A Media Event on Clinical Developments in Gene and Cell Therapy

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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.

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.

Dr. Fabio Condotti, MD is a tenured faculty at the National Human Genome Research Institute where he currently leads the Disorders of Immunity Section of the Genetics and Molecular Biology Branch. Dr. Candotti’’s research and clinical interests are in the field of genetic defects of immunity with particular focus on adenosine deaminase deficiency, reticular dysgenesis and Wiskott-Aldrich syndrome He currently serves on the Board of Directors of the American Society of Gene and Cell Therapy.

3:20 – 3:30 pm: Question and answer period

3:30 pm – 4:00 pm: Coffee Break

4:00 pm – 4:20 pm: Gene therapy for vision disorders
Gene therapy has the potential to reverse disease or prevent further deterioration of vision in patients with incurable inherited retinal degeneration. In patients with Leber's Congenital Amaurosis due to mutations in the RPE65 gene, a single subretinal injection of an adeno-associated virus (AAV) vector expressing human RPE65 led to improved vision/retinal function. We have now demonstrated the recovery of retinal and visual function after re-administration of the vector to the second eye. Cortical responses are observed soon after gene delivery but take approximately three months to plateau. There does not appear to be an age limit to rescue, although the extent of disease- (and age-) related retinal degeneration influences characteristics of the responses.

Jean Bennett, MD, PhD is the F.M. Kirby Professor of Ophthalmology and Cell and Developmental Biology and a Senior Investigator in the F. M. Kirby Center for Molecular Ophthalmology at the University of Pennsylvania Perelman School of Medicine. Dr. Bennett has pioneered gene transfer and gene therapy strategies for eye diseases over the past two decades.

4:20 pm – 4:30 pm: Question and answer period

4:30 pm – 4:50 pm: Oncolytic virotherapy for cancer
Engineered animal viruses that selectively replicate in cancer cells but not in normal cells are under development as novel bio-therapeutics for a variety of solid tumors, which is collectively known as oncolytic virotherapy. We have designed a targeted and transgene-armed vaccinia virus product (JX-594) to combine attractive features of both oncolytic virus and gene therapy approaches with improved IV potency. The past year has shown two breakthroughs: 1) reproducible dose-dependent IV targeting of widespread metastases and 2) an overall survival benefit in a randomized Phase 2 dose-ranging trial in patients with advanced liver cancer. A Phase 2b trial is underway in end-stage liver cancer patients comparing survival with JX-594 vs placebo.

David Kirn, MD is the Founder and Chief Medical Officer of Jennerex Biotherapeutics in San Francisco, CA. He is a global leader in the clinical research and development of oncolytic viruses for cancer. He designed and led clinical development programs and trials with over 10 different agents, and with over 10 different companies and leading academic programs, involving hundreds of patients (Phase 1-3).

4:50 pm – 5:00 pm: Question and answer period

5:00 pm – 6:00 pm: Networking and interview opportunities with the speakers (light hors d’oevres)

The press event will take place in Salon K, located on the 5th floor of the Philadelphia Marriott Downtown. Please note that only registered members of the media will be admitted to the press event. Thank you for your understanding.

The American Society of Gene & Cell Therapy (ASGCT) is a professional nonprofit medical and scientific organization dedicated to the understanding, development and application of genetic and cellular therapies and the promotion of professional and public education in the field. For more information on ASGCT, visit its website, http://www.asgct.org.

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