Amarna Therapeutics is a privately held, pre-clinical biotech company developing transformative, potentially curative gene therapies for a range of both rare and prevalent diseases.
The company is pioneering the development of gene therapies based on simian virus 40 (SV40), a macaque polyoma virus to which humans are immunologically naïve. Amarna has created a proprietary production cell line (SuperVero™) that, for the first time, makes it possible to produce SV40-derived vectors suitable for therapeutic use.
Combining SuperVero™ with its genetically engineered Nimvec™ viral vector, the company’s fully-integrated gene therapy platform is truly unique in its intrinsic capability to deliver transgenes without eliciting immune responses to the vector nor to the transgene product.
Leveraging this ‘natural’ non-immunogenicity feature of its gene delivery system in humans, Amarna is developing a broad pipeline of safe, effective and durable gene therapies focused on genetic disorders, autoimmune diseases and chronic inflammation.
Founded in 2008, Amarna’s head office is based in Leiden Bio Science Park, the Netherlands, with a research subsidiary located in Seville, Spain.
Dr. Henk Streefkerk is a distinguished physician scientist (MD PhD) with a unique blend of clinical experience in Ear, Nose, and Throat surgery, a PhD in Neurovascular surgery, and more than two decades in the pharmaceutical industry. His extensive career spans both big pharma (since 2006) and biotech (since 2017), driven by a professional passion for coaching people and advancing drug development projects to benefit patients.
Professional Experience
Leadership in Pharmaceutical Industry, Dr. Streefkerk's career highlights include:
Strategic Expertise
Regulatory and Quality Management
Peter has a virology background and has been active in R&D for more than twenty-five years.
Peter’s academic career started as a Virologist at the Wageningen University in The Netherlands, where he obtained his PhD in 1991. In 1991 he joined Novartis Seeds where he held several research management positions in the field of plant biotechnology. He is a co-founder of Phytovation B.V., formerly active in biopharmaceuticals and vaccine production, and of its subsidiary Viruvation B.V., which was active in developing therapeutic AIDS and HCV vaccines.
Throughout his career Peter has worked on applications of virus-associated technologies. He has a long-standing expertise in virology, molecular & cell biology, immunology and is highly experienced in molecular cloning activities, tissue culture of mammalian cells and production of (recombinant) virus particles in mammalian cell systems.
Peter is the (co-) author of more than fifty peer reviewed scientific articles. Next to this, he is the company’s intellectual property (IP) manager. Peter has a longstanding expertise with IP matter as (co-) inventor of more than twelve different awarded patents and patent applications.
Aurelia brings more than 25 years of healthcare industry experience with expertise in business development, strategy and global marketing including P&L responsibilities spanning diverse geographies (Europe, Middle East, Japan, Asia Pacific). She held global leadership roles for amongst others Novartis, led multiple enterprise transformation programs and implemented strategic growth initiatives to achieve business goals while fostering the culture of inclusion and enterprise mindset.
Aurelia is known for her unique understanding of both the patient and business side of the industry, as well as for the determination and empathy she brings due to the personal experience of losing several members of her family to difficult-to-treat diseases. She has found tremendous success in her belief that when you put the patient and the team first, the business achievements follow. She is pursuing a meaningful mission to work with teams who envision to cure difficult-to-treat diseases.
Sybrand has a background in engineering and has worked in the biotechnology field for over 20 years. He has been responsible for the upstream process development and technology transfer for several phase I/II (viral vector) and phase III (antibody) projects. Sybrand joined Amarna in January 2011 and is responsible for ensuring that all projects progress smoothly and meet their timelines and milestones.
Henk is a Physician Scientist with more than 15 years of experience in the pharmaceutical industry and clinical Development. This includes different expert roles as a member of the submission teams that reached worldwide approvals of Rasitrio™, Entresto™, and Kisqali™. He has broad cross-indications experience in clinical outsourcing GCP inspections and CAPA implementation/execution. Henk is currently the CEO and Medical Director of Amarna Therapeutics.
Thomas was the co-founder of Recipharm AB, a Swedish pharmaceutical contract development and manufacturing company, in 1995. Under his leadership, the organization grew to be a major player with approx. 9000 employees, at 30 facilities located in ten countries, offering pharmaceutical companies full service by taking products all the way from early development to commercial production. Thomas is currently President at life-science investor Flerie Invest and at the Board of several private and public companies including Kahr Medical Ltd, Prokarium Ltd, Toleranzia AB, Nanologica AB, NorthX Biologics AB and Sixera Pharma AB.
Thomas has a graduate degree from Linköping Institute of Technology, Sweden.
