Speaker Biographies

Jay A. Barth, MD, joined LEXEO Therapeutics as Executive Vice President & Chief Medical Officer in October 2020. Most recently, Dr. Barth was Chief Medical Officer at Amicus Therapeutics, where he led a clinical research, operations, regulatory affairs, safety, and medical affairs organization focused on rare disease and gene therapy. Dr. Barth oversaw Phase 1 through Phase 4 development and clinical and regulatory activities leading to FDA, EMA, and global regulatory approvals. Prior to joining Amicus, Dr. Barth held roles of increasing responsibility at PTC Therapeutics culminating in a position as Senior Vice President, Clinical Development. Dr. Barth has over two decades of experience in drug development in both public and private company settings, translating science to therapeutics across multiple therapeutic areas. Dr. Barth received his medical degree from the University of Pennsylvania School of Medicine and his undergraduate degree from Columbia University.

Veronique Blouin, PhD, has over 25 years of experience in viral vectors production and process development. She is currently manager of the vector core research grade and process development (CPV) at Translational Gene Therapy for Genetic Diseases at Target UMR-INSERM1089 Nantes University, France. This activity covers the development and transfer of rAAV product processes with associated quality controls from the research laboratory bench to the GMP clinical scale. The Translational Vector Core recently obtains the label “Intégrateur Industriel” (manufacturing integrator), as part of the program National Big Challenge in Biomedicine (“Grand défi national Biomédicaments”). In addition, she has experience in gene transfer in small animal model of diseases. In 2004-05, she acquired experience in rAAV clinical vector production at the University of Florida, Dr Snyder. Since 2005, she has been participating in the Pharmacopeia group (actually the ATMPs group) as expert for the ANSM. In particular, she has participated in writing the 5.14 European Monograph relating to the gene therapy products. She obtained the PhD in Virology from the University of Nantes in 2007 and a Master of biochemistry, cell and molecular biology. She currently holds an IR1 Inserm position.

I have received my PhD in Biochemistry and Molecular Genetics from Northeastern University, Boston, MA, and did the post-doc at Boston University Medical Center. I worked for 14 years at Momenta Pharmaceuticals, Cambridge, MA in research and development focusing on the development of protein, peptide and heparin-based therapies for the broad spectrum of the inflammatory and autoimmune indications. Some of the activities I was involved in were: translational research, analytical methods development of the complex biologics, bioprocess development and bioassays for preclinical and clinical development including d potency. For the last two years I have worked at Ultragenyx Gene Therapy supporting several programs for the rare and ultrarare diseases for the AAV-based therapies. My major focus is on the potency matrix development during clinical development and registration.

Liching Cao is a Director of Bioanalytical Operations at Sangamo Therapeutics. At Sangamo, she manages bioanalytical assay outsourcing supporting nonclinical and clinical studies across the company’s portfolio. She is also responsible for interfacing with the clinical team, qualifying and overseeing external laboratories for assay setup and sample analysis. Liching has over 20 years of industry experience with 15 years focused on regulated nonclinical and clinical bioanalysis. Before joining Sangamo Therapeutics in 2016, she served as a bioanalysis supervisor managing the GLP laboratory at XOMA.

Dr. Chung is the Chief Medical Officer for SparingVision, an ocular genomic medicine company, focusing on gene agnostic gene therapy and CRISPR gene editing approaches to combat blinding diseases. Prior to his recruitment to SparingVision, Dr. Chung was the Ophthalmology Therapeutic Leader for Spark Therapeutics, where he led the medical affair group and contributed to areas of clinical development and operations, marketing, commercial, patient advocacy, pre-clinical research and development and business development. Dr. Chung was intimately involved with the development of Luxturna, the first gene therapy approved by the FDA and EMA for use in a blinding genetic disease. Prior to joining Spark Therapeutics, he was a senior investigator/instructor at the FM Kirby Center for Molecular Ophthalmology at the Scheie Eye Institute at the Perelman School of Medicine of the University of Pennsylvania, working in retinal gene transfer and therapy. Concurrently, he served as the scientific advisor on the RPE65 gene therapy study team for phase 1 and 3 of the clinical trial at the Children’s Hospital of Philadelphia (CHOP).

