Andra E. Miller, Ph.D PROFESSIONAL EXPERIENCE:

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1 Andra E. Miller, Ph.D PROFESSIONAL EXPERIENCE: Biologics Consulting Group, Inc, Alexandria, VA July 2000 to present Director, Cellular and Gene Therapies Provides consultation for the development of regulatory strategies to facilitate rapid development of cell, gene and regenerative therapies using a science based approach. Support for CMC submission, interpretation of FDA and NIH/RAC guidelines, pre-ind, IND and BLA preparation, phase I, II and III product development strategies and assessment of cgmp, cgtp and quality system compliance. Areas of experience include: somatic and adult stem cell products, viral and non-viral vectors, combination products, cartilage regeneration, wound healing, bone healing, spinal cord injury, and immunotherapy. Center for Biologics Evaluation and Research (CBER), FDA, Bethesda, MD 1993-July 2000 Expert Microbiologist and Gene Therapy Group Leader Division of Cellular and Gene Therapies Office of Therapeutics Expert knowledge of CBER regulations, recommendations and scientific principles relevant to somatic cell and gene therapies and cytokine product areas. Responsible for identification of policy issues, and development of industry guidance and regulation for cellular and gene therapies and related products. Proposed research questions, carried out analyses to improve current RCR recommendations for product testing and patient follow-up and authored new Guidance to Industry, which was published in Nov Represented CBER to the public and other government agencies as invited speaker at internationally and nationally sponsored scientific meetings and symposia. Served as agency representative (alternate) to Recombinant DNA Advisory Committee and as FDA liaison to Office of Biotechnology Activities, NIH. Responsible for review of pre-ind, IND and BLA materials with regard to completeness and consistency of the manufacturing process, characterization and safety testing of the product and scientific rationale. Laboratory of Molecular Pharmacology, CBER, FDA. Bethesda, MD National Research Council Fellow As a member of the Unit on Developmental Biology, research focused on the study of genes involved in specification of body pattern during the early stages of development in Xenopus laevis

2 Laboratory of Mammalian Genes and Development, NICHD, NIH. Bethesda, MD Using a transgenic mouse model, studied the effect of cytokines and mitogens on the activation of immunoglobulin gene enhancer and promoter elements. This work was submitted as a dissertation to the George Washington University. EDUCATION Ph.D. Genetics. George Washington University, Washington, D.C Areas of competency: Molecular Biology, Immunology, Developmental Biology Dissertation: A transgenic mouse model to study immunoglobulin gene enhancer and promoter functional regulation. B.A. Biology. Manhattanville College, Purchase, NY 1980 SELECTED AWARDS AND HONORS CBER Scientific Achievement Award (2000) For exemplary leadership in negotiating, designing and development of procedures to insure adequate oversight of gene therapy trials by FDA and NIH RAC. Center Director s Distinguisher Service Award (2000) For outstanding response to a public health crisis using teamwork, regulatory science and public interaction. FDA Commendable Service Award (1999) For superior collaborative policy development based on review of precedent setting IND s culminating in Guidance for Human Somatic Cell Therapy and Gene Therapy, March 1998 FDA Award (1998) For leadership in development of policy in the area of Gene Therapy. Commissioner s Special Citation (1996) For outstanding contributions and exceptional efforts that resulted in the efficient planning and operation of the National Task Force on AIDS Drug Development PROFESSIONAL MEMBERSHIPS AND AFFILIATIONS: Member-RAPS 2010 Annual Conference Planning Committee Member-Organizing Committee ASGCT 2010 Clinical Trials Training Course Member-ASGT Advisory Council Member-ASGT Clinical and Regulatory Affairs Committee Member- Somatic Cell Therapy Committee, American Association of Blood Banks 2002

3 Member-International Society for Cellular Therapy 2002 present Member-The American Society for Gene Therapy 1997-present Member -The USP Gene and Cell Therapies Advisory Panel Agency Representative (Alternate)-NIH

4 PUBLICATIONS Mendell Jerry R., Andra Miller. Gene Transfer for Neurologic Disease: Agencies, Policies and Process in Gene Therapy of the Central Nervous System: From Bench to Bedside. Elsevier, Inc Mendell, J.R. and A. Miller Gene Transfer for Neurologic Disease: Agencies, Policies and Process. Neurology 2004, 63: Miller, A.E. and S.L. Simek Regulatory Aspects of Gene Therapy, in Gene Therapy: Therapeutic Mechanisms and Strategies. Chapter 18, pages Marcel Decker, Inc. Epstein, S., S. Bauer, A. Miller, A. Pilaro, and P. Noguchi FDA comments on phase I clinical trials without vector biodistribution data. Nature Genetics, 22:326, Wilson, C.A., TH Ng and A.E. Miller Evaluation of Recommendations for Replication-Competent Retrovirus Testing Associated with Use of Retroviral Vectors. Human Gene Therapy 8: Mathers, P.H., A.E. Miller, T. Doniach, M. Dirksen and M. Jamrich Initiation of anterior headspecific gene expression in uncommitted ectoderm of Xenopus Laevis by ammonium chloride. Developmental Biology 171: Miller, A.E., D.L. Ennist, K. Ozato and H. Westphal Activation of immunoglobulin control elements in transgenic mice. Immunogenetics 35: Major, E.O., A.E. Miller, P. Mourrain, R.G. Traub, E. dewidt and J. Sever Establishment of a line of human fetal glial cells that supports JC virus multiplication. Proc. Natl. Acad. Sci., USA 82: Nierman, W.C., A.E. Miller, S.L. Tobin, T.B. Ignolia, F. Sanchez, U. Rdest, E. Zulouf, B.J. McCarthy In vitro transcription of drosophila actin and 70K heat shock protein genes. J. Biological Chem. 258: ABSTRACTS Wilson, C.A., Miller, A.E., Ng, TH Experimental evaluation of assays used to detect murine replication competent retrovirus. FDA Science Forum, Washington, DC, December Ng, TH, C. Wilson, A. Miller and P. Lachenbruch. Testing for replication-competent retrovirus. Forum 1996: FDA/NIH Gene Therapy Conference, Bethesda, MD, July 1996.

