Cambridge Healthtech Institute’s Inaugural

Gene Therapy

Advancing from Bench to Clinic

May 6-7, 2020


2019 represents a big year for gene therapy, with 3 FDA approvals since August 2017. To date, there are over 2600 trials in 38 countries, with the majority of them in Phase I/II trials. With the heightened interest and the push to move quickly from bench to clinic, it is imperative for companies to have a better understanding of the implications of regulatory, commercialization, preclinical and clinical strategies on their gene therapies. At CHI's Gene Therapy: Advancing from Bench to Clinic, we invite scientists to share their latest updates on their gene therapy programs for rare diseases, inherited retinal diseases, neurological diseases, etc., and to discuss the various challenges they need to consider – from regulatory to analytical development, from preclinical translatability to clinical strategies.

Preliminary Agenda

REGULATORY STRATEGY & CONSIDERATIONS IN DEVELOPMENT OF GENE THERAPIES

KEYNOTE PRESENTATION:
Clinical Development of Gene Therapies

Mike Singer, MD, PhD, Medical Officer, Office of Tissues and Advanced Therapies, Division of Clinical Evaluation and Pharmacology/Toxicology, CBER, US FDA

An International Collaboration: Towards the Standardization of Gene Therapy

Yuan Zhao, PhD, Principal Scientist, Leader of Gene Therapy Section, Advanced Therapy Division/NIBSC, Medicines & Healthcare Products Regulatory Agency

OPTIMIZING DELIVERY OF GENE THERAPIES

Measure AAV Quality Attributes with SEC-UV-MALS-dRI

John Champage, Northeast Regional Manager, Senior Applications Scientist, Wyatt Technology

Targeted Tau Repression Using Engineered Zinc Finger Protein Transcription Fingers

Asa Hatami, PhD, Scientist II, Sangamo

DNA-Based Nanobody Delivery

Carlo Boutton, PhD, Head, Nanobody Explorative Technologies, Ablynx, a Sanofi company

GENE THERAPIES FOR RARE/INHERITED DISORDERS

Therapeutic Applications of CRISPR/Cas9 in Rare Diseases

Seokjoong Kim, PhD, Executive Director, R&D Strategy & Strategic Alliances, ToolGen

Updates on XLRP/Achromatopsia in Phase I/II Trials

Theresa Heah, MD, MBA, CMO, AGTC

Taking Gene Therapy beyond Rare Diseases

Alex Bloom, Vice President, Regulatory Affairs and Quality Assurance, Gyroscope Therapeutics

GENE THERAPY FOR NEUROLOGICAL DISEASES

Gene Therapy Development for Neurological Diseases

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

CRISPR-Cas9-Mediated Genome Editing in Huntington's Disease Mouse Model

Thomas Gaj, PhD, Associate Professor, Bioengineering, University of Illinois



* The program is subject to change without notice, due to unforeseen reason.

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Update History
2019/11/25
Sponsor updated



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