Avax to Develop Gene Therapy
For Immune-Deficient Children
By ANTONIO REGALADO
Staff Reporter of THE WALL STREET JOURNAL
Avax Technologies Inc. said it plans to pursue commercial development of a successful gene-correcting treatment for immune-deficient children.
The treatment appears to have permanently cured several children of severe combined immunodeficiency, or SCID, a rare disease that leaves them vulnerable to infections. The procedure is based on research by French scientists and works by adding functioning DNA to bone-marrow cells.
Richard Raimey, vice president for finance and administration at Avax , said the company believes it already has enough data to approach regulatory agencies in Europe and the U.S. to win a green light for marketing its treatment. "It has a high probability of being the first gene therapy approved for human application," predicted Mr. Raimey.
Gene-therapy treatments have proved tricky to develop, with some failing spectacularly and none yet approved for commercial use. According to the National Institutes of Health in Bethesda, Md., nearly 400 gene-therapy trials are under way in the U.S.
Last April, the Food and Drug Administration gave so-called orphan drug status to the Overland Park, Kan., company's SCID-treatment. The designation, given for rare diseases where there is limited financial incentive to develop treatments, would provide the company several years of market exclusivity, as well as certain tax and other credits, if it is first to win marketing approval.
The form of SCID targeted by Avax is extremely rare, affecting perhaps only 100 children a year in the U.S. SCID is an inherited gene disorder that leaves children without a functioning immune system, and usually causes death within a year if left untreated. The condition is sometimes known as the "bubble-boy" syndrome after a Texas patient who lived for a period inside a sterile chamber. |