Kate Cygnar is currently a Director of Genome Engineering Technologies at Regeneron in Tarrytown, NY. She is currently focusing on new technology development to enable the creation of new medicines for lysosomal diseases and other genetic diseases. She also works on novel applications of bispecific antibody technologies in several therapeutic areas. Before joining Regeneron, she completed her undergraduate training at the University of Minnesota Twin Cities, and her PhD and postdoctoral training at The Johns Hopkins University in Baltimore, focusing on genetics and neuroscience.

Lauren M. Drouin is Director of the Genomic Medicine Analytical Development group at Alexion, AstraZeneca Rare Disease. Her team supports analytical testing for AAV gene therapy and gene editing products, including initial method development, assay qualification, and product characterization, in addition to assisting CMC operations with plasmid DNA, cell bank, and DS/DP batch release and stability programs. Lauren managed CMC analytical operations for the Methylmalonic Acidemia clinical program, the first in vivo gene editing therapy delivered systematically to pediatric patients, and was significantly involved in both the manufacturing campaign and subsequent regulatory submission. Her current research interests include novel AAV capsid characterization and developing a robust understanding of the factors that influence potency of gene therapy products. Previously, she worked at Voyager Therapeutics where she was responsible for analytical method development and overseeing CMC analytical operations for the Parkinson’s Disease clinical gene therapy program. Lauren received her PhD in Biochemistry and Molecular Biology from the University of Florida where she utilized molecular, biophysical, and structural techniques to characterize the AAV capsid for improved gene delivery applications. She currently serves as Chair of the AAV Gene Therapy Products Expert Panel for the United States Pharmacopeia and is a member of the Global Outreach Committee for the American Society of Gene & Cell Therapy, working to expand access to cell and gene therapies on a global scale.

Dr. Susan Faust is the Founder and CEO of NxGEN Vector Solutions, LLC. She has experience in scientific research, academic technology transfer, patent prosecution, and business development. Dr. Faust received a B.S. in Microbiology from the Pennsylvania State University with Minors in Biochemistry and Molecular Biology, a Ph.D. in Cellular and Molecular Biology and a concurrent Master’s Degree in Human Genetics from the University of Michigan. She held a postdoctoral position at the University of Pennsylvania studying the immune response to viral vectors used in gene therapy. Dr. Faust is an inventor on multiple patent applications and has received numerous awards for her work, including a Young Investigator Award from the American Transplant Congress and the Sanjeev Kumar Excellence in Science Award from the University of Pennsylvania.

Earned B.S. in Biochemistry and Molecular Biology from Michigan State University in 2011; performed graduate work in the lab of Dr. James M. Wilson at the Gene Therapy Program at the University of Pennsylvania in the field of AAV gene therapy and defended doctoral thesis titled "Immunological and biochemical evaluation of the AAV capsid to advance next-generation gene therapy vector design" in the spring of 2018; currently a Scientist II in the Gene Transfer Technologies group at REGENXBIO with a focus on capsid engineering and the development of novel therapeutic modalities.

Boris Gorovits is a VP of Bioanalytical & Non-Clinical Biomarkers at Sana Biotechnology. Boris earned a Ph.D. in Enzymology from the Moscow State University and later completed postdoctoral research studies in Protein biophysics at the Medical Center, the University of Texas at San Antonio, TX. In 2000, Boris joined Wyeth Research (later Pfizer Inc.) to lead a Bioanalytical group responsible for many aspects of the Regulated and Non-Regulated Bioanalytical support for pan-Pfizer Biotherapeutic portfolio. Boris co-chaired Pfizer internal Immunogenicity Expert Working Group responsible for review of the biotherapeutic immunogenicity risk assessment and mitigation strategies. Later, Boris transitioned to Sana Biotechnology where he continues to be involved in development of various gene therapy and cell therapy modalities. Boris has been actively involved in industry discussions focusing on PK and immunogenicity assessment bioanalytical support of various modalities, including mAbs, bispecific antibodies, antibody-drug conjugates, gene therapy, and CAR Ts.

