Next generation gene engineering of HSPC: Lessons from first-in-human stem cell gene editing trial

ISSCR – The Global Stem Cell Event

San Francisco, CA
June 15 – June 18, 2022

The 2022 ISSCR Annual Meeting gathers the world’s leading scientists, clinicians, business leaders, ethicists, and educators from more than 65 countries. Together, we celebrate 20 years of excellence, advances, and contributions of stem cell research and regenerative medicine. Discovery, careers, advocacy, inspiration, new business, and relationships start here.

Featured Event

Title:

Next generation gene engineering of HSPC: Lessons from first-in-human stem cell gene editing trial
Date: Friday, June 17th
Time: 8:00 – 8:30 AM
Location: Room 2003

Abstract

Cell engineering via genome editing has revolutionized medical research and opened new avenues for therapeutic intervention, from correction of genetic diseases to target-specific immunotherapies. We conducted the first clinical trial in which autologous stem cells were gene edited and infused into patients (NCT-02500849). In this session, we will present data of the manufacturing of gene-edited hematopoietic stem progenitor cells (HSPC) using first-generation zinc-finger nuclease technology, and relevant clinical data, as the first subjects reach 6 years post-infusion. The challenges and lessons from this trial have guided us to the development of custom, closed-system workflows for the clinical manufacture of genetically-engineered HSPC, and of more efficient gene-editing platforms. We will discuss some data using next-generation gene editing constructs and the challenges and opportunities of translating these technologies into the clinic.

Speaker:

Dr. Angelo A. Cardoso, MD, PhD
Director of the Laboratory for Cellular Medicine (LCM), Beckman Research Institute at City of Hope.
Dr. Angelo A. Cardoso, MD, PhD is the Director of the Laboratory for Cellular Medicine (LCM) at the Beckman Research Institute a City of Hope (COH), and the Research Director of the COH’s Alpha Stem Cell Clinic. The LCM is a translational laboratory with a pipeline of cell and gene therapy projects, at different stages of development. Our activities range from basic research and technology innovation, to the scaling and qualification of IND-enabling GMP processes, to the implementation of phase I clinical trials.

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