Cambridge Healthtech Institute’s Inaugural

CNS, Gene and Cell Therapy

Progress in CNS and Rare Diseases, Gene and Cell Therapy

September 16-17, 2020


Significant progress in the fields of genetics, protein science, and drug discovery is driving a renewed interest in emerging indications outside of oncology, and important indications, such as CNS and rare diseases. Moreover, new and exciting technologies, such as cell, gene, and gene-edited therapies are offering a new way of treating previously untreatable diseases, and are quickly advancing through the clinic.

CHI's CNS, Gene and Cell Therapy conference provides a platform for pharma, biotech, and academia to discuss and benchmark the latest advances in developing new drug targets and novel therapies across the fields of neuroscience and  rare diseases, as well as the unique challenges facing cell and gene therapy development. How do your drug discovery and translational strategies compare?

Preliminary Agenda

MATCHING THE RIGHT MODALITY TO THE RIGHT MECHANISM

Matching the Right Modality to the Right Mechanism for the Right Patient

Julie Chen, PhD, Chemical and Structural Biology Lead, Eisai Center for Genetics Guided Dementia Discovery (G2D2)

Small Molecule Treatment of Brain Diseases: A Perspective on the Path Forward, Looking Up from the Bottom

Dario Doller, PhD, Director, Medicinal Chemistry, Sunovion Pharmaceuticals

Leveraging the Power of Human Genetics to Go beyond A-Beta and Tau

Tiernan O'Mally, PhD, Associate Director, Translational Human Biology, Eisai Center for Genetics Guided Dementia Discovery (G2D2)


CNS DRUG DEVELOPMENT AND TRANSLATION

Machine Learning Models in CNS Drug Discovery and Penetrating the BBB

Istvan Enyedy, PhD, Director, Black Diamond Therapeutics

PET Imaging of Neuroinflammation in Neurodegenerative Diseases

Changning Wang, PhD, Assistant Professor of Radiology, Athinoula A. Martinos Center for Biomedical Imaging, Massachusetts General Hospital, Harvard Medical School

A Neuroinflammatory Translational Pipeline for Neurodegenerative Diseases

Jonathan Levenson, PhD, Vice President, Translational Biology, Tiaki Therapeutics

GENE THERAPIES FOR NEUROLOGICAL DISEASEs

Gene Therapy Development for Neurological Diseases

Gabriele Proetzel, PhD, Director, External Neuroscience Innovation, Neuroscience Drug Discovery Unit, Takeda Pharmaceuticals

Preclinical Development of an Intrathecally-Administered ASO to Treat Spinal Muscular Atrophy

Kenneth S. Loveday, PhD, DABT, Principal Investigator, Preclinical Safety, Biogen

Tissue Distribution and PK of Antisense Oligonucleotides following Intrathecal Administration

Natasha Penner, PhD, Director, Clinical Pharmacology and Pharmacometrics, Biogen

EMERGING TARGETS AND PATHWAYS

Lysosomal Dysfunction in Common Neurodegenerative Diseases: From Genetics to the Clinic

Pablo Sardi, PhD, R&D Senior Director, Rare and Neurological Diseases, Sanofi

Targeting Ataxin-2 against TDP43 Pathology Associated with Amyotrophic Lateral Sclerosis and Frontotemporal Dementia

Yinghui Hu, PhD, Associate Principal Scientist, Neurodegeneration Group, Merck

 

CELL AND GENE THERAPY

Authentic Cell Therapies for Intractable Neurological Diseases

Stefan Irion, MD, Vice President, Translational Neuroscience, BlueRock Therapeutics

 Disruption of RNA Metabolism in Neurological Diseases and Emerging Therapeutic Interventions

Matthew Nolan, PhD, Post Doc Research Fellow, Harvard Medical School and Massachusetts General Hospital

 


For more details on the conference, please contact:
Bridget Kotelly
Conference Producer
Cambridge Healthtech Institute
Phone: (+1) 781-972-5404
Email: bkotelly@healthtech.com

For partnering and sponsorship information, please contact:
Rod Eymael
Manager, Business Development
Cambridge Healthtech Institute
Phone: (+1) 781-247-6286
Email: reymael@healthtech.com