Kathy Ivey is Vice President of Gene Therapy Research at Tenaya Therapeutics, where her team is developing transformative treatments for heart disease. Her group focuses on 1) capsid engineering to create novel AAV vectors with improved properties and more selective tropism for particular cardiac cell subtypes and 2) developing and testing cassettes to drive optimized, cardiac-selective gene augmentation to prevent or reverse specific forms of cardiac disease. Prior to joining Tenaya at its founding, Kathy was a Staff Research Investigator at The Gladstone Institutes and served as Director of the Gladstone Stem Cell Core. She has co-authored over 30 peer-reviewed publications on cardiac development and disease since 2001. Dr. Ivey is trained in Molecular and Cell Biology and holds a B.S. from Texas A&M University and PhD from UT Southwestern Medical Center. She also trained as a California Institute of Regenerative Medicine (CIRM) Postdoctoral Scholar at The Gladstone Institutes.

Smitha is the Director of Neuroscience/Pain – New Modalities at the Boston Innovation Hub, Grunenthal. New modalities will shape the field of pain research to create breakthrough medicines for pain patients and push the frontiers of modern medicine. Prior to joining Grunenthal, Smitha was in the Genomic Medicine Unit at Sanofi Genzyme and led several AAV gene therapy projects in the rare neurological disease area. Smitha received her PhD in Neuroscience from Columbia University, NY. She did her postdoctoral fellowship in University of California in San Francisco, CA and was a Research Scientist at the Whitehead and Picower Institutes at MIT.

Darshana Jani is Head of Global Bioanalytical Sciences at ModernaTx. Prior to that, she served as a Director at Agenus and Scientific Associate Director at Pfizer, Cambridge, MA, USA, where her role was to serve as scientific and technical lead for both CROs and internal teams for development, validation, and application of bioanalytical and biomarker assays to comply with scientific and regulatory requirements. She has over 25 years of experience in supporting preclinical to clinical studies holding positions with Sanofi, MedImmune, Biogen, and Pfizer. She has risen from the ranks, at the outset developing and applying bioanalytical methods, while assuming numerous responsibilities germane to regulatory compliance of the validation and application of group procedure. She has also guided bioanalytical lab groups held responsible for overseeing any and all phases of drug recovery and development; a task which incorporated product characterization, potency determination, immunogenicity testing, as well as surrogate biomarker assay development, validation, and sample testing. Lastly, she has published several recommendation white papers with industry, academic, and regulatory peers in the area of biomarkers, as well as immunogenicity.

Jeremy is currently serving as Group Leader of Analytical Development at Asklepios Biopharmaceuticals, Inc. in RTP, North Carolina. Jeremy has more than 20 years of experience in the development and manufacturing of therapeutic proteins, and has overseen analytical biopharmaceutical development for recombinant proteins at all stages of development and commercialization. He is working to update analytical methodology utilized in structural characterization of AAV vectors by utilizing Q-TOF LC/MS for quantitation and peptide analysis.

Jennifer is the creative force behind NysnoBio, responsible for the business and scientific focus of the company as CEO. She has over 20 years of experience in the biopharmaceutical industry, including 13 years in neurodegenerative diseases at Elan Pharmaceuticals. As VP, Head of Discovery Research at Elan, Jennifer delivered multiple novel molecules for Parkinson’s disease from initial discovery to candidate selection and lead the team that published the first crystal structure of the Parkin protein, and described its novel enzymatic activity. In 2015, she co-founded An2H Discovery, focused on ubiquitin pathway enzymes, and successfully led the company to identification of lead candidates with potent in vivo activity. In 2019, Jennifer co-founded NysnoBio to focus on the development of small molecule and gene therapy approaches for Parkin E3 ligase. She has studied the Ubiquitin pathway throughout her education, from her Ph.D. at Dartmouth, to subsequent work on E3 ligases at Caltech with Alex Varshavsky, and to her discovery of Aggresomes at Stanford with Ron Kopito, resulting in more than ten patents and over 40 publications. She has served as a reviewer for multiple scientific journals, and on the SAB of the Michael J. Fox Foundation for over 15 years. Outside the office, Jennifer has been a member of the USA Track & Field 100Km Team, earning bronze and silver medals in the national championships, as well as holding the record for the most wins in the Angeles Crest 100-mile trail race.

