Success Factors in Creating Science- Based Business. Saskia Biskup, Tuebingen, Germany

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1 Success Factors in Creating Science- Based Business Saskia Biskup, Tuebingen, Germany

2 The biggest motivation for me is: to translate research findings into a clinical setting to risk and to succeed to work in a team 2

3 The technical revolution Next Generation Sequencing 3

4 Why Founding a Company Making things happen Creating something Taking responsibilities Choosing own team 4

5 The Team of 110 Dr. Dr. S. Biskup Dr. D. Biskup Managing Directors Dr. J. Ködding Laboratory T. Scheurenbrand Dr. F. Battke Bioinformatics & IT Dr. K. Hörtnagel Diagnostics Automation Data Processing Administration Customer Solutions IT Infrastructure Genetic Counseling Neurodegenerative and Neuromuscular Diseases QM & Accreditation NGS Routine Project Analysis Eye Diseases Epilepsy & Migraine Project Management Research & Development Software Development Tumor Syndromes Single Gene Testing and Exome Diagnostics Sales & Marketing Sanger Lab 5

6 What else is needed An idea and a vision State of the art equipment A business concept 6

7 CeGaTs development and growth is accompanied by several milestones Employees 7,500 Patients p.a. 4, , , Foundation CeGaT GmbH in Tuebingen Introduction of Diagnostic Panels Accreditation according to DIN EN ISO 15189:2007 Awardee Best German Start-Up Company Strategic interest B.Braun (20%) Finalist Clarity Challenge Introduction of Tumor Diagnostics Ernst & Young Entrepreneur of the Year Start up Beginning with CAP/CLIA Certification process Foundation of B.Braun CeGaT, LLC together with B.Braun Move in own building Tübingen 7

8 Skills and technology Skills Human geneticists (MDs / PhDs) Bioinformaticians (PhDs / M. Scs.) Scientific personnel Molecular Biotechnologists and Biochemists Laboratory Technicians Business Administration Technology Sequencing platforms 3x HiSeq 2500, Illumina 1x MiSeq, Illumina 1x Ion Torrent PGM, Life Technology 3x 96 Capillary-Sequencer, ABI 3730xl, Life Technology High throughput automation QIAsymphony, QIAGEN Fragment Analyzer, Advanced Analytical Bravo Liquid Handling Workstation, Agilent Technologies MICROLAB STAR Liquid Handling Workstation, Hamilton QuantStudio 12K Flex, Life Technologies 8

9 What do we do: We transfer the genetic code to diagnosis and therapy Using next generation sequencing in a clinical setting High probability to find the causative gene variation Sequencing of relevant genes Bioinformatic analyses Medical interpretation 9

10 One example 10

11 11

12 The Clarity Challenge 23 Teams worldwide were selected to participate Participants (1/2) BGI (Shenzhen, China) Brigham and Women's Hospital, Division of Genetics (Boston, Massachusetts) British Columbia Cancer Agency (Vancouver, Canada) Children's Hospital of Eastern Ontario (Ottawa, Canada) Clinical Institute of Medical Genetics (Ljubljana, Slovenia) Genedata AG (Basel, Switzerland) CeGaT (Tübingen, Germany), Institute of Pathology, Bonn, Genomatix (Munich) Genome Institute of Singapore Agency for Science, Technology and Research (A*STAR) (Biopolis, Singapore) HudsonAlpha Institute for Biotechnology (Huntsville, Alabama) Institute for Systems Biology (Seattle, Washington) IRCCS Casa Sollievo della Sofferenza (San Giovanni Rotondo, Foggia, Italy) National Institutes of Health (Bethesda, Maryland) NextBio (Santa Clara, California) Omicia Inc/University of Utah (supported by LocusDev Inc) (Emeryville, California) Pearlgen (Chapel Hill, North Carolina) Radboud University Nijmegen Medical Center (Nijmegen, Netherlands) 12

13 Participants (2/2) The Clarity Challenge Sanofi (Cambridge, Massachusetts) Science For Life Laboratory (SciLifeLab), Karolinska Institute (Solna, Sweden) Scripps Genomic Medicine, Scripps Translational Science Institute(San Diego, California) Seven Bridges Genomics (Cambridge, Massachusetts) SimulConsult / Geisinger (Chestnut Hill, Massachusetts / Danville, Pennsylvania) SNPedia (Potomac, Maryland) Strand Life Sciences (Bangalore, India) Tel Aviv University (Israel) The Medical College of Wisconsin (Milwaukee, Wisconsin) The Research Institute at Nationwide Children's Hospital (Columbus, Ohio) The University of Texas Health Science Center at Houston, Universidad de Cantabria (Santander, Spain) University of Iowa (Iowa City, Iowa) Yale School of Public Health, Division of Biostatistics (New Haven, Connecticut) 13

14 Bostons CLARITY Challenge Finalist 2012 Bostons CLARITY Challenge Goals of CLARITY challenge: Identification of best methods and practices for the analysis, interpretation and reporting of individuals DNA sequence data Provision of the most meaningful results to clinicians, patients and families Participants: 23 teams, representing 10 countries [ ] First prize went to the Division of Clinical Genetics at Brigham and Women s Hospital in Boston, which received $15,000 for what judges said was the best blend of analysis, clarity and utility in the reports it generated for the three families. A teams from the University of Iowa [..] and a German team that was the only group to accurately identify every potential genetic mutation in all of the families. 14

15 The Clarity Challenge Results 15

16 The vision Every patient gets genetic testing before treatment targeted and efficient treatment less side effects lower costs 16

17 Every tumor is different Tumor Entities Targetable Mutations 93.5% of cases could be analyzed (tumor content >10%) Targetable mutations were detected in 79% of samples On average, 1.7 targetable mutations were detected per patient 17

18 Tumors: From diagnostics to personalized treatment

19 Development of a personalized peptide vaccine Sample Extraction Sequencing & Data Analysis Immuno- Informatic Analysis Peptide Selection Synthesis & Vaccination Isolation of DNA Identification of Prediction of Selection of the Personalized from tumor and cancer-specific immunogenic optimal set of treatment normal tissue mutations and mutated peptides peptides HLA types

20 Summary Every patient is individual Precision diagnostics Precision medicine 20

21 CeGaT GmbH Paul-Ehrlich-Str. 23 D Tübingen Tel: / Fax: / info@cegat.de