- 중추신경계 모델과 중개연구 전략 -

중추신경계 모델과 중개연구 전략을 테마로 한 이 컨퍼런스 프로그램에서는 유망 전임상 연구 모델의 분석과 평가가 진행되고, 보다 정확하고 신뢰성 높은 전임상 시험의 실현을 향한 향후 경로 등이 논의됩니다.

Final Agenda

Recommended All Access Package:

11월 27일: 단세포 분석

11월 27일 Dinner Course: 쇼트코스 1:Drug Discovery 연구자를 위한 면역학 기초 강좌, 파트 1:면역계 개요

11월 28-29일: 리드 화합물의 최적화와 약물 독성 예측

11월 29-30일: 중추신경계 모델과 중개연구 전략

11월 29일 Dinner Course: 쇼트코스 4:Drug Discovery 연구자를 위한 면역학 기초 강좌, 파트 2:암면역과 자가면역

11월 29일(목)

12:00 Registration

12:20 Bridging Luncheon Presentation (Opportunity Available) or Enjoy Lunch on Your Own

12:50 Session Break

중추신경계 질환 치료제 Discovery의 획기적 성과

13:25 Welcome Remarks

Joel Hornby, BSc, Conference Director, Cambridge Healthtech Institute

13:30 Chairperson’s Opening Remarks

William Z. Potter, MD, PhD, Senior Advisor, National Institute of Mental Health, National Institutes of Health (NIH)

13:35 Principles for Delivering Breakthrough Drug Discovery for CNS Disorders

Stuart W. Hughes, PhD, Director and Head of Pharmacology, Biological Sciences, Vertex Pharmaceuticals

CNS drug discovery presents a set of unique challenges that renders this therapeutic area a particularly tough nut to crack. Whether it be identifying novel validated targets that are grounded in robust human biology, clearly defining the translational value of specific animal models or creating appropriate biomarkers that enable effective development of clinical molecules, delivering success has proven far from easy. In this talk, I will discuss a set of key principles that address all of these pivotal issues.

14:05 Testing the Diseased Brain on the Chip: From Gene Expression to Functional Phenotyping of Patient-Derived Neuronal Cultures

Dirk Schubert, PhD, Assistant Professor, Group Leader “Cellular Neurophysiology”, Cognitive Neuroscience Department, Donders Institute for Brian, Cognition & Behaviour, Radboud University Medical Clinic Nijmegen

Human induced pluripotent stem cell derived neurons (iNeurons) from control lines as well as from patients can be used to unravel the mechanisms that underlie impaired network function and synaptic communication in patients with neurodevelopmental disorders, such as intellectual disability. Studying neuronal network formation and maturation by investigating molecular, structural and functional parameters revealed robust disease phenotypes that offer the platform for testing pharmacological interventions.

14:35 Sponsored Presentation (Opportunity Available)

15:05 Interactive Breakout Discussion Groups

This session features various discussion groups that are led by a moderator/s who ensures focused conversations around the key issues listed. Attendees choose to join a specific group and the small, informal setting facilitates sharing of ideas and active networking. Details on the topics and moderators are available on the conference website.

16:05 Refreshment Break in the Exhibit Hall with Poster Viewing

줄기세포와 iPS 세포 기반 중추신경계 모델

16:45 Testing Drugs That Modulate Mitochondrial Biogenesis and Function in iPSC Models of Neurodegenerative Diseases

Michela Deleidi, PhD, Helmholtz Young Investigator Group Leader, DZNE

While mitochondrial dysfunction is emerging as key in neurodegenerative diseases, a central question remains whether mitochondria are actual disease drivers and boosting mitochondrial biogenesis and function ameliorates pathology. We outline how patient-derived induced pluripotent stem cells and genome editing can be used for modelling mitochondrial demise occurring in neurodegenerative conditions such as Parkinson’s and Alzheimer’s disease. We outline progress in screening for compounds that improve energy metabolism and ameliorate neurodegenerative phenotypes.

