Stem cells induced from blood or skin cells are generated from each patient, which are then reprogrammed into brain cells that are genetically and functionally identical to the brain cells in the patient's head.
The patient-specific brain cells are precisely characterized by molecular and functional investigations.
Data from individual patients and the corresponding disorder cell models will enable the development of new therapeutic approaches and suitable diagnostic tools.
Team
Carsten Pfeffer, PhD
Carsten Pfeffer, PhD
Carsten Pfeffer, PhD
Co-Founder and CEO
Gaia Novarino, PhD
Carsten Pfeffer, PhD
Carsten Pfeffer, PhD
Co-Founder and Scientific Advisor
Christoph Bock, PhD
Carsten Pfeffer, PhD
Christoph Bock, PhD
Co-Founder and Scientific Advisor
Eva Reinthaler, PhD
Melanie Pieraks, MSc
Melanie Pieraks, MSc
Research Scientist
Melanie Pieraks, MSc
Melanie Pieraks, MSc
Melanie Pieraks, MSc
Research Specialist
Lab Manager
Karin Stecher, PhD
Melanie Pieraks, MSc
Karin Stecher, PhD
Research Scientist
Federico Salaris, PhD
Maria Fraga Pires, MSc
Federico Salaris, PhD
Research Scientist
Camilla Bosone, PhD
Maria Fraga Pires, MSc
Federico Salaris, PhD
Research Scientist
Maria Fraga Pires, MSc
Maria Fraga Pires, MSc
Maria Fraga Pires, MSc
Research Specialist
Press release
Meyer Hospital Florence and Neurolentech GmbH announce clinical collaboration to produce cellular models for genetic epileptic encephalopathies
Florence - Italy; Klosterneuburg - Austria, August 8, 2022 –
Meyer Hospital Florence (https://www.meyer.it/) and Neurolentech GmbH (https://neurolentech.com) today announced a new collaboration to generate patient-specific cellular models for genetic epileptic encephalopathies. Their aim is to investigate disease mechanisms at the cellular, functional and molecular level with the goal to develop tools and generate data to foster therapeutic drug development.
Genetic epileptic encephalopathies are hundreds of disorders, each affecting the expression and function of a gene that influences the development, connections and performances of neuronal circuits. Currently, there are no adequate targeted drug treatments for genetic epilepsies. Research into the molecular and cellular mechanisms of genetic epilepsies is therefore needed to develop therapies in order to help patients and their families.
The Center of Excellence for Neurosciences of the Children’s Hospital Meyer, Florence, is internationally renowned for its expertise in epilepsy with its EEG, MRI, neuropsychological, genetic aspects and functional characterization of identified mutations using neurobiological approaches. The main area of expertise of Prof. Guerrini’s team is the medical care of patients with rare and complex epilepsies, the presurgical assessment and surgical treatment of children with drug resistant epilepsy, and the identification of new causative genes for developmental and epileptic encephalopathies and new genotype/phenotype correlations for these conditions.
Neurolentech is specializing in rapid and cost-efficient generation and characterization of patient-specific cellular disease models for neurodevelopmental disorders, bridging the gap between clinical research and drug development. Under the terms of the collaboration agreement Neurolentech will receive primary epilepsy-patient cells in order to develop patient-specific neuronal cultures and characterize them using omics-approaches as well as functional assays with the goal of identifying biomarkers, biochemical pathways and targets that will facilitate pre-clinical drug discovery for genetic epilepsies.
"We are thrilled to enter this collaboration with Prof. Guerrini at the Center of Excellence in Neurosciences of the Meyer Hospital Florence. Genetic epilepsies are a disease area of high unmet medical need. The excellence in clinical and genetic diagnostics of Prof. Guerrini´s team together with our high-quality detailed characterization of patient-specific disease models forms the foundation for understanding disease pathology and takes the first hurdle towards targeted drug development. Our mutual long-term commitment will put the individual patient at the nexus of research while speeding up commercial drug development benefiting patients and their families," said Dr. Pfeffer, co-founder and CEO of Neurolentech. Dr. Novarino, co-founder and professor of neuroscience at IST Austria added, "Genetic epilepsies are devastating diseases with a huge burden on patients, families and society. Basic research using patient-specific models will push the boundaries of precision medicine for epileptic patients. We are enthusiastic to collaborate with such innovative, engaged and interconnected clinical partners in order to advance the best research for genetic epilepsies."
