Gene editing medicines

Professor Robin Ali is Professor of Human Molecular Genetics at King’s College London and Director of KCL Centre for Gene Therapy and Regenerative Medicine. He is also Director of the KCL Gene Therapy Vector Facility. Robin is a Fellow of the Academy of Medical Sciences, NIHR Senior Investigator (Emeritus) and a recent past President of the European Society of Gene & Cell Therapy. The focus of his research is the development of gene and cell therapy for the treatment of retinal disorders; he is a founder and former Chief Scientific Officer of MeiraGTx PLC, a Nasdaq-listed clinical-stage gene therapy company. Robin Ali and members of his team have received numerous prizes for their work on developing new treatments for retinal degeneration, including the €1M Champalimaud Vison Award.

Robin has published over 200 peer-reviewed papers with landmark papers and preclinical therapeutic proof-of-concept studies for many different ocular disorders. As chief investigator, he established the world’s first clinical trial of gene therapy for retinopathy and his group is now developing a first-in-human clinical trial of transplantation of ES-derived cone photoreceptors for the treatment of macular degeneration. 

Fyodor Urnov is a Professor of Molecular Therapeutics at UC Berkeley and a Scientific Director at its Innovative Genomics Institute (IGI). He co-developed the toolbox of human genome and epigenome editing, co-named genome editing, and was on the team that advanced all of its first-in-human applications to the clinic. He also led the effort that identified the genome editing target for an approved medicine to treat sickle cell disease and beta-thalassemia. A major goal for the field of genome editing and a key focus of Fyodor's work is expanding access to CRISPR therapies for genetic disease. As part of that effort Fyodor directs the Danaher-IGI Beacon for CRISPR Cures - a first-in-class academia-industry partnership developing and advancing to the clinic scalable CRISPR-based approaches to treat diseases of the immune system.

Professor Robin Ali is Professor of Human Molecular Genetics at King’s College London and Director of KCL Centre for Gene Therapy and Regenerative Medicine. He is also Director of the KCL Gene Therapy Vector Facility. Robin is a Fellow of the Academy of Medical Sciences, NIHR Senior Investigator (Emeritus) and a recent past President of the European Society of Gene & Cell Therapy. The focus of his research is the development of gene and cell therapy for the treatment of retinal disorders; he is a founder and former Chief Scientific Officer of MeiraGTx PLC, a Nasdaq-listed clinical-stage gene therapy company. Robin Ali and members of his team have received numerous prizes for their work on developing new treatments for retinal degeneration, including the €1M Champalimaud Vison Award.

Robin has published over 200 peer-reviewed papers with landmark papers and preclinical therapeutic proof-of-concept studies for many different ocular disorders. As chief investigator, he established the world’s first clinical trial of gene therapy for retinopathy and his group is now developing a first-in-human clinical trial of transplantation of ES-derived cone photoreceptors for the treatment of macular degeneration. 

Dafni is a Pharmaceutical Executive with 30 years of experience in Product Development and Manufacturing Science and Technology.  She is currently SVP and Global Head of Pharmaceutical Technology and Development (PT&D) in Astrazeneca’s Global Operations.  She leads a global, integrated network of scientists and engineers (1500+) in 8 countries, who transform synthetic molecules into medicines and secure approval and robust supply across the product life cycle.   

Prior to AstraZeneca Dafni led the Manufacturing Science and Technology organization in Bristol-Myers Squibb in New Brunswick, NJ, and spent 10 years with MERCK’s Global Pharmaceutical Commercialization in West Point, PA.  She began her career at Procter & Gamble’s European Technical Center in Brussels, Belgium.  She holds a Ph.D. in Materials Science and Engineering from the University of Pennsylvania, a Master of Science in Materials Science and Engineering from the University of Rochester, NY, and a Diploma in Chemical Engineering from the National Technical University of Athens, Greece. 

Mr. Vann leads the commercial activities for Autolus. An industry veteran with 30 years of experience in both development and business roles, he joined Autolus from Hoffmann-La Roche’s Swiss headquarters, where he was latterly responsible for leading the lung cancer commercial team and general management of the Tarceva brand. He has extensive experience in global lifecycle management of oncology products as well as implementing marketing strategy at a regional and national level. This includes launching several oncology, immunology, and transplant products in countries including China, Japan, Romania, Russia, South Africa, the UK, and the U.S. He gained his degree in Toxicology and Pharmacology from the School of Pharmacy, University of London.

