FINAL PROGRAM - American Society of Gene & Cell Therapy

13 th AnnUAL MEETing | Washington, DC USA May 19-22, 2010 47Program ScheduleCatherine McKeon, PhDPThe NIDDK Molecular Therapy Centers and Cell Therapy ProgramThis presentation will cover the NIDDK Molecular Therapy Centers and the Cell Therapy Program, as well as some other programs that offer resources to investigators.David G. Badman, PhDThe NIH Rapid Access to Interventional Development Program (NIH-RAID)The NIH Roadmap has established the NIH-RAID Program (Rapid Access to Interventional Development), to make available, on a competitive basis, critical resourcesneeded for development of new small molecule or natural product-derived therapeutic agents. This program, part of the Translational Research component of Reengineeringthe Clinical Research Enterprise, uses resources of the National Cancer Institute’s (NCI) Developmental Therapeutics Program and the National Heart Lung and BloodInstitute’s (NHLBI) Gene Therapy Resource Program. Services provided depend upon the project and strength of the preliminary data. Services potentially available include:bulk supply, GMP manufacturing, formulation, assay development suitable for pharmacokinetic testing, and animal toxicology. Assistance also can be provided in the regulatoryprocess. Currently, animal efficacy studies are not supported; neither are syntheses nor formulation of recombinant proteins or monoclonal antibodies. However, theNIH-RAID Pilot will consider requests for services to support later-stage preclinical development of these entities. Manufacture of certain gene vectors (non-GMP viral andnon-viral gene vectors and GMP-grade adeno-associated virus and lentivirus vectors) can be requested, and preclinical studies of vectors may be supported. Additionally, theNIH-RAID Pilot will now consider requests for manufacture of small molecule or natural product material for any clinical study. Proposals must originate from academic ornon-profit investigators, as well as companies eligible for Small Business Innovation Research Program (SBIR). Collaboration with larger industry partners is encouraged.Roy S. Wu, PhDThe NCI Experimental Therapeutics Program (NExT) and the Chemical Biology Consortium (CBC)The NCI has recently consolidated their drug discovery and development efforts into a single pipeline for increased timeliness and efficiency and with an emphasis onunmet clinical needs. Researchers in academia, government or industry, either nationally or internationally, may apply. NCI is committed to moving high-priority discoveryand development projects through to proof-of-concept clinical trials in a partnership relationship. NExT is not a grant or contract mechanism of support. Rather, projectswill be evaluated and ranked by a peer committee followed by internal committees that will assess clinical need, develop project budgets and milestones with go/no godecisions, and provide access to the NCI’s drug discovery and preclinical and clinical development resources at no cost to the applicant. The resources include activities suchas production of pharmaceutical-grade material, development of clinical formulations, and first-in-human studies. The discovery arm of NExT, known as the NCI ChemicalBiology Consortium or CBC, is a collaborative network of 12 Centers that provide access to high-throughput screens and chemistry to optimize compounds for human use.Applications for potential NExT projects are accepted online four times a year. Visit http://next.cancer.gov/ for application instructions and more information.Danilo A. Tagle, PhDThe NINDS Translational Research ProgramThe NINDS Translational Research Program is specifically focused on pre-clinical therapy development necessary to begin clinical testing of therapeutic candidates forneurological disorders. This program supports the pre-clinical development of drugs, biologics, and devices including activities that lead to Investigational New Drug (IND)or Investigational Device Exemptions (IDE) applications to the Food and Drug Administration (FDA). Supported projects can include lead optimization, pre-clinical efficacytesting, predictive ADMET (absorption, distribution, metabolism, excretion, and toxicology) testing, formulation, manufacture, pharmacology, toxicology, and IND or IDEsubmission. For entry to the program, projects must have one or more identified therapeutic leads and convincing proof-of-principle on efficacy demonstrated in crediblemodels of the disease or against defined disease targets.Thursday, May 20 thLisbeth Welniak, PhDProduction Assistance for Cellular Therapies (PACT)The PACT Group is an NHLBI-funded initiative that is committed to the advancement of effective cell therapies. This program was renewed in January, 2010 for a secondfive-year period of funding. PACT supports the development of novel somatic cell therapy products by providing production assistance to the cell therapy community, aswell as educational training via web seminars and at meetings. Production assistance is provided through the funded contract manufacturing of therapeutic cell products atPACT processing facilities. These facilities offer scale-up capability and expertise in the translational development needed to produce cell therapy products under cGMP andcGTP regulations. PACT’s educational training focuses on three general areas: translational development/scale-up and manufacture of cell therapy products; and qualityassurance and regulatory issues. The objective of the PACT program is to continue to advance cellular therapy research in the areas of regeneration of damaged/diseasedtissues, organs, and biologic systems, and targeted treatments for serious diseases without effective therapies. The cell processing facilities will support translationalresearch in novel cell therapies to advance the field of cellular therapy and to enable scientifically meritorious basic science research to reach the clinical trial stage. Pleasevisit the PACT website at www.pactgroup.net for more information regarding the PACT program.