He was previously a consultant for Merck, Bristol Myers-Squibb and Hoechst. He was Chairman of Physiology at the University of Florida, where he established a modern gene-oriented Department of Physiology and founded the Division of Functional Genomics.
Now currently at KGI, he is pursuing his stem cell therapy studies full time and directs the Center for Rare Disease Therapies, which has many research activities including the search for drugs that can be developed to treat rare diseases. The Center runs workshops with the FDA, projects sponsored by drug companies, and patient advocate groups specializing in rare diseases.
The conference is part of the Drug Discovery Summit 2013, and will run parallel with three other exciting conferences:
3rd Epigenetics in Drug Discovery
6th Imaging in Drug Discovery and Development
7th Drug Design and Medicinal Chemistry