Oscar Segurado

Chief Medical Officer, ASC Therapeutics, USA

Title: Genomic medicine as a powerful discovery engine for gene and CRISPR therapies

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Biography

Oscar Segurado is the Chief Medical Officer for ASC Therapeutics, a fast-growing biotechnology company focused on developing curative gene-based therapies for inherited blood disorders, initially focusing on hemophilia A and B and Beta-Thalassemia. As a leading biopharma discovery company with over 12 years of experience in gene editing and stem cell technologies, we have created an end-to-end platform for gene therapy and gene editing.

Former CMO for Symvivo, Myriad Genetics and CellMax Life, Vice President for Becton Dickinson and Global Medical Head for Abbott/AbbVie (Humira).  Executive veteran with extensive global leadership experience in translational science, clinical development and global medical affairs.

Author and co-author of over 100 peer-reviewed publications, including Nature and Lancet, books and medical articles and member of several scientific and medical societies. Holding a tenured Professorship of Immunology at the University of Leon, Spain. Received PhD from the University of Wuerzburg, Germany and MD from the University of Salamanca, Spain.

Abstract

Over the past 20 years, DNA sequencing throughput has progressed from 1K base pairs per day to more than 1K bases per second today. Since the first proof-of-concept human application in the early ’90s, the field of gene therapy has entered an exciting stage of drug discovery, clinical translation and medical transformation.

In 2020 the first patient with sickle-cell disease was treated with ex-vivo CRISPR. This is the start of applying Nobel prize technology allowing to use enzymes able to cut and paste genes in the nucleus, the core of the generic machinery. We have discovered genes responsible for over 5K rare genetic diseases and over 100K genes associated with common diseases from diabetes to heart disease.

At this session we will discuss the principles, challenges, and future directions of discovery and development of human gene therapies, including CRISPR and base editing technologies. Genomic medicines possess the intrinsic benefit of being precise tools for manipulating human biology and are reserved for indications where there is a clear pathophysiology. While recent breakthrough therapeutics offer new hope for patients, they have also made biomedical innovation more complex, riskier, and more expensive. In the face of multiple growing uncertainties, the need for greater accuracy in predicting clinical trial outcomes has also grown. More accurate diagnostics mean fewer drug failures and faster approval times to bring new and better therapies to patients sooner.

As a case study we will discuss the experience of ASC Therapeutics for gene therapy and CRISPR therapy in hemophilia A, an optimal target due to the monogenic nature of inheritance and the observation that even minimal increases in clotting factor activity can significantly improve quality of life.