Gene editing medicines

Linda Partridge works on the biology of ageing. Her research is directed to understanding the mechanisms by which healthy lifespan can be extended in laboratory model organisms and humans. Her work has focused in particular on the role of nutrient-sensing pathways, such as the insulin/insulin-like growth factor signalling pathway, and on the role of diet.

She is the recipient of numerous awards, including the Royal Society's Croonian Prize Lecture, and was honoured with a DBE for Services to Science in 2009. She is a Fellow of the Royal Society, the Academy of Medical Sciences and a foreign member of the American Academy of Arts and Sciences. She is Director of the Institute of Healthy Ageing at UCL and the founding director of the Max Planck Institute for Biology of Ageing in Cologne, Germany.

Professor Julian Gillmore is a clinical academic who undertook MD and PhD degrees in the field of amyloidosis. His research interests include pathogenesis, diagnosis and treatment of amyloidosis. He was appointed Centre Head at UCL’s Centre for Amyloidosis in 2019 and is research lead at the UK National Amyloidosis Centre. He highlighted for the first time in a systematic fashion, the relationship between the abundance of amyloid fibril precursor proteins, the course of amyloid deposits and clinical outcome in a number of different forms of amyloidosis thereby establishing the paradigm for the treatment of all the systemic amyloidoses. He established the ‘non-biopsy’ diagnostic criteria for cardiac transthyretin (ATTR) amyloidosis which have been adopted worldwide and have directly contributed to the huge increase in awareness and diagnoses of this form of cardiomyopathy.

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.

Marcello Maresca is a Senior Director at AstraZeneca and currently leads the Genome Engineering department. He received his PhD in Molecular Biology from the Max Planck Institute of Molecular Cell Biology and Genetics in Dresden, where he focused on developing recombineering, a widely used genome engineering tool in bacteria. After his PhD, he pursued postdoctoral work at the Novartis Institute of BioMedical Research in Cambridge, MA, USA, where he worked on developing and applying genome editing tools in drug discovery. Currently, at AstraZeneca and Alexion, Marcello and his team are working to establish Genome Editing as a novel therapeutic modality.

Angelo Lombardo is a Professor of Tissue Biology and Regenerative Medicine at Vita-Salute San Raffaele University (UniSR; Milan, IT) and a Group Leader at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget; Milan, IT). He is also a co-founder of Chroma Medicine Inc. (Boston, MA, USA). His research focuses on the development and application of innovative gene therapy technologies, including genome and epigenome editing. Throughout his career, he has published over 40 articles in peer-reviewed journals, including Cell, Nature, Nature Biotechnology, and Nature Methods. He is an inventor on several patent applications related to his work and has received numerous prestigious international awards, including the Young Investigator Award and the Excellence in Research Award from the European Society of Gene and Cell Therapy.

Laura Sepp-Lorenzino, Ph.D. joined Intellia Therapeutics in 2019 as Chief Scientific Officer and is responsible for Research and Early Development. Intellia is harnessing CRISPR-based technologies to revolutionize the future of medicine. Laura previously held leadership positions at Vertex, Alnylam and Merck. She serves on the Board of Directors of Taysha Gene Therapies and the Alliance for Regenerative Medicine, and on the Scientific Advisory Boards for Thermo Fisher Scientific, the U.K. Nucleic Acid Therapies and Arsenal Capital Partners. She received her professional degree in Biochemistry from the University of Buenos Aires, Argentina, and both her M.S. and Ph.D. in Biochemistry from New York University.

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.

Luigi Naldini, M.D., Ph.D., is Professor of Cell and Tissue Biology and Gene and Cell Therapy at the San Raffaele University and Scientific Director of the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. He has pioneered the development and applications of lentiviral gene transfer vectors, one of the most widely used tool in biomedical research, which are providing safe and effective therapies for several diseases, some of which are now registered medicines. He contributed to the first demonstration of T-cell receptor gene editing and of targeted gene correction in human HSC and introduced hit-and-run epigenetic editing. Has published >300 papers (SCOPUS h-index 108), has been President of the European Society of Gene and Cell Therapy (ESGCT), received numerous awards including Outstanding Achievement Awards from the American Society of Gene and Cell Therapy (ASGCT) and from ESGCT, the Beutler Prize from the American Society of Hematology and the 2019 Jeantet-Collen Prize for Translational Medicine. He is also co-founder of the biotech start-ups Genenta (listed on Nasdaq), Chroma Medicine and Genespire.

Dr. Kulkarni obtained his PhD from the University of British Columbia and has over 12 years of academic and industry experience in the nanoparticle drug delivery field. He has published over 40 peer-reviewed articles in prestigious journals and co-inventor on numerous patents. Dr. Kulkarni’s research has focused on the role of the various lipid components in LNP and the biophysics that governs particle formation. His work has contributed to clinical translation, including scale-up and manufacturing of LNP systems in accordance with GLP and GMP regulations. Dr. Kulkarni is a leader in the design and development of lipid nanoparticle (LNP) formulations of small molecule and nucleic acid therapeutics. He currently serves as the Chief Scientific Officer of NanoVation Therapeutics, an LNP-RNA formulation developer.

Robin Lovell-Badge is interested in decisions of cell fate, especially within the context of sex determination in mammals. Robin discovered that a single gene, Sry, present on the Y chromosome, sets cells of the reproductive organs on the path to becoming male — famously propelling ‘Randy’, a female mouse he engineered to develop into a male, to front-page news.

His work also includes a focus on stem cells. These self-renewing cells have the potential to transform into any other cell type and hold promise for treating a wide range of diseases. By studying the genes that confer these special abilities, Robin is examining how the decisions that determine the ultimate role of a cell in the body are made.

Some of Robin’s research is conducted using mouse models, which he compares with findings in chicks to add an evolutionary perspective. Consequently he is very interested in the public perception of using animals in research. He also advises on policy relating to stem cells and their use in medical treatments.

My name is Alyssa. I am 15 and am currently studying for my GCSEs. When I was 12 I was diagnosed with T-Cell Acute Lymphoblastic Leukemia. A few weeks later we found out that it wasn’t working as well as hoped so I went onto have a bone marrow transplant which also failed. After that my cancer was diagnosed as terminal. However my consultant found a new trial in Great Ormond Street Hospital, I met all of the requirements and was the first person in the world to receive this amazing new treatment. It used T-Cell editing to kill the cancerous cells and it worked! After getting a second chance in life I love spending time with my family and our Labrador Holly as well as riding my bike and enjoying life as much as possible and love working with charities to help them raise more money for life-saving research.

An actively practicing cardiologist and committed teacher, Dr. Kiran Musunuru is Professor of Cardiovascular Medicine and Genetics in the Perelman School of Medicine at the University of Pennsylvania. His research focuses on the development of gene editing therapies for cardiovascular and metabolic diseases. He is a recipient of the Presidential Early Career Award for Scientists and Engineers from the White House, the American Heart Association’s Award of Meritorious Achievement and Joseph A. Vita Award, the American Philosophical Society’s Judson Daland Prize for Outstanding Achievement in Clinical Investigation, the American Federation for Medical Research’s Outstanding Investigator Award, Harvard University's Fannie Cox Prize for Excellence in Science Teaching, and the University of Pennsylvania's Jane M. Glick Graduate Teaching Award. He is author of The CRISPR Generation: The Story of the World's First Gene-Edited Babies and Genome Editing: A Practical Guide to Research and Clinical Applications. He is a co-founder of Verve Therapeutics.

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”.

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.

Waseem Qasim is Professor of Cell and Gene Therapy at UCL Great Ormond Street (GOS) Institute of Child Health in London. He has led ‘bench-to-bedside’ development of new treatments that use emerging genome editing strategies to reprogram immune cells to treat cancers. Clinical studies at GOS were the first to show the potential of donor T cell modified, initially using TALENs and then CRISPR/Cas9 and, most recently, ‘base editing’ technology. Similar approaches are now being developed to repair genes inside living cells to help children with inherited blood or immune disorders, and these developments are setting the scene for a new generation of genetic medicines.