A Media Event on Clinical Developments in Gene and Cell Therapy

Milwaukee, WI (PRWEB) May 02, 2012

Academic and industry leaders in gene and cell therapy will be featured at a Media Event in Philadelphia, PA on May 15, 2012, immediately preceding the 15th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) on May 16-19, 2012. The event will focus on recent clinical developments in targeted biotherapeutics for various diseases.

Members of the media will also receive complimentary full-access registration to the ASGCT 15th Annual Meeting at the Pennsylvania Convention. To complete your registration to the Media Event and the Annual Meeting, please visit the ASGCT website.

2:00 pm 2:20 pm: Gene and cell-based immunotherapy for cancer Many cancers are known to acquire the ability to suppress anti-tumor immune responses in the host. The genetically modified cells developed and used in this clinical trial are designed to reawaken immune cells that have been suppressed by the leukemia and stimulate the generation of so-called memory T cells, which can provide ongoing protection against recurrence. Although long-term effectiveness of this novel treatment is not yet known, the doctors have found that months after infusion, the new cells had multiplied and continued their seek-and-destroy mission against cancerous cells throughout the patients bodies. The new paradigm provides a tumor-attack roadmap for the treatment of other cancers including lung, myeloma, ovarian cancer and melanoma.

Carl June, MD is currently the Director of Translational Research at the Abramson Cancer Center,, an Investigator of the Abramson Family Cancer Research Institute, and a tenured Professor of Pathology and Laboratory Medicine at the Perelman School of Medicine of the University of Pennsylvania.

2:20 pm 2:30 pm: Question and answer period

2:30 pm 2:50 pm: Gene therapy for hemophilia B Hemophilia B is an X-linked bleeding disorder caused by a deficiency of blood coagulation Factor IX, and patients need to be infused with the newly developed long-acting protein concentrates twice a month. Advances in gene therapy using intravenous infusion of an AAV vector expressing human Factor IX in a clinical trial on hemophilia patients have significantly improved circulation of Factor IX levels that transformed their disease from severe hemophilia to mild hemophilia. Other strategies are also under development that may confer the ability to correct the disease permanently by gene therapy.

Katherine High, MD is the William H. Bennett Professor of Pediatrics at the University of Pennsylvania School of Medicine, an Investigator of the Howard Hughes Medical Institute and Director of the Center for Cellular and Molecular Therapeutics at the Children Hospital of Philadelphia. Dr. High has pioneered safe and effective clinical translation of genetic therapies for inherited disorders including hemophilia B, and she is a Past President of the American Society of Gene and Cell Therapy.

2:50 3:00 pm: Question and answer period

3:00 pm 3:20 pm: Gene and cell-based therapy for severe combined immunodeficiency Severe combined immunodeficiency (ADA-SCID, a.k.a. bubble boy disease) is a rare disorder of immunity and without treatment; children affected with ADA-SCID will succumb to infections within the first two years of life. Over the past years >40 patients have been treated with gene-corrected hematopoietic stem cell transplantation, which has led to the regeneration of immune cells and the reconstitution of immune function in >70% of the patients. While hematopoietic stem cell transplantation from a matched donor remains the therapy of choice for ADA-SCID, the results obtained by the current clinical trials have indicated that gene and cell therapy should be considered as the first treatment option in the absence of a matched donor.

Read the original here:
A Media Event on Clinical Developments in Gene and Cell Therapy