Eric Kelsic, PhD, is CEO and co-founder of Dyno Therapeutics, a VC-backed biotech located in Cambridge, Massachusetts. Dyno is leading a machine learning revolution to develop enhanced capsid proteins that enable new gene and genome editing therapies. Eric co-developed the technology underlying Dyno’s machine-guided protein engineering platform in George Church’s lab at the Wyss Institute of Harvard Medical School. He holds a PhD in Systems Biology from Harvard University and a BS in Physics from Caltech.

Dr. Kishimoto is the former Chief Scientific Officer of Selecta Biosciences, a biotechnology company developing tolerogenic ImmTOR technology to mitigate unwanted immune responses. Prior to joining Selecta, Dr. Kishimoto was Vice President of Research at Momenta Pharmaceuticals where he led multidisciplinary teams in inflammation, oncology, and cardiovascular disease. Previously he was Senior Director of Inflammation Research at Millennium Pharmaceuticals, where he provided the scientific leadership for four programs in clinical development, and an Associate Director of Immunology at Boehringer Ingelheim. Dr. Kishimoto received his doctoral degree in Immunology from Harvard University and his post-doctoral training at Stanford University. He is currently an independent consultant.

Dr. Sigrid C. Kuebler has over 20 years of experience advancing analytical instrumentation at technology companies. For the past 15 years, she has served in various customer-facing roles in Marketing, Product Strategy, and Applications. She has a diverse background in biopharmaceutical and particle applications and customer advocacy. Her Ph.D. work was performed at the Max Planck Institute for Polymer Research.

Joseph Lee received his Doctorate in Biochemistry from McGill University (Montreal, QC, Canada). He further trained at the NIH (NCI and NIDDK). Collectively, Joe’s research background focused on signaling pathways leading to the dysregulation of cell growth – much of it centered on viral and cellular oncogenes. Following his post-doctoral fellowship, he led the Development Services/Molecular Biology group at BioReliance where he helped set up the Gene and Cell Therapy testing programs. Joe has held additional posts at Shire and Takeda supporting gene therapy and vaccine programs from both the CMC and Clinical perspectives. He was later recruited to build the Analytical Development (gene therapy) at group at PTC Therapeutics in New Jersey. Currently Joseph is leading the CMC Early Development Analytics group where his team is responsible for vectorology and assay development support for all of the cell therapy programs.

Jing Liao is Director of Vector Development and Operations at LogicBio Therapeutics. Her team is focusing on capsid engineering to improve the properties of the capsids. She is also working on plasmid optimization and cell line engineering to increase the yield of AAV production. Dr. Liao was trained in Molecular, Cell and Stem cell Biology. She got her PhD from Institute of Biochemistry and Cell Biology (IBCB), Chinese Academy of Sciences (CAS) focusing on gene editing and reprogramming. She held a postdoctoral position at Harvard University studying epigenetic regulations.

Dr. Martin Linhult is currently working at Diamyd Medical AB, Stockholm as a CMC Lead. He is working in scaling up and tech transfer of recombinant processes. Dr. Linhult has over 20 years of industry experience and has been involved in several different BLA and MAA submissions.

Anna Pavlova is a Scientist III at Sangamo Therapeutics, where she develops biochemical and biophysical techniques for characterization and release of AAV gene therapy products. She is passionate about her work in gene therapy where there are so many opportunities for discoveries, deriving new knowledge and advancing this rapidly emerging field. Her experience also includes analytical development work in biotherapeutic proteins and monoclonal antibodies. Anna obtained her Ph.D in Biochemistry from the University of California Santa Barbara.

