Cambridge Healthtech Institute’s Inaugural

Emerging Indications and Modalities

Progress in CNS and Rare Diseases, Gene and Cell Therapy, Fibrosis and Liver Diseases

June 2-4, 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, rare diseases, fibrosis and liver disease. 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 Emerging Indications and Modalities 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, rare diseases, fibrosis and liver diseases, as well as the unique challenges facing cell and gene therapy development. How do your drug discovery and translational strategies compare?


Matching the Right Modality to the Right Mechanism for the Right Patient
Nadeem Sarwar, Founder & President, Eisai Center for Genetics Guided Dementia Discovery (G2D2), Eisai Inc.

Preclinical Development of an Intrathecally Administered ASO to Treat Spinal Muscular Atrophy
Kenneth S. Loveday, PhD, DABT, Principal Investigator, Preclinical Safety, Biogen

Preclinical Translational Strategies for the Neuroinflammatory Aspects of Neurodegenerative Disease
Jonathan Levenson, PhD, Vice President, Translational Biology, Tiaki Therapeutics

Disruption of RNA Metabolism in Neurological Diseases and Emerging Therapeutic Interventions
Clotilde Lagier-Tourenne, MD, PhD, Associate Professor of Neurology, Healey Family ALS Endowed Chair for Research, Massachusetts General Hospital and Harvard Medical School

Machine Learning Models in CNS Drug Discovery
Istvan Enyedy, PhD, Principal Scientist, Biogen

Leveraging the Power of Human Genetics to Go beyond A-Beta And Tau
Representative, Eisai Inc.


Authentic Cell Therapies for Intractable Neurological Diseases
Stefan Irion, MD., VP, Translational Neuroscience, BlueRock Therapeutics

NIH Somatic Cell Genome, Editing Consortium: Beyond One Disease at a Time, Platform Approaches to Rare Disease Therapies
Philip John (P.J.) Brooks, PhD, Program Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), National Institutes of Health (NIH)

Tissue Distribution and PK of Anti-Sense Oligonucleotides Following Intrathecal Administration
Natasha Penner PhD, Director, Clinical Pharmacology and Pharmacometrics, Biogen


Role of Fibroblast Expression of Integrin-alpha V in Fibrosis and Inflammation
Kevin Hart, PhD, Principal Scientist, Inflammation, Pfizer

Targeting Integrins for Fibrosis
Katerina Leftheris, PhD, Vice President, Chemistry, Pliant

A ‘Fibrosis Biomarker Tool Kit’ for the Lung and Beyond
Vanessa Morales-Tirado, PhD, Senior Scientist III, Translational Immunology, Immunology Discovery, AbbVie Bioresearch Center

Progress toward a Combination Therapy to Cure HBV
Mike Sofia, PhD, Chief Scientific Officer, Arbutus Biopharma