Punam Malik, MD

At-Large Director (2023-2026) | Science + Education Council Chair

Punam Malik, MD, is the Marjory J. Johnson Chair of Gene and Cell Therapy and professor of Pediatrics at the Cincinnati Children’s Hospital Medical Center (CCHMC) and the University of Cincinnati.

Dr. Malik is a physician-scientist. Her research interests lie in studying the genetic therapies for hematopoietic stem cell disorders with a focus on hemoglobinopathies. She also works on pathophysiology of sickle cell anemia (SCA) and developing targeted therapies, such as gene therapy. I have been studying gene transfer into murine and human hematopoietic stem cells (HSCs) using different viral vector platforms (γ-retroviral, foamy virus, lentivirus and adeno-associated virus vectors) and optimizing HSC gene transfer and gene editing. This has included studies on mechanisms by which genetic modification of the HSC genome affects HSC fate and its long term engraftment potential, ways to improve titers and safety of lentivirus vectors, including structure function studies and identification of novel chromatin insulator elements to reduce genotoxicity and improve engraftment specifically of genetically modified human HSC.

Dr. Malik's lab developed a potent antisickling globin by modifying the human γ-globin gene and when delivered via a lentivirus vector, it ameliorates sickling of RBCs at a much lower antisickling globin concentration, successfully ameliorating SCA with reduced intensity conditioning. She has enabled GMP translation of genetic therapies for inherited hematological disorders including immune deficiency disorders such as X-SCID, leukocyte adhesion deficiency, hemophagocytic lympho-histiocytosis, beta-thalassemia and SCA. More recently, Dr. Malik has embarked on gene editing approaches for hematopoietic disorders, and developed custom Cas9 nucleases that reduces error prone repair and promote targeted correction specifically at the Cas9 cut sites and are optimizing this approach for correcting larger genomic edits, especially in large genes with multiple mutational hot-spots, which cannot be corrected by base/prime editing.