Lyndi Rice, PhD is the Head of Gene Therapy Analytical Technologies (GTAT) at BioMarin, focusing on analytical strategies from early phase development through commercialization for gene therapy products. GTAT works closely with Technical Development teams, CMC, Regulatory, site QC, QA, and several other departments to streamline method development, optimization, transfers and validations, and to platform methods with a focus on method lifecycle strategies. Previously, she served as Head of QC Viral Vector Analytical at BioMarin and has been with the company for almost four years. Her team focused on routine testing and method life cycle management for gene therapy products, including method transfer, validation, and optimization work. Prior to BioMarin, Lyndi was the Director of Analytics at the Gene Therapy Program (GTP), University of Pennsylvania, where she led multiple teams that conducted analytical development and QC release of toxicology and GMP material. Prior to GTP, she spent nearly a decade working at a biotechnology company where she oversaw a team that worked on oncology and cardiovascular disease therapeutics, spanning target identification/validation through pre-clinical development.

Dr. Weiping Shao is currently Sr. Group Director and Head of US GxP Testing Lab at AstraZeneca (AZ), where he leads regulated bioanalysis and scientific innovation to support the development of biologics modalities across all therapeutic areas. Weiping brings more than 20 years of experience and leadership in pharmaceutical / biotech industry, including his previous role as Vice President of Biologics Services at a global contract research organization and Director of Bioanalytical Operations at Regeneron Pharmaceuticals, Inc. He has built and led cross-functional organization that supported biologics development, bioanalytical services and biomarker discovery. Weiping has published over 40 peer reviewed manuscripts/commentaries, filed US patents and co-authored industry Best Practices. He holds leadership roles at ISBER organization, chairs conferences and has given numerous presentations. He earned his Ph.D. from Nanjing University and completed his post-doctoral Fellowship in Biochemistry at University of California, San Diego.

Jae-hyucsk Shim received his Bachelor’s and Master’s degrees in Biotechnology from Yonsei University, Seoul, Korea. In 2000, he obtained his PhD degree from the Immunobiology program at Yale Medical School, New Haven under the supervision of Dr. Sankar Ghosh. In 2006, he joined the laboratory of Dr. Laurie Glimcher at Harvard School of Public Health as a postdoctoral fellow and completed his training in 2010 and worked as a research scientist/instructor. With a unique cross-disciplinary combination of immunology and bone biology (osteoimmunology), he began independent research in the field of aging- or inflammation-associated bone diseases at the Weill Cornell Medicine, New York, as an assistant professor of Pathology in 2012. In 2016, he relocated to the University of Massachusetts Medical School as an associate professor of Medicine (Rheumatology). He also serves as a Director of Bone Analysis Core from 2019.

Inderpal Singh is an Immunogenicity Prediction Lead in Spark Therapeutics. He is providing scientific leadership focused on immunogenicity risk assessment and prediction strategies for AAV-mediated gene therapy including immunomodulation and mitigation strategies to overcome any potential vector immunogenicity. Inderpal earned his Ph.D. from the University of Cambridge, UK. After completing his postdoctoral training at Tufts University, Inderpal worked in Sanofi, where he was managing projects related to immunological assessments of vaccines candidates and then later, he joined Teva Pharmaceuticals to establish immunogenicity prediction capabilities to support early lead candidate selection for large molecules and regulatory approvals for complex peptide projects. Overall, his experience involves assessing innate and adaptive (cellular and humoral) immune responses to different modalities (gene therapy/biologics/peptides/vaccines) and establishing mitigation strategies to overcome immunogenicity-related challenges.