Prof. Guerrini, Full Professor of Child Neurology and Psychiatry, and Director of the Center of Excellence for Neurosciences of the Children’s Hospital Meyer said, “This is a highly needed step forward for research in the epileptic encephalopathies. Through this collaboration with Neurolentech GmbH it will be possible to carry on morphological, electrophysiological and pharmacological studies directly on neural cells derived from the patients and move forward with the most appropriate experiments towards precision medicine”.
ABOUT MEYER Children’s Hospital
Meyer Children’s Hospital is a 250-bed public hospital that is part of the Italian National Health System and is integrated with the University of Florence, with which it carries out healthcare, teaching, and research activities. Meyer is a highly specialized children’s hospital and is a national reference center for complex diseases, with all pediatric medicine and surgery services available. The hospital aims for excellence in all of its activities, mixing modern technology, research and new therapies with a holistic approach toward the care of young patients and their families. The hospital is currently undergoing a rapid phase of expansion, including a new training center including an advanced simulation center (SimMeyer), a family center, and an outpatient center. The strategic planning for a new research center is underway. Meyer is a member of the Italian Association of Children’s Hospitals (AOPI) and the European Children’s Hospitals Organisation (ECHO).
For more information, visit the www.meyer.it website.
ABOUT NEUROLENTECH GmbH
Neurolentech GmbH develops cellular disorder models for drug development and diagnostics focusing on Autism, Epilepsy and Intellectual Disability, neurodevelopmental disorders with a huge unmet medical need and large burden on patients, families and society. Neurolentech's precision medicine platform approach combines the generation of patient- and disorder-specific models with several molecular and physiological analysis technologies that will allow the directed development and validation of novel drugs and therapeutics. Neurolentech is supported by funds from the Austrian federal promotional bank (AWS, https://www.aws.at/), an investment from IST cube (https://ist-cube.com) as well as grants from TESS foundation (https://www.tessresearch.org/) and Foundation for USP7-related diseases (https://www.usp7.org/). Neurolentech GmbH was founded by Carsten Pfeffer, Gaia Novarino, Christoph Bock, together with IST Austria (ISTA, https://ist.ac.at/).
For more information visit: https://neurolentech.com/
Contact for MEYER HOSPITAL FLORENCE
Valerio Conti, PhD, Neuroscience Department, Children's Hospital A. Meyer, Head of the Neurobiology Lab.
Renzo Guerrini, MD, FRCP, FAES, Director of the Neuroscience Department, Children's Hospital A. Meyer.
Contact for NEUROLENTECH GmbH
Carsten Pfeffer, PhD
CEO and co-founder
Neurolentech GmbH
University Hospital Frankfurt and Neurolentech GmbH announce clinical collaboration to produce cellular models for neurodevelopmental disorders
Frankfurt/Main - Germany; Klosterneuburg - Austria, August 8, 2022 –
University Hospital Frankfurt (www.kgu.de) and Neurolentech GmbH (https://neurolentech.com) today announced a new collaboration to generate patient-specific cellular models for neurodevelopmental disorders such as Autism Spectrum Disorder(ASD). Their aim is to investigate disease mechanisms at the cellular, functional and molecular level with the goal to develop tools and generate data to foster therapeutic drug development.
ASDs are genetic disorders affecting the expression and function of hundreds of genes that influence the development and performance of neuronal circuits. Currently, there are no available drug treatments targeting the core symptoms of ASDs. Research into the molecular and cellular mechanisms of ASDs are therefore needed to develop therapies in order to help patients and their families.
The Department of Child and Adolescent Psychiatry, Psychosomatics and Psychotherapy (CAPPP) at the Center for Mental Health of the University Hospital Frankfurt is specialized in diagnosing and treating individuals with psychiatric conditions, especially ASD with the main aim of providing value to the patient’s and practitioners. Their research focuses on improving diagnosis, understanding prognosis and identifying new treatment options. Specifically, the Molecular Genetics Laboratory at the CAPPP is committed to elucidate the genetic drivers and their cellular pathogenic mechanisms behind neurodevelopmental disorders and translate this knowledge into clinical applications. To target the complexity of the goal, and to address the personalized approaches the CAPPP joins efforts with the highly innovative start-up Neurolentech.
Neurolentech is specializing in rapid and cost-efficient generation and characterization of patient-specific cellular disease models for neurodevelopmental disorders, bridging the gap between clinical research and drug development. Under the terms of the collaboration agreement Neurolentech will receive primary ASD-patient cells in order to develop patient-specific neuronal cultures and characterize them using omics-approaches as well as functional assays with the goal of identifying biomarkers, biochemical pathways and targets that will facilitate pre-clinical drug discovery for ASDs.
