Donald B. Kohn, M.D.

Kohn is credited with the development of one of the first successful blood stem cell gene therapies targeting adenosine deaminase–deficient severe combined immunodeficiency, or ADA-SCID. Kohn’s therapy for the condition, in which babies are born without an immune system and often do not survive past the first two years of life, has cured more than 50 babies to date. He was also the first researcher to perform a gene transfer into CD34+ cells from umbilical cord blood in a clinical trial for ADA-SCID and the first in the United States to initiate clinical trials of a gene therapy for pