Sabah Sallah

WORK HISTORY

EDUCATION

ABOUT SABAH SALLAH

Critical evaluation of liver-directed gene transfer lead products. Appropriate design of the construct including the selection of the vector, promoter/enhancer, transgene and poly A signal with emphasis on enhanced transduction potency through capsid design and protein engineering. Evaluation and recommendation on the most appropriate small and large animal models for gene transfer. Ongoing assessment of the cellular immune response to the capsid and transgene to include the appropriate diagnostic and monitoring laboratory assays. Prominent attention to the interface between basic science and clinical development and the ability to rigorously question research assumptions and to identifying inconsistencies and analyzing and proposing solutions in a structured and objective way. Leading role in weighing risk versus clinical benefit for lead molecules and applying medical and scientific rigor to the design of proof of concept and pivotal trials in addition to the generation and execution of clinical development plan in line with corporate strategy and in alignment with regulatory requirements. Substantial leadership experience across various functions including clinical, research, clinical assays and regulatory with attention to fostering a culture of medical, scientific diligence and ethical behavior. Oversee strategic portfolio management for multiple products across all stages of development in both adult and pediatric indications. Within the past 12 years, this translated in leading the efforts to the submission of a significant number of pre-IND/INDs, BLAs, the global approval of five recombinant protein products and the approval of Hemgenix by FDA and EMA as the first gene therapy that gained approval in the hemophilias. Clinical and scientific areas of expertise include rare disorders, hematology, lysosomal storage disease, liver metabolic disorders, oncology, bleeding and thrombotic diseases, basic and applied immunology and molecular biology.