"We are excited to start this collaboration with Prof. Freitag and Prof. Chiocchetti at the Center for Mental Health, University Hospital Frankfurt. Our common goal to develop treatments for patients with neurodevelopmental disorders will put the individual in the center of research and development. Characterizing the disease within cellular models, together with high quality clinical and genetic diagnostics are the basis for understanding disease pathology and the first steps towards drug development. This long-term collaboration will advance both, academic research and commercial drug development for the benefit of patients and their families in a disease area of high unmet medical need," said Dr. Pfeffer, co-founder and CEO of Neurolentech. Dr. Novarino, co-founder and professor of neuroscience at IST Austria added, "Basic research into the mechanisms of neurodevelopmental diseases like Autism Spectrum Disorder will provide the most promising approach to help the patients. We are thrilled to work together with strong and well-connected clinical partners in order to advance the best research for ASDs."
Dr. Freitag, professor for Child and Adolescent Psychiatry and director of the Department of Child and Adolescent Psychiatry, Psychosomatics and Psychotherapy and Dr. Chiocchetti, professor for Translational Child Psychiatry and head of the Molecular Genetics Laboratory agreed that, “combining the excellent clinical characterization with high-end cellular models and methods, will significantly speed-up the identification and characterization of the underlying pathology. The collaboration with economic partners will tighten the link between patient care, pharmacological intervention and basic research. The focus has to be to increase our understanding of the diversity of the etiology underlying a complex disorder such as ASD. There is not one ASD, and thus there is no “one-size-fits” all solution. We are convinced that the collaboration with the highly innovative company Neurolentech, will pave the way towards a biology and data-driven personalized treatment approach”.
ABOUT THE CENTER FOR MENTAL HEALTH; Department of Child and Adolescent Psychiatry, Psychosomatics and Psychotherapy, FRANKFURT GOETHE UNIVERSITY HOSPITAL
The CAPPP currently has 48 inpatient and 30 day-care places, a large outpatient clinic and an autism therapy and research center. Children and adolescents with disorders from the entire spectrum of child and adolescent psychiatry and psychosomatics are treated. In addition to sound clinical training and activity, the clinic offers an excellent research environment in the areas of autistic disorders, aggressive and hyperkinetic behavior disorders as well as anxiety disorders. The Molecular Genetics Laboratory is investigating the molecular biology behind these disorders since more than ten years and significantly contributed to the identification of the genetic variants associated with the diagnoses. The CAPPP hosts one of the largest biobanks for neurodevelopmental disorders of the childhood in Europe, and combines molecular genetics and neuronal cell models with bioinformatics and machine learning. Intertwined with the clinical departments ensures high quality characterization of the disorder and close collaboration with clinicians and patients. The research on neurodevelopmental disorders is funded by the European commission, the German Ministry of Science and Education, the Hessian State and the German Research Foundation, among others. Prof. Dr. Dipl. Theol. Christine Freitag is director of the CAPPP and Prof. Dr. Andreas G. Chiocchetti is head of the Molecular Genetics Laboratory.
For more Information visit
https://chiocchettilab.uni-frankfurt.de/
ABOUT NEUROLENTECH GmbH
Neurolentech GmbH develops cellular disorder models for drug development and diagnostics focusing on Autism, Epilepsy and Intellectual Disability, neurodevelopmental disorders with a huge unmet medical need and large burden on patients, families and society. Neurolentech's precision medicine platform approach combines the generation of patient- and disorder-specific models with several molecular and physiological analysis technologies that will allow the directed development and validation of novel drugs and therapeutics. Neurolentech is supported by funds from the Austrian federal promotional bank (AWS, https://www.aws.at/), an investment from IST cube (https://ist-cube.com) as well as grants from TESS foundation (https://www.tessresearch.org/) and Foundation for USP7-related diseases (https://www.usp7.org/). Neurolentech GmbH was founded by Carsten Pfeffer, Gaia Novarino, Christoph Bock, together with IST Austria (ISTA, https://ist.ac.at/).
For more information visit: https://neurolentech.com/
Contact for CENTER FOR MENTAL HEALTH GOETHE UNIVERSITY HOSPITAL FRANKFURT
Prof. Andreas G. Chiocchetti
Head of Molecular Genetics Laboratory
Department of Child and Adolescent Psychiatry, Psychosomatics and Psychotherapy
Goethe University Frankfurt
Contact for NEUROLENTECH GmbH
Carsten Pfeffer, PhD
CEO and co-founder
Neurolentech GmbH
Partners
