ASGCT Press Release

ASGCT 13th Annual Meeting:
Gene Therapy Breakthroughs Presented at Presidential Symposium

WASHINGTON – Breakthroughs in gene therapy are improving the ability to treat patients with rare inherited diseases, including certain types of congenital blindness and anemia. Pioneering researchers will discuss their advances during the Presidential Symposium at the American Society of Gene & Cell Therapy 13th Annual Meeting.

The Presidential Symposium begins at 4:30 p.m. Friday, May 21.

An Aye for Gene Therapy: Lessons from a Clinical Trial for Congenital Blindness
Gene therapy has restored sight in patients with Leber’s Congenital Amaurosis (LCA), an inherited blindness that begins at birth.

Jean Bennett, MD, PhD, the lead researcher on a study at The Children’s Hospital of Philadelphia, will share new safety and efficacy results from 12 patients, up to 2.5 years post-treatment.

All of the children enrolled in the study are now able to navigate independently, read and write, participate in normal classroom activities, and participate in sports. Adults in the study also have more independence due to improvements in their vision.

This the first pediatric human gene therapy study involving a non-lethal disease, and it is also the first gene therapy study of treatment of inherited retinal degeneration in which pediatric subjects have been enrolled.

Jean Bennett, MD, PhD, has led several trials of treatments for Leber’s Congenital Amaurosis over 18 years at the University of Pennsylvania.

New Approaches to Gene Therapy for Fanconi Anemia
Clinical studies show that patients with Fanconi anemia – a rare syndrome that leads to bone marrow failure and, often, cancer – lack enough functional blood stem cells for successful gene therapy to correct the defect.

David A. Williams, MD, has completed extensive research on methods for reprogramming standard cells to serve as substitutes for stem cells.

These induced pluripotent stem cells (iPS cells) are derived through a genetic modification and mimic the characteristics of genuine stem cells, including ability to duplicate. Pluripotent cells have the ability to differentiate and develop into any type of cell in the body.

David A. Williams, MD, is Leland Fikes Professor of Pediatrics, Harvard Medical School and past editor-in-chief of ASGCT's Journal, Molecular Therapy (2004 – 2009).

Members of the media interested in attending the ASGCT Annual Meeting should download the press registration form at www.asgct.org/am10.

The American Society of Gene & Cell Therapy (ASGCT) is a professional non-profit 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, www.asgct.org.

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