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disabling a gene to remove a dominant mutant or to engender infection resistance, such as HIV by deletion of the HIV receptor on CD4 cells.
Hemophilia B can be treated with the protein clotting factor, Factor IX, but may require bi-weekly injection at an annual cost of $100,000 to $200,000. Preliminary results from an ongoing trial suggest single administration of a gene therapy, delivered by peripheral intravenous injection, may lead to sustained therapeutic levels of Factor IX, without the need for protein prophylaxis. This suggests a pharmacoeconomic rationale for gene therapy; reduced health care cost, space for reimbursement, perhaps eventual availability in the developing world.
These advances are leading to more positive media commentary and are attracting attention from large Pharma and biotech companies. Last year a GlaxoSmithKline initiative on rare diseases drove their collaboration with the Telethon Institute in Milan, Italy to advance gene therapy into Phase III testing for rare diseases including immune deficiencies. This year, Amgen acquired Woburn, MA-based Biovex because of their oncolytic virus for glioma that showed impressive results in Phase II testing and is now advancing in Phase III trials. This week, Shire announced a new initiative in gene therapy and stem cells for rare diseases.
A few years ago, public interest and support for gene and cell therapy waned, in connection with negative publicity from a few severe adverse events. Investors came to view the field as moribund. The sentiment was exacerbated by the perceived risk, long product development timelines and liquidity concerns. They lost heart. Consequently, a severe adverse event was inflicted on this segment of the biotech industry.
With the exciting results accruing in the clinic and the strong interest from Big Pharma and biotech players, hopefully investors will not be far behind. Meanwhile, the old adage that “success builds success” is still true. To drive this field forward we need continued translation to clinical success and this is a primary mission and vision of the ASGCT.
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