17:15 Exploiting Human Stem Cells to Recapitulate Pathological Signatures of Human Brain Microenvironment

Catarina Brito, PhD, Lab Head, Advanced Cell Models Lab – Animal Cell Technology Unit, iBET & ITQB-NOVA

We have been exploiting platforms based on spheroid culture in perfusion stirred tank bioreactors for generation of functional neurospheroids - 3D structures containing neurons, astrocytes and oligodendrocytes, derived from human induced pluripotent stem cells. Recent data on the characterization of the cell microenvironment generated within neurospheroids will be presented. The application of neurospheroids as in vitro models to address molecular defects associated with neurological disorders that affect neural microenvironment homeostasis and as potency assays for drug testing will also be discussed.

17:45 Modeling ALS with Patient-Specific iPSCs

Shila Mekhoubad, PhD, Scientist II, Translational Cellular Sciences, Biogen

Significant advances in human stem cell biology and neuronal differentiations have provided a new platform to study neurodegenerative disease-specific mechanisms and phenotypic outcomes in vitro. Here we describe our use of induced pluripotent stem cells (iPSCs) from patients with Amyotrophic Lateral Sclerosis (ALS) to establish new assays and tools that could help empower in vitro disease modeling efforts and contribute to the development and validation of novel ALS therapeutics.

18:15 Close of Day and Dinner Short Course Registration


19:0021:30 Recommended Dinner Short Course*

쇼트코스 4:Drug Discovery 연구자를 위한 면역학 기초 강좌, 파트 2:암면역과 자가면역

* Separate registration required.

11월 30일(금)

8:00 Registration and Morning Coffee

중추신경계 연구를 위한 동물 모델의 비교

8:25 Chairperson’s Remarks

Dario Doller, PhD, Senior Director, Exploratory Research, Sage Therapeutics

8:30 Nonclinical Models Supporting Orphan Drug Designations in Rare Neurodegenerative Conditions

Dinah Duarte, PhD, Assistant Professor, Lisbon University; Senior Assessor, INFARMED; PT Member, Committee for Orphan Medicinal Products, European Medicines Agency (EMA)

Many disease-specific animal models have been used to test emergent medicines in neurology. There is a body of evidence, however, that there is a substantial difficulty in choosing/accessing an optimal model or choosing measurements which would be truly informative of the product’s efficacy. We intend to present a critical revision of preclinical models that may be used to support orphan drug designations in rare neurodegenerative conditions, which are validated for each condition and to evaluate assays pertinent to the core features of selected conditions or otherwise relevant from the clinical standpoint.

9:00 Refining Use of Animal Models in CNS Disease to Promote Translation

Caroline Zeiss, PhD, Professor of Comparative Medicine and of Ophthalmology and Visual Science, Yale University

Translation of promising preclinical results in animal models of neurodegenerative disease is becoming an increasingly vexing issue. Study designs suitable for development of symptomatic therapies will be contrasted with key aspects of design needed to develop disease-altering therapies for neurodegenerative conditions, such as Alzheimer’s and Parkinson’s diseases. Fit-for-purpose choice of animal models will be illustrated, with emphasis on utility of model diversity to improve generalizability of animal data to humans.

9:30 Sponsored Presentation (Opportunity Available)

10:00 Coffee Break in the Exhibit Hall. Last chance for poster viewing.

중추신경계 연구의 성과를 치료법 개발로 중개

10:45 Translating Neuroscience into Treatments: De-Risking through Partnerships

William Z. Potter, MD, PhD, Senior Advisor, National Institute of Mental Health, National Institutes of Health (NIH)

Translating the findings of neuroscience since the 1980’s into novel treatments for brain disorders has proven much more challenging than anticipated. The definitive methods required to be sure that one is validating or rejecting hypotheses on the potential of novel molecular interventions are sparse and unlikely to be developed within any single entity with the notable exception of PET ligands for orthosteric antagonists. The field is therefore exploring multiple public/private partnerships to develop and share the tools for ruling in or out the utility of novel intervention. A still-to-be-implemented possibility would be a true pre-competitive effort to de-risk a particular mechanism through a shared validation effort followed by some means of distributing the commercial rewards of finding the best molecule to affect the validated target.