5 Miller, A.E., Dirksen, M.L., Jamrich, M. A novel fork head domain containing gene involved in early regionalization of Xenopus brain. Keystone Symposia, Keystone, CO, March Miller, A.E., Ennist, D., Ozato, K., and Westphal, H. A transgenic mouse model to study immunoglobulin gene enhancer and promoter functional regulation. FASEB 89, New Orleans, LA, March PRESENTATIONS Sept 26-27, 2011 May 17, 2010 Jan 26, 2009 Sept, 29, 2008 Oct. 1-3, 2008 ASGCT NIH Gene Therapy Symposium Title: Regulatory Realities vs Resources Bethesda, MD ASGCT Clinical Trials Training Course Session Moderator: Planning for a Clinical Trial Washington, DC Phacilitate Cell & Gene Therapy Forum 2009 Title: Designing Cell and Gene Therapy Clinical Trials Washington, DC Taiwan Department of Health/ITRI Workshop on GTP Regulations Workshop Organizer and Co-presenter Taipei, Taiwan 3rd Annual Taipei International Symposium on Human Cell and Tissue-based Products Title: Current US FDA Regulation of Cell Therapy Title: Current Progress in Cell and Tissue Therapy in US Taipei, Taiwan Sept. 25, 2007 RAPS 2007 Annual Conference Title: The Path to IND: Focus on Product Boston, MA Oct. 17, 2005 RAPS 2005 Annual Conference Session Chair: Challenges in the Development & Regulation of Cell-Based Combination Products Baltimore, MD March 14, 2005 Jan. 25, 2005 Venture Investor Association of New York Panel Discussion of Gene Therapy New York, NY Phacilitate Cell & Gene Therapy Forum 2005 Title: Regulatory and Commercial Impact of Autologous Cell Products

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7 Washington, DC June 2, 2004 Sept. 14, 2003 AABB-ISCT Audioconference Series -Current Good Tissue Practice Title: Good Tissue Practice Overvie 3rd Annual Somatic Cell Therapy Symposium: Regulatory Issues for Scientists and Clinicians. Panel Discussion: Somatic Cell Therapies- Matching Facility to Product. Chesapeake Bay, MD June 3, 2003 Clinical Gene Transfer Training Course, ASGT 6th Annual Meeting Title: What Vector Information Needs to be Submitted, and How Do I Know the Latest Requirements? Washington, DC Sept. 28, nd Annual Conference on Mesenchymal and Nonhematopoietic Stem Cells: Focus on Adult Stem Cells Title: Cellular Therapies: The FDA IND Process New Orleans, LA June 4, 2002 May 5, 2002 June 17, 2001 May 29, 2001 Clinical Gene Transfer Training Course, ASGT 5th Annual Meeting Title: What Vector Information Needs to be Submitted, and How Do I Know the Latest Requirements? Boston, MA International Society for Cell Therapy; Somatic Cell Therapy Symposium Panel Discussion: Somatic Cell Therapy: The Scientist s Viewpoint Sanibel Island, FL International Society for Hematotherapy and Graft Engineering, 7th Annual Symposium Panel Discussion: Mesenchymal Stem Cells, Regulatory and Clinical Issues Quebec City, Canada Clinical Gene Transfer Training Course, ASGT 4th Annual Meeting Title: What Vector Information Needs to be Submitted, and How Do I Know the Latest Requirements? Seattle, WA March 21, 2001 Nov 7, 2000 ASGT:Gene Therapy Vector Production Conference Title:cGMP Expectations for Phase I & Phase II Clincal Trials Bethesda, MD Willamsburg BioProcessing Foundation: Viral Vectors and Vaccines Title: Gene Transfer Product Development Issues Lake Tahoe, Nevada May 31, 2000 American Society of Gene Therapy 3rd Annual Meeting Moderator and Speaker Title: The IND Process and Gene Transfer Product Evaluation Denver, Colorado

8 June 1, 2000 American Society of Gene Therapy 3 rd Annual Meeting Title: Ensuring Product Quality Denver, Colorado