Bernd has over 25 years' experience in pharmaceutical research and drug development, having held leadership roles at Merck/Merck Serono, Sanofi-Aventis, Aventis and Hoechst Marion Roussel. He has expertise in a broad range of disease areas including oncology, immuno-oncology, immunology, neurological disorders and cardiometabolic diseases. In the eight years to 2013, he worked at Merck/Merck Serono, becoming a member of the Board and Executive Vice-President, Global Research & Early Development. He was responsible for a budget of 1 billion euros and a global team of over 2,500 associates. In his last three years at Merck Serono, he led the successful growth of the company's R&D portfolio, with over 70 programmes, doubling the number of Phase II assets in this period. Bernd is currently Chairman of OMEICOS Therapeutics and GeneQuine Biotherapeutics and a board member of Redx, BioMedX, Enlivex Therapeutics as well as an advisor to the board of KAHR Medical.
Bernd was awarded his PhD in Biochemistry and Physiological Chemistry from the University of Konstanz, Germany.
Maarten is a bio-entrepreneur working primarily in oncology and rare diseases. He did basic research at Lund University, Harvard Medical School and Uppsala University. He worked as a sell-side analyst at two investment banks: Aragon and Swedbank Markets. For the last 15+ years he has been involved in clinical development at Sanofi and Sobi, before co-founding Cormorant Pharmaceuticals. Cormorant brought a mAb against IL-8 into the clinic as an immune-oncology agent and was sold to BMS in 2016. In the same year he co-founded Buzzard Pharmaceuticals, where a potent IL-1 inhibitor is being developed as an immune oncology agent. He also serves as CEO of Sixera Pharma, developing a NCE treatment for the rare skin disease Nethertons, and is VP Medical Strategy and Innovation at Camurus. He currently also serves on the boards of Atrogi, OxThera, Cavis Technologies and Beactica.
Maarten holds a MD as well as a PhD from Lunds University, Sweden.
Pim is the CEO and co-founder of the Dutch IT company Schuberg Philis and has been one of Amarna’s pioneering informal investors for over 10 years. He was managing director in the Netherlands of SiteSmith Inc., a US-based Internet infrastructure management services company. In 2000 SiteSmith Inc. was acquired by Metromedia Fiber Network Inc. (MFN) in a transaction valued at approximately $1.4 billio, following which he became Senior Vice President of MFN’s European Region. In a management buyout by Pim and two partners in 2003 the Benelux subsidiary of MFN Inc. became Schuberg Philis. Before this, Pim held several senior executive positions at the Dutch IT companies Pink Elephant and PinkRoccade (now part of KPN).
Pim holds a master’s degree in Computer Engineering and Economics from the Hogeschool IJsselland, Netherlands.
Markwin is an independent entrepreneur with 25 years of experience, in the fields of research, development, operations and management. After transferring from the US academical to the Dutch biopharmaceutical sector, Markwin served, among several other management positions, four years as CSO at the Dutch biotech company AM-Pharma, with responsibility for R&D. In 2013 he consulted with the Dutch biotech T-Cell Factory (TCF) where he played a key role in the acquisition of the company to form Kite Pharma EU B.V. Markwin following joined Kite in 2015 as VP Operations where he managed the R&D of new T cell receptors alongside the implementation of Kite’s new manufacturing facility for cell therapy in the Netherlands. In addition, Markwin is former chairman of the supervisory board of HollandBio, the branch association for Biotech in the Netherlands and supervisory board member at ISA Pharmaceuticals in Leiden.
Markwin was awarded a PhD in Molecular Immunology from Leiden University in the Netherlands.
A Professor of Pathology, Anatomy and Cell Biology at Thomas Jefferson University and the Sidney Kimmel Cancer Center in Philadelphia at Thomas Jefferson University, Philadelphia, Pennsylvania, for the last 30 years, Professor Strayer is also an attending pathologist at Thomas Jefferson University-Affiliated Hospitals.
His career in clinical research has spanned immunology, pathology, virology and gene therapy, including studying the mechanisms of viral oncogenesis and virus-related immunosuppression and the study of clinical and immunological aspects of pulmonary surfactant as therapy for respiratory distress syndrome and the development. In applied virology and translational clinical trials his work has focused on viral gene delivery vehicles for potential application in treating diverse human diseases. This involved studying virus-host cell interactions and the impact on replication. His laboratory re-engineered simian virus-40 (SV40) for use as a gene delivery vehicle including exploiting its ability to evade the immune system. His team has modified the SV40 genome to carry genes of therapeutic interest, and used the resulting vectors effectively to deliver those genes to many cell types and organs, including bone marrow stem cells, neurons, liver, lung and other organs, demonstrating that repeated injection of SV40-derived vectors in vivo is feasible, effective and safe.
