ISCT - Intl Society for Cell & Gene Therapy

Celebrating the Progress of Advanced Therapies and Building the Future Through Translations

May 29 - June 1

Vancouver, Canada

Booth #422

With a core focus on cell and gene therapy translation, the ISCT Annual Meeting takes an integrative approach across ALL stakeholders to address key topics spanning translational research and preclinical development through to clinical trials, regulatory approvals, commercialization and ultimately patient access. Topics are curated to address the biggest bottlenecks in cell and gene therapy development.

Come visit us at booth #422 and discover MaxCyte's cGMP-compliant non-viral cell engineering platform and how it enables the acceleration of therapeutic development from concept to clinic.

While at the meeting, attend our feature presentation:

Featured Event

Gene Editing in Hematopoietic Stem Cells

Date: May 31, 2024
Time: 12:45 - 1:45 PM
Location: TBD
Presenter: Donald Kohn, MD & Zulema Garcia Romero, Ph.D., University of California, Los Angeles

Abstract

Hematopoietic stem cells (HSC) are responsible for life-long production of all blood cells and can be collected, manipulated ex vivo for gene addition or gene editing, and re-engrafted by intravenous infusion. Gene therapy using lentiviral vectors has successfully achieved clinical improvements for more than a dozen hematological disorders, including primary immune deficiencies (PID), hemoglobinopathies, lysosomal storage and leukodystrophy disorders. While the safety profile of lentiviral vectors in HSC has been good, lentiviral vectors lack precision in integration and may not recapitulate precise expression patterns needed for physiologically regulated genes.  Gene editing of endogenous loci has the potential for precise insertion and physiologically regulated expression. A collaboration between investigators at UC Berkeley, UC San Francisco and UC Los Angeles has led to a clinical trial that will use Cas9-mediated homology-directed repair with a single-stranded oligonucleotide donor to revert the Sickle Cell Disease (SCD)-causing mutation in HBB. Pre-clinical IND-enabling activities involved translation of methods for gene editing in SCD patient HSC to Good Manufacturing Practice-compatible processes with subsequent cryopreservation formulation and release testing. Unlike SCD where all patients have the identical single nucleotide mutation, most human genetic diseases have heterogeneous mutations across the affected gene in different patients.

Presenters

Kohn Don DBK 040116 headshot

Donald B. Kohn M.D.

Distinguished Professor, Departments of Microbiology, Immunology & Molecular Genetics (MIMG) and Pediatrics, University of California, Los Angeles

Garcia Zulema Romero-09r5x

Zulema Garcia Romero, Ph.D.

Assistant Adjunct Professor, Department of Microbiology, Immunology and Molecular Genetics, University of California, Los Angeles