Final Program - American Society of Gene & Cell Therapy

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Program Schedule, Thursday, May 16, 2013 J. Fraser Wright, PhD AAV Vectors for Clinical Trials: Challenges, and Strategies for Success Recombinant viral vectors based on adeno-associated viruses (AAV) have shown tremendous promise for clinical therapeutic gene delivery, and an acceleration of gene-based product development programs has been catalyzed by recent clinical successes. This presentation will describe strategies for advancing AAV-based investigational product chemistry, manufacturing and controls to effectively support the translational pathway from IND-supporting pre-clinical studies through clinical studies. An important requirement for successful translational research includes well deined manufacturing processes capable of reliable and reproducible yields vector of high purity, potency and safety. Another critical requirement is the set of analytical methods required to ensure comprehensive investigational product characterization and quality control testing. In this presentation the vector manufacturing processes and characterization methods used to support AAV vector-based translational research supported by GTRP will be presented. Kenneth Cornetta, MD Lentiviral Vectors: Can We Move Past Phase I/II Lentiviral vectors have advanced into early Phase clinical trials in the setting of genetic diseases, cancer, and other applications where integration and long-term expression is required. Compared to traditional integrating vectors, the methods used in manufacturing lentiviral vectors are well suited to rapid implementation into Phase I clinical trials. Unfortunately, this property can present challenges to generating a licensed product. This talk will discuss the current status of lentiviral vectors and highlight the challenges of moving these vectors from research tools to an FDA approved agent. Susan B. Sepelak, MS, MSM Regulatory Requirements: Navigation is Easier with Assistance The GTRP Clinical Coordinating Center provides assistance to investigators in navigating the regulatory processes in translational gene therapy research. For approved requests, assistance may be provided directly to the Investigator - assistance ranging from help with scheduling pre-preIND meetings with the FDA through organization, compilation and submission of IND applications. Regulatory support with clinical site-related issues may also be requested by Investigators to help site staff and Study Coordinators prepare for study implementation and manage ongoing regulatory requirements. Investigators who receive clinical trial funding assistance through the GTRP may, additionally, apply for support with site selection, study monitoring, and preparation of NHLBIrequired study reports. Thursday, May 16, 2013 Lunch Break (On Own - Not Provided; Pre-ordered boxed lunches available for pick up at Registration Counter) 12:30 pm - 2:00 pm Meet-the-Investigator Lunch Session 220 12:30 pm - 2:00 pm ROOM: 251 E New Frontiers – Hemoglobinopathies SPEAKERS Donald B. Kohn, MD Punam Malik, MD, PhD Meet-the-Investigator Lunch Session 221 12:30 pm - 2:00 pm MEET AT THE SOUTH ENTRANCE OF THE SALT PALACE CONVENTION CENTER Huntsman Cancer Institute Tour Boxed lunches will be available at the South Entrance shortly before departure to the Huntsman Cancer Institute. Final Program SALT LAKE CITY, UTAH May 15–18, 2013 51
Program Schedule, Thursday, May 16, 2013 Oral Abstract Session 230 2:00 pm - 4:00 pm ROOM: BALLROOM A Clinical Gene & Cell Therapy CO-CHAIRS: Carl H. June, MD and Harry L. Malech, MD (Abstracts 57 through 62; see page 92) Oral Abstract Session 231 2:00 pm - 4:00 pm ROOM: BALLROOM B RNA Virus Vectors CO-CHAIRS: Brian D. Brown, PhD and Jennifer E. Adair, PhD (Abstracts 63 through 70; see pages 92-93) Thursday, May 16, 2013 Oral Abstract Session 232 2:00 pm - 4:00 pm ROOM: BALLROOM C AAV Vector Biology CO-CHAIRS: Casey A. Maguire, PhD and David V. Schaffer, PhD (Abstracts 71 through 78; see page 93) Oral Abstract Session 233 2:00 pm - 4:00 pm ROOM: BALLROOM D Stem Cell Engineering and Therapy CO-CHAIRS: Hans-Peter Kiem, MD, FACP and Eirini P. Papapetrou, MD, PhD (Abstracts 80 through 86; Abstract 79 Withdrawn and Abstract 470 moved from Poster Session II; see pages 93-94) Oral Abstract Session 234 2:00 pm - 4:00 pm ROOM: 150 DEFG Musculo-skeletal Gene & Cell Therapy I CO-CHAIRS: Charles A. Gersbach, PhD and Louise R. Rodino-Klapac, PhD (Abstracts 87 through 94; see pages 94-95) Oral Abstract Session 235 2:00 pm - 3:00 pm ROOM: 150 ABC Cardiovascular Gene & Cell Therapy CO-CHAIRS: Dongsheng Duan, PhD and Joseph E. Rabinowitz, PhD (Abstracts 95 through 98; see page 95) 52 Final Program SALT LAKE CITY, UTAH May 15–18, 2013
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Page 18 and 19: Faculty List Thomas W. Chalberg, Jr
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