Vanessa Almendro, Ph.D, MBA, is Vice President of Science and Technology, Head of Innovation at Danaher Corporation, leading multiple initiatives to position Danaher at the forefront of innovation in science, technology, and medicine. Vanessa is also the Founder and Principal for the Brain Cancer Investment Fund, a philanthropic fund aiming at catalyzing the development of cures for brain cancer. Prior to Danaher, Vanessa was the Head of External Strategy and Innovation at the Eisai Center for Genetics Guided Dementia Discovery (G2D2). In this role, Vanessa led the creation of the Eisai Innovation Center Biolabs, the first incubator space specialized in neurology. She also led the development of academic affairs and external innovation strategies in concert with academic investigators, consortiums, biotech, and large pharmaceutical companies. Prior to this role, Vanessa worked as Head of Strategy and Operations for Cogen Immune Medicines, and she held multiple leadership roles at Vertex Pharmaceuticals both in the R&D and Commercial organizations. Prior to industry Vanessa was a Research fellow at the Dana-Farber Cancer Institute, Harvard Medical School and prior to that served as a Group Leader in the Department of Medical Oncology at IDIBAPS in Barcelona, Spain. Vanessa is a Ph.D. in Biochemistry and Molecular Biology from University of Barcelona, a Post-doc in Translational Medicine from the Dana Farber Cancer Institute, Harvard Medical School, and an Executive MBA from MIT-Sloan. She is a mentor for start-up companies at MassConnect, Nucleate Boston Activator, and a member of the Commercial Advisory Board for the School of Biomedical Engineering at UBC.

Robin Lovell-Badge obtained his PhD at University College London in 1978 and was a postdoc in Cambridge, both with Martin Evans. After an EMBO fellowship in Paris he established his independent laboratory in 1982 at the MRC Mammalian Development Unit, UCL, directed by Anne McLaren. In 1988 he moved to the MRC National Institute for Medical Research, which was incorporated into the Francis Crick Institute in 2015. In 1990, his lab discovered Sry, the Y-linked sex determining gene and the Sox gene family. Major themes of his past and current work include sex determination, development of the gonads, nervous system and pituitary, and the biology of stem cells within these systems. He is also very active in both public engagement and policy work. He has a strong interest in the prospects for applications of human genome editing and was on the organising committee for the three international Summits held on this, including as chair for the 2023 meeting.

Stephanie Krogmeier is the head of Global Regulatory Affairs for Cell and Genetic Therapies at Vertex Pharmaceuticals and has been with Vertex for 19 years. Stephanie earned a Ph.D. in Pharmaceutical Chemistry from the University of Kansas. Stephanie began her career at Vertex in Formulation Development before transitioning to Global Regulatory Affairs CMC. Stephanie has led the CMC preparation of multiple marketing applications which have included Quality by Design strategies, continuous manufacturing processes, and real time release testing strategies. Most recently she led the cross functional submission and approval of Casgevy one-time, autologous CRISPR-edit cellular treatment for sickle cell disease and transfusion dependent beta thalassemia in multiple regions including the US, EU, UK, and KSA. Prior to her current role, Stephanie held multiple leadership positions at Vertex including project leader for the hemoglobinopathies project team and Disease Area Executive for the Hemoglobinopathies Disease Strategy Team.

Toni Cathomen is a Professor of Cell and Gene Therapy at the University of Freiburg, Germany, and serves as the Director of the Institute for Transfusion Medicine and Gene Therapy at the University Medical Center. He received his Ph.D. from the University of Zurich, Switzerland. Previously, Dr. Cathomen held positions at the Salk Institute in San Diego, California, the Charité Medical School in Berlin, Germany, and the Hannover Medical School in Germany. Dr. Cathomen's research focuses on the development of immune cell therapies and hematopoietic stem cell preparations through genome editing. His work aims to provide innovative treatments for patients with primary immunodeficiencies, HIV infection, and certain cancers. He has also helped advance the safety of gene therapy by pioneering methods to assess the genotoxic risk of gene editors and gene-edited cell products.