11:15 From Gut-Brain Explorations to Effective Innovative Therapies in Parkinson Disease

Patrice Garnier, PhD, CEO, Amabiotics

Due to millions of years of coevolution, complex metabolic interactions exist between human and its gut microbiota. Not surprisingly, the number of reports associating dysbiosis and systemic diseases increased drastically over the past years. Amabiotics is a biopharmaceutical company that develops innovative diagnostics and microbiome-derived medicines to cure neurodegenerative diseases. Its lead compound, AMA-101 is targeted against Parkinson’s disease.

11:45 Where the Tire Meets the Road: From Allostery to Translational Chemistry

Dario Doller, PhD, Senior Director, Exploratory Research, Sage Therapeutics

Characterization of functional effects subsequent to drug binding to its biological target is key to inventing new medicines, enabling precise translation to human trials. Allosteric drugs and probe compounds for ion channels and GPCR targets tailor such functional effects with higher sophistication than previously thought, enabling new paths into personalized medicine. Examples of functional molecular fine-tuning and consequences relevant to drug design are discussed.

12:15 Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own

12:45 Session Break

중추신경계 질환 치료제 개발의 새로운 전략

13:40 Chairperson’s Remarks

Stuart W. Hughes, PhD, Director and Head of Pharmacology, Biological Sciences, Vertex Pharmaceuticals

13:45 Dissecting Pathologic Molecular Signatures in Neurodegenerative Diseases through Chemical Chaperomics – From Mechanisms to Diagnostics and Treatment

Gabriela Chiosis, PhD, Professor, Member, Attending, Chemical Biology and Medicine, Memorial Sloan Kettering Cancer Center

I aim to discuss chemical chaperomics, a functional proteomics platform applicable for systematic proteomewide investigations of molecular alterations in Alzheimer’s disease and other neurodegenerative diseases. The method provides large-scale unbiased information on global protein-protein interactions and pathologic protein function changes in response to genetic and environmental factors. Implementation of this method to the analysis of patient-derived specimens may provide important information that is unavailable through, but complementary, to other ‘omics’ methods.

14:15 Antibodies for Brain Disorders Using the Brain Shuttle Blood-Brain Barrier Transport Technology

Per-Ola Freskgård, PhD, Vice Director & Expert Scientist, Neuroscience, Roche Pharma Research & Early Development

This talk will briefly describe the status of the brain delivery field of biologics to the brain by highlighting recent advancements. In particular, the Brain Shuttle technology will be described, which is designed to be engineered into a standard therapeutic antibody and other types of biologics for successful BBB transport. Efficiency and safety aspects will be addressed illustrated with recent experimental data.

14:45 Progress in Neuroengineering for Brain Repair: From in vitro to in vivo Studies and Beyond

Michela Chiappalone, PhD, Researcher (Team Leader), Rehab Technologies, Italian Institute of Technology (IIT)

In recent years, biomedical devices have been developed to target different neurological disorders. To reach useful therapeutic results, these tools need a multidisciplinary approach and a continuous dialogue between neuroscience and engineering, a field named Neuroengineering. In this talk, I will highlight the importance of developing novel neurotechnologies for brain repair (exploiting both in vitro and in vivo animal models) and present the major challenges expected for the next years.

15:15 Developing Bioengineered Vascularized Human Brain Tissues for Drug Development and Mechanistic Innovation

Cheryl Wellington, PhD, Professor, University of British Columbia

15:45 Close of Conference

* 주최측 사정에 따라 사전 예고없이 프로그램이 변경될 수 있습니다.

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