The significance of his work in gene therapy led to his appointment as Founding Chair of the NIH Gene and Drug Delivery Study Section, and countless presentations at national and international meetings.
He has held prior positions at Washington University, St. Louis, Missouri, the University of California, San Diego, Yale University and the University of Texas, Houston. He is the author over 150 original articles, over 200 abstracts and over 75 invited and review articles, and books, and is editor of a major textbook of Pathology (Rubin's Pathology: Mechanisms of Human Disease), published by Wolters-Kluwer.
He has an undergraduate degree in chemistry, from Cornell University, Ithaca, New York and received his PhD in immunology and MD degree from the University of Chicago, Chicago, Illinois.
Ben Samelson-Jones, MD, PhD is an Assistant Professor of Pediatrics and a member of the Gene Therapy and Vaccine (GTV) Graduate Program at the University of Pennsylvania. He is an attending pediatric hematologist and member of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics at The Children's Hospital of Philadelphia (CHOP). And he is the Associate Director of the Clinical In Vivo Gene Therapy group at CHOP.
He provides medical care for patients with blood diseases with a special interest in hemophilia and other bleeding disorders. He is a principal investigator on clinical gene therapy trials for hemophilia A and hemophilia B and has translational laboratory focused on developing novel treatments for bleeding disorders. His laboratory is supported by funding from the National Heart, Lung, and Blood Institute, the National Hemophilia Foundation, as well as by a number of sponsored research agreements with biotech partners. His research includes defining the biochemical mechanism of the hyperactivity of factor IX (FIX) Padua, which is used in all current gene transfer clinical trials for hemophilia B. He has published over 30 peer-reviewed papers including in The New England Journal of Medicine, Blood, and Molecular Therapy and filed for three patents. He is an abstract reviewer for the American Society of Hematology (ASH) and the International Society of Thrombosis and Haemostasis (ISTH) and is currently a member of the Translational Research Committee of the American Society of Gene and Cell Therapy (ASGCT).
He received his undergraduate degree in physics from Amherst College and his MD and PhD from Albert Einstein College of Medicine. He completed residency at the Children’s National Medical Center and fellowship at CHOP.
Dr. Kishimoto is a highly accomplished immunologist and drug developer with over 30 years of experience in biopharma. He is a creative scientist and strategic thinker driving discovery of innovative therapeutic modalities and out-of-the-box solutions to development challenges. His expertise is in immune tolerance, autoimmune diseases, vaccines, immunogenicity, and gene therapy. Over the course of his career, Dr. Kishimoto has had broad experience in developing small molecule drugs, vaccines, antibody-based therapies, enzyme therapies, sugar-based therapies, and gene therapies.
Dr. Kishimoto was most recently the Chief Scientific Officer of Selecta Biosciences, a biotechnology company developing a novel technology to induce immune tolerance. During his 12+ years at Selecta, he led the discovery and development of a novel nanoparticle-based antigen-specific immune tolerance technology which was successfully validated in phase 3 clinical trials.
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, where he led programs in leukocyte cell adhesion.
Dr. Kishimoto received his doctoral degree in Immunology from Harvard University in the laboratory of Dr. Timothy Springer, where he cloned the CD18 gene and characterized the molecular basis of leukocyte adhesion deficiency. He completed his post-doctoral training at Stanford University, where he identified antibodies that defined human L-selectin and discovered the dynamic shedding of L-selectin upon neutrophil activation. Dr. Kishimoto has published 100 peer-reviewed articles in scientific journals, including first and/or last author publications in Nature, Science, Cell, and the New England Journal of Medicine.
Didac Mauricio, MD PhD, is Director of the Department of Endocrinology & Nutrition, Hospital de la Santa Creu i Sant Pau in Barcelona, and Professor with the Faculty of Medicine, University of Vic & Central University of Catalonia (UVic/UCC).
He is leading the Diabetes Research Group at his current institution, that belongs to the excellence diabetes research network CIBER of Diabetes and Associated Metabolic diseases (CIBERDEM), Instituto de Salud Carlos III, in Spain. He has published over 330 peer-reviewed articles (WoS H-index: 52), and has contributed to multiple books. He has received the highest awards from the Spanish Society of Diabetes and the Spanish Society of Endocrinology & Nutrition. He is currently vice-president of the Spanish Society of Endocrinology & Nutrition (term expiring 2025). He has also served as a member in several local and international committees. D. Mauricio has been principal investigator of multiple research projects funded by national and international agencies. He keeps a strong interest on current clinical issues in diabetes management, including randomised clinical trials and clinical research on diabetic complications, as well as in studies on real-world evidence.