John is responsible for the delivery of the commercial and managed access function within the Centre for Health Technology Evaluation.  The role involves developing the strategy and directing the work of the team that engages with NHS England, the Department of Health and the life science industry on patient access schemes and commercial and managed access arrangements for the NHS. John previously worked at the Medicines Policy Analysis Team (MPAT) at NHS England where he supported a number of commercial policy areas including horizon scanning, Advanced Therapy Medicinal Products (ATMPs) and managed access. 

Professor Robin Ali is Professor of Human Molecular Genetics at King’s College London and Director of KCL Centre for Gene Therapy and Regenerative Medicine. He is also Director of the KCL Gene Therapy Vector Facility. Robin is a Fellow of the Academy of Medical Sciences, NIHR Senior Investigator (Emeritus) and a recent past President of the European Society of Gene & Cell Therapy. The focus of his research is the development of gene and cell therapy for the treatment of retinal disorders; he is a founder and former Chief Scientific Officer of MeiraGTx PLC, a Nasdaq-listed clinical-stage gene therapy company. Robin Ali and members of his team have received numerous prizes for their work on developing new treatments for retinal degeneration, including the €1M Champalimaud Vison Award.

Robin has published over 200 peer-reviewed papers with landmark papers and preclinical therapeutic proof-of-concept studies for many different ocular disorders. As chief investigator, he established the world’s first clinical trial of gene therapy for retinopathy and his group is now developing a first-in-human clinical trial of transplantation of ES-derived cone photoreceptors for the treatment of macular degeneration. 

Bobby is a world-renowned scientist and physician and accomplished strategic and organizational leader with more than 25 years of experience in medicine and biotechnology. He is co-founder and chief executive officer of Orchard Therapeutics, a global gene therapy leader recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe. Bobby has been a pioneer in gene therapy and the evolution of hematopoietic stem cell (HSC) gene therapy technology—including some of the first studies in patients with severe primary immune deficiencies—bringing it from some of the first studies in patients into late-stage clinical trials. He has led Orchard through the approval of Lib/Lenmeldy in the EU and USA for the treatment of metachromatic leukodystrophy (MLD). His unparalleled expertise and deep relationships with key physicians and treatment centers around the world are integral to Orchard’s efforts to identify patients with MLD and other severe genetic conditions through targeted disease education, early diagnosis and comprehensive newborn screening.

Bobby is an honorary professor of pediatrics and immunology at the UCL Great Ormond Street Institute of Child Health and has led multiple clinical trials that have shown that gene therapy can successfully correct the genetic defect in immune deficiencies. He studied medicine and surgery at Kings College in London before completing his Ph.D. at the UCL Great Ormond Street Institute of Child Health.

Stefano Benvenuti is Head of Public Affairs and Market Access at Fondazione Telethon (Italy). He is vice-chair of the funders constituent committee of the International Rare Diseases Research Consortium (IRDiRC) and co-chair of the Working Group on Health Economic Value of Personalised Medicine Approaches of the International Consortium for Personalized Medicine (ICPerMed). He is also member of the Italian National Committee on Rare Disease. He graduated in International Cooperation for Development at the University of Bologna in 2007 and later earned a master degree in health technology Assessment at Università Cattolica in Rome. As expert of EU policy and experienced manager of EU funded project, he joined Fondazione Telethon in 2016 to set-up the EU affairs office and coordinate the participation of Fondazione Telethon in European and International initiatives.

Dr. Maru, an academic physician, neuroimmunologist and health economist with extensive global experience in clinical research and development, translational medicine, health economics, real-world evidence, and medical affairs. His scientific and industry career has focused on neurology, neuroimmunology, immuno/haemato-oncology, and biological therapies for inflammatory diseases, nephrology, dermatology and rheumatology, and rare/ultra-rare disease. His career has also focused on health economic modelling and real-world evidence generation for the above areas as well as close collaboration with market access representatives. He has supported numerous gene therapy development programs and commercialisation of AAV based gene therapy. Interest in developing programs with patients first and developing with “end in mind”.

Julian oversees the portfolio that is designed to ensure the quality and access of products to the UK market - this includes scientific advice, licensing assessment, marketing authorisations for all innovative and established medicines.

