Akshay Sharma, MBBS, MSc

The long-term goal of my research is to develop cellular and genetic therapies with curative potential for children with inherited disorders of the hematopoietic system, focusing on sickle cell disease (SCD) and bone marrow failure syndromes. The bulk of my work concentrates on the use of hematopoietic cell transplantation, graft engineering and genomic editing with an emphasis on learning how best to implement these approaches to reduce therapy-associated toxicities.

To learn how best to reduce toxicities, I leverage my postdoctoral fellowship work, in which I studied the mechanisms of antigen presentation in allogeneic hematopoietic cell transplantation (HCT) to understand and reduce adverse effects of HCT. In my clinical fellowship, I conducted laboratory studies using CRISPR-Cas9-based genome editing to identify novel regulatory regions in genes associated with hemoglobin switching. This basic science research work led me to develop the skills necessary to develop novel translational therapeutic approaches using cutting-edge genetic technologies and cellular therapies.

In my time as a faculty member at St. Jude, I have served as the site-Principal Investigator (PI) for several academic consortia and industry-led clinical trials assessing hematopoietic cell transplantation and gene therapy for SCD. Leading my independent clinical research work has been an area of pride where I’ve initiated a pilot clinical trial testing a novel gene editing approach in individuals with SCD, known as  SAGES1: The study will genetically modify the patient’s own stem cells using a CRISPR/Cas9 gene editing approach to increase fetal hemoglobin in the red blood cells as a way to treat SCD.

The insights I learn from conducting clinical research into these therapies will advance our knowledge about how best minimize long-term adverse effects of these therapies, thereby improving the quality of life of these patients.