D. Mauricio is a member of the editorial board of several scientific journals and is currently a member of the Guideline Oversight Committee of the European Association for the Study of Diabetes.
Luiza Caramori, MD, MSc, PhD is a Staff Physician at the Institute of Endocrinology and Metabolism at Cleveland Clinic, and at the Department of Cardiovascular and Metabolic Sciences, Lerner research Institute, Cleveland, and an Adjunct Associate Professor at the University of Minnesota. Dr. Caramori received her MD degree in Brazil (1990) and did her fellowships in Endocrinology and Diabetes in Brazil and in the United States. After receiving her Master’s in Sciences degree (1997), Dr. Caramori completed her research training in diabetic kidney disease (DKD) at the University of Minnesota (1998-2002), initially sponsored by the Brazilian government and later by JDRF. Dr. Caramori’s clinical passion lies in providing outstanding care to patients with diabetes. She was the director of the Joint Commission Accredited Inpatient Diabetes Service at the University of Minnesota Medical Center between 2015-2022, when she relocated to Cleveland Clinic and the Lerner Research Institute.
Dr. Caramori’s main research interests include studies on the relationships between kidney structure and function, early molecular and structural predictors of DKD, and clinical trials studying repurposed and new drugs for the prevention and treatment of DKD. Dr. Caramori has authored more than 60 publications in peer- reviewed journals and 20 book chapters. She has been funded by grants from the National Institutes of Health (NIH), JDRF, and the National Kidney Foundation (NKF) of Minnesota, among others. Currently, Dr. Caramori is the principal investigator of NIH R01 grant to study protective factors in DKD and the Co-PI of a NIH U01 grant as part of the Kidney Precision Medicine Project (KPMP).
Dr. Caramori served as Chair for the diabetic nephropathy subcommittee for the American Diabetes Association (ADA) Scientific Sessions (2018-2020). She was a member of the NKF - Kidney Disease Outcomes Quality Initiative (KDOQI) work group - and helped develop the 2007 Clinical Practice Guidelines and Recommendations for Diabetes and CKD. She is also a member of the Kidney Disease: Improving Global Outcomes (KDIGO) task force. She also volunteers her time toward important initiatives of JDRF, ADA, and American Society of Nephrology.
Sylvaine You, PhD, is a distinguished researcher specializing in the immune mechanisms driving type 1 diabetes (T1D) and the development of therapeutic strategies. She obtained her PhD in 2000 from the University of Nantes, France, where she focused on T-cell immune responses towards porcine islet xenografts. Following her PhD, Dr. You completed a postdoctoral fellowship in the laboratory of Chih-Pin Liu at City of Hope Medical Center in California. There, she analyzed autoantigen-specific CD4+ T cells using MHC class II tetramers in the NOD mouse model of T1D. In 2003, she joined the team of Pr. Lucienne Chatenoud at Necker Hospital in Paris. By 2007, she secured a permanent position at the National Institute of Health and Medical Research (INSERM) and was promoted to senior research scientist in 2011. In 2017, Dr. You joined the Cochin Institute in Paris, where she co-leads the Diabetes & Autoimmunity Research Laboratory (DeARLab) with Prof. Roberto Mallone. She was promoted to INSERM Research Director in 2021 and currently leads a team of 19 researchers developing both experimental and clinical studies on T1D. Dr. You's expertise lies in understanding the pathogenic and regulatory mechanisms that control autoreactive T cells in human and mouse models of T1D. Her research focuses on immune strategies to promote and restore tolerance using biological agents such as CD3 monoclonal antibodies, low-dose IL-2, CD28 antagonists, and Fc-fused preproinsulin. Some of these strategies have shown promising results in T1D clinical trials. In April 2023, Dr. You and Prof. Mallone established a satellite laboratory at the Indiana Biosciences Research Institute (IBRI) in Indianapolis. This lab focuses on protecting beta cells from autoimmune destruction and identifying therapeutic targets.
Areas of Expertise:
● Autoimmunity, type 1 diabetes, T cells, immunoregulation, immune tolerance, immunotherapies, mouse models, islet transplantation
R. Mallone received his MD PhD degree from the University of Turin, Italy. After a Postdoc with Jerry Nepom at the Benaroya Institute in Seattle, he moved to Paris where he is currently Professor of Immunology at Université Paris Cité, Diabetologist at the Cochin Hospital and leads a research team at the INSERM Cochin Institute. He is also director of a satellite research lab at the Indiana Biosciences Research Institute in Indianapolis. His research spans from preclinical studies with human samples and mouse models to clinical trials. It focuses on autoimmune T cells and the understanding of type 1 diabetes mechanisms in order to develop T-cell-based biomarkers and therapeutics.