Dr. Taiel completed her doctorate in Medicine with board certified in Ophthalmology from Lariboisiere Saint Louis University, Paris, France, in 1993, and her Associate Professor degree in 1998. Dr Taiel completed her internship at academic Paris hospitals, was an Associate Professor of Ophthalmology, served as an Ophthalmology Department Head, and ran Surgical and Medical Ophthalmology private practice. After 13 years of Ophthalmology public and private practice, Dr. Taiel has been engaged in the Pharma Industry for 20 years; she brings extensive experience and expertise in drug clinical development, gene therapy, and medical affairs. She started her carrier at Servier company headquarter, and then worked in Ophthalmology area at Pfizer for several years; she then held international and management positions in various therapeutic areas, including both technical and supervision duties, at Eli Lilly Company for many years. Then, as VP Clinical Development, she led Clinical Development and Operations, to develop antisense oligonucleotides in Inherited Retinal diseases at ProQR Therapeutics. She then moved to GenSight-Biologics in 2018, to supervise the Medical Department and lead Gene Therapy programs in Inherited Retinal and Neuro-Ophthalmology diseases, as the CMO of the company. Dr. Taiel has authored numerous protocols and articles published in peer reviewed journals, and made critical contributions to successful clinical development and launch of many products. She brings extensive years of experience from both academic medicine and pharma industry.

Huimin Tao is a principal scientist in the analytical development department at Novartis gene therapy. She leads of AD Molecular biology team in the development, qualification, and QC transfer of high-throughput sequencing and ddPCR assays to characterize AAV drug products. She has more than 15 year’s industry experience in molecular biology assay development, a member of the British Pharmacopeia Advanced Therapy Medicinal Products Working Party. Huimin used to be a physician in China, specializing in Neurology. She received her master's degree in clinical medicine from Wuhan University, China, and her master's degree in public health from the University of Virginia, United States.

Lawrence (Larry) Thompson is an Associate Research Fellow and Group Leader in Analytical R&D within BioTherapeutic Pharmaceutical Sciences at Pfizer. He is an analytical CMC SME for Pfizer’s adenoviral & plasmid DNA based immunotherapeutics, mRNA drug substances and nucleic acid starting material pipeline (used in rAAV and mRNA production). Prior to joining Pfizer, he worked at a couple of small biotech companies developing of serum-based cancer diagnostics. He received his PhD in Biochemistry from Vanderbilt University and did his post-doctoral work at the University of Tennessee. His work has generated several peer reviewed publications and presentations at scientific conferences.

Dr. Lili Wang is a research associate professor and a research director at the Gene Therapy Program at the University of Pennsylvania. She has been working in the field of AAV gene therapy for over 20 years and has extensive experience in the development of in vivo gene therapy to liver. She has led translating preclinical successes to phase I - III clinical trials of gene therapy for genetic diseases. She further expanded her research interest to in vivo genome editing as novel therapy approaches to treat rare genetic disease and demonstrated efficacy in preclinical studies in animal models such as hemophilia B and urea cycle disorders.

Li Chin Wong is the Director of Biomarker and Bioanalytical Development at Prevail Therapeutics, a wholly owned subsidiary of Eli Lilly and Company. Overseeing both the clinical and nonclinical biomarker strategies and execution, Li Chin is responsible for translational biomarkers for all neurodegenerative programs at Prevail. Prior to Prevail, Li Chin was at Merck and Company in the Translational Molecular Biomarkers team. Li Chin was involved in Discovery neurodegeneration research at PTC Therapeutics. She held two postdoctoral fellowships, at NYU School of Medicine focusing on behavioral neuroscience using in vivo electrophysiology recording, and at Memorial Sloan Kettering Cancer Center, studying neurodevelopment. Li Chin earned a Ph.D. in Molecular Biology at Princeton University and a B.S. in Biology at Indiana University Bloomington.

Zoe Zhang is a formulation scientist at REGEXBIO, an adeno-associated virus gene therapy company based in Maryland. Before REGENXBIO, Zoe has worked at Novavax and Fresenius Kabi on formulation and process development for vaccines, peptides, and small molecules. She has published multiple research articles and meeting presentations. Zoe received her Ph.D. in Biopharmaceutical Sciences from the University of Illinois at Chicago and worked on microRNA delivery with polymeric nanoparticles for her Ph.D. thesis.