Outstanding achievement award
Christof von Kalle & Manfred Schmidt (†)
Prof. Dr Christof von Kalle is the BIH Chair for Clinical Translational Sciences, and Founding Director of the Joint Clinical Study Center (CSC) of Charité and BIH
Since June 2019, Christof von Kalle has been BIH Chair for Clinical Translational Sciences at Charité Berlin and Founding Director of the common Clinical Study Center (CSC) of Charité and the Berlin Institute of Health (BIH). Since February 2019, he has been Member of the Advisory Council on the Assessment of Developments in the Health Care Sector (Federal Ministry of Health, BMG, Germany). From July 2005 to May 2019, Christof von Kalle was Head of the Department of Translational Oncology at the German Cancer Research Center (DKFZ). Moreover, he was Managing Director and Head of the Department of Translational Oncology of the National Center for Tumor Diseases (NCT) Heidelberg from July 2005 to March 2018. From April 2018 to May 2019, he acted as Chief Research Officer at Sidra Medicine, Doha, Qatar.
As a physician scientist with a clinical background in haematology/oncology and over 200 high-impact publications, Christof von Kalle is an internationally renowned leading scientist in stem-cell research, mutation analysis, and gene transfer. He studied Medicine at Cologne University and obtained his doctorate at the Department for Internal Medicine of Cologne University. Christof von Kalle received the Eva Luise Köhler Award for Rare Diseases (2011) and the Pioneer Series Award/Human Gene Therapy (2014) among others.
Dr. Manfred Schmidt (†) was a pioneer in the gene and cell therapy field, with more than 25 years of experience in the sector. He was the Co-Founder and CEO of GeneWerk GmbH, based in Heidelberg, Germany. His most recent academic role was as Section Head of Molecular and Gene Therapy at the German Cancer Research Center, also part of the National Center for Tumor Diseases in Heidelberg, Germany.
Manfred obtained his doctoral degree from the University of Freiburg, and a Diploma degree in Biology in University Hohenheim, Germany. He authored more than 120 peer-reviewed publications in international high-impact journals.
Manfred was instrumental in the development of Linear Amplification-Mediated PCR (LAM-PCR), which allowed the highly sensitive identification and sequencing of unknown DNA or RNA sequences, which are flanked by a known DNA or RNA region.
The application of LAM-PCR and its follow up development have been widely used in vector safety assessment of cell and gene therapy. The analysis of the efficiency and safety of vector systems for clinical gene therapy, combined with the application of high throughput sequencing technologies and the development of project specific bioinformatical analysis and data management systems, is his major research area.
Manfred passed away on 13 January 2022, aged 54. All of us at ESGCT extend our sincere condolences to his family, friends and colleagues.
Young Investigator Awards
Eliana Ruggiero received her Ph.D. from the University of Heidelberg in 2013, trained at the laboratory of Christof von Kalle/Manfred Schmidt, where she focused on the characterization of the TCR repertoire by high-throughput sequencing. In 2015, she joined Chiara Bonini’s lab at Ospedale San Raffaele (Milan) with the aim of identifying new tumor-specific TCRs for T cell engineering. Her work resulted in 32 publications and 6 patents. Her research has been supported by national and international fundings (Italian Ministry of Health, Marie Sklodowska-Curie, iCARE-AIRC, Helmholtz Alliance Immunotherapy of Cancer) and awards (ASGCT Excellence in Research Award, WIC-SITC Young Investigator Award).
Raul Torres-Ruiz's research career has focused on genome engineering tools development broadly applicable to studying and treating human genetic diseases. Delivery of gene editing tools is perhaps one of the main barriers to the widespread use of genome editing technologies. He has been involved in several research projects to engineer viral delivery systems that can be combined with genetic editing nucleases such as CRISPR/Cas9. In the last years, he has focused his efforts in the development of new therapeutic approaches -based on gene editing and gene therapy- for the treatment of rare cancers and monogenic clotting disorders.
Dr. Jude Samulski received his PhD in medical microbiology and immunology from the University of Florida. His graduate work (1978-82) demonstrated the first use of AAV as a viral vector and culminated in the development of the first FDA AAV drug approved for blindness. Dr. Samulski has worked with AAV for over 40 years, including a post doc with Dr Tom Shenk at Princeton, and, for 25 years, he was director of the University of North Carolina Gene Therapy Center. At UNC, he has focused on development of AAV as a gene delivery system, which has led to the first long-term gene delivery in humans. His development of novel AAV variants as alternative vectors and improvements in AAV production facilitated the first clinical trial for gene delivery to the brain and his development of the first chimeric AAV facilitated gene delivery for muscular dystrophy. He continues to derive delivery systems for safe, efficient use in human gene therapy to facilitate the progression and translation of gene therapy research into clinical trials for treatment of human diseases.
Dr. Samulski has advanced therapeutics into human clinical trials for hemophilia, Duchenne muscular dystrophy, giant axonal neuropathy, Pompe, Canavan and heart failure. He published over 500 peer reviewed articles & holds more than 200 patents related to AAV technology.