Major contributions to the field include the demonstration of the role of CD8+ T cells in beta-cell autoimmunity, the description of the antigen display (immunopeptidome) of human beta cells, the development of T-cell assays to clarify disease mechanisms and therapeutic modifications, and the exploration of perinatal vaccination strategies. More recently, he described a universal state of ‘benign’ islet autoimmunity imprinted in the thymus and the antiviral T-cell responses mounted against Coxsackieviruses.
Desmond Schatz, MD is Professor of Pediatrics, Medical Director of the Diabetes Institute and Director of the Clinical Research Center within the CTSI at the University of Florida, Gainesville. He served as Interim Chair of Pediatrics from 2019-2022 and as President of Science and Medicine of the American Diabetes Association in 2016. Dr. Schatz has been involved in Type 1 diabetes research since the mid 1980’s and has published over 400 manuscripts, the majority related to the prediction, natural history, genetics, immunopathogenesis and prevention of the disease, as well as the management of children and adolescents with Type 1 diabetes. He is the Principal Investigator (PI) on several NIH awards. He is the PI of the University of Florida Clinical Center participating in the NIH-funded TrialNet (a founding member of the DPT-1 in 1994). He is co-PI on a Program Project Grant seeking to understand the immunopathogenesis of Type 1 diabetes, and serves as PI for the NIH-funded international newborn genetic screening (TEDDY) program in North Central Florida. He is the PI on the NIH-funded Human Islet Research Network (HIRN) Grant evaluating novel markers and mechanisms of beta cell destruction. Dr. Schatz has served in numerous capacities (Committees, Study Sections) for the American Diabetes Association, JDRF, and NIH. He is the recipient of multiple awards including the Mary Tyler Moore and S Robert Levine JDRF Excellence in Clinical Research Award, the 2009 Cure Award from the American Diabetes Association, the 2010 Public Policy Leadership Award, the prestigious Banting Medal for service from the American Diabetes Association, the ISPAD Prize for achievement (the highest honor of the International Society for Pediatric and Adolescent Diabetes) both in 2016 and, more recently in 2021, the distinguished George Eisenbarth Award from the JDRF for outstanding research advances toward the prevention of Type 1 diabetes. In 2022 he was elected into the prestigious Academy of Science, Engineering and Medicine of Florida (ASEMFL).
Professor of Clinical Diabetes and Metabolism, Cardiff University School of Medicine.
Senior Clinical Researcher, University of Oxford.
Director, Cardiff Joint Research Office.
Colin Dayan trained in medicine at University College, Oxford, and Guy’s and Charing Cross Hospitals in London, UK before obtaining a PhD in the immunology of Graves’ Disease in the Laboratory of Marc Feldmann. He then spent a year as an endocrine fellow at the Massachussetts General Hospital in Boston, USA before completing his specialist training in diabetes and endocrinology as a Lecturer in Bristol. In 2010, he was appointed to the Chair of Clinical Diabetes and Metabolism and Head of Section at Cardiff University School of Medicine and in 2020 as part-time Senior Clinical Researcher in the Nuffield Department of Medicine at the University of Oxford.
Prof Dayan has a long established interest in translational research in the immunopathology of type 1 diabetes promoting progress to “insulin free T1D”. He has published first-in-man clinical studies on the development of peptide immunotherapy and the use of nanoparticles in T1D as well as pioneering the use of lymph node and injection site sampling to monitor the response to therapy. Since 2015 he has been a leading member of the UKT1D-Research Consortium which has hosted more than 15 clinical trials in early T1D over 30 sites. He is currently leading on novel trial designs including the T1D Plus platform trial in Europe and Australia to rapidly test combinations of therapies. In collaboration with the Critical Path Institute, he led the TOMI consortium of 21 clinical trials to define the relationship between C-peptide preservation and clinical endpoints and build an FDA/EMA approved clinical trial simulation tool. He is now seeking to define novel endpoints in preclinical T1D as part of the EDENT1FI European programme. Prof Dayan is a committed advocate to finding ways to bring beta cell preservation and delaying the need for insulin therapy into routine clinical practice. In this regard he supported the application for regulatory approval of Tzield to the FDA in 2021 and is working with colleagues to introduce immunotherapy and population screening into the UK.