Experienced Leader with a demonstrated history of Quality, Regulatory and Operations in the pharmaceuticals industry. Skilled in Quality, Operations and Digitalisation of Systems, Operations Management delivery including OpEX / Six Sigma with a love for people development. Strong links to industry and regulatory bodies with quality focus. A Fellow of the RSC, with an MBA from Henley Business School, adding to BSc (Hons), Chartered Chemist and Chartered Scientist.

Steve Rees is Senior Vice-President of Discovery Sciences at AstraZeneca with responsibility for the discovery of novel drug candidates, using multiple therapeutic modalities, for projects for all AstraZeneca therapy areas. Prior to his current appointment Steve held positions of increasing responsibility at AstraZeneca including leadership of the Discovery Biology and Screening Sciences and Sample Management departments. He has served as Chair of the European Laboratory Research and Innovation Group, Chair of the European Council of the Society of Laboratory Automation and Screening and as Industry Trustee of the British Pharmacological Society and is a member of  the Royal Society Science, Industry and Translation Committee. Steve was awarded an OBE by Her Majesty the Queen in 2021 for services to science and the COVID19 response.

Fyodor Urnov is a Professor of Molecular Therapeutics at UC Berkeley and a Scientific Director at its Innovative Genomics Institute (IGI). He co-developed the toolbox of human genome and epigenome editing, co-named genome editing, and was on the team that advanced all of its first-in-human applications to the clinic. He also led the effort that identified the genome editing target for an approved medicine to treat sickle cell disease and beta-thalassemia. A major goal for the field of genome editing and a key focus of Fyodor's work is expanding access to CRISPR therapies for genetic disease. As part of that effort Fyodor directs the Danaher-IGI Beacon for CRISPR Cures - a first-in-class academia-industry partnership developing and advancing to the clinic scalable CRISPR-based approaches to treat diseases of the immune system.

Suk See De Ravin is a Senior Research Physician at the National Institutes of Allergy and Infectious Diseases (NIAID), National Institutes of Health. She received her paediatric training at Sydney Children’s Hospital, Australia, and after completion of her PhD in Primary Immunodeficiency Diseases (PID), joined Laboratory of Host Defences at NIAID to continue work on novel gene and cell therapies of PID. She has been working on haematopoietic stem cell gene therapy for X-linked Severe Combined Immunodeficiency and Chronic Granulomatous Disease. Current lentivector gene therapy is helpful for many patients with PID but random vector insertions and lack of gene regulation remain important concerns.  Using state-of-art functional gene correction strategies, she has applied base-editing to precisely repair CGD and XSCID-causing mutations, a precise medicine that will improve safety and efficacy of gene therapy that will hopefully be expanded into treatment of the broader range of PIDs.

John is responsible for the delivery of the commercial and managed access function within the Centre for Health Technology Evaluation.  The role involves developing the strategy and directing the work of the team that engages with NHS England, the Department of Health and the life science industry on patient access schemes and commercial and managed access arrangements for the NHS. John previously worked at the Medicines Policy Analysis Team (MPAT) at NHS England where he supported a number of commercial policy areas including horizon scanning, Advanced Therapy Medicinal Products (ATMPs) and managed access. 

Dr. Birgit Schultes is the SVP, Head of Cell Therapy, at Intellia Therapeutics Inc. She brings over two decades’ experience in the biotechnology and pharmaceutical industry and leads a multidisciplinary cell therapy team focused on ex vivo applications of CRISPR/Cas9 since 2017 and acts as the Program Lead for a preclinical stage CAR-T cell for solid tumors. Her work is focused on CRISPR-engineered CAR-T and TCR-T cells as well as synthetic Tregs for immuno-oncology and autoimmunity indications, including allogeneic and solid tumor solutions. Prior to joining Intellia, Birgit served as a Senior Director at Unum Therapeutics (now Cogent Biosciences, Inc.), where she directed the preclinical efforts for Unum’s universal T cell programs. Earlier in her career, Birgit held a variety of translational research roles, working on different biologics for immuno-oncology and immunology applications at Momenta Pharmaceuticals, United Therapeutics and AltaRex Corp. Dr. Schultes holds a Ph.D. in Immunology and a M.S. in Cell Biology from the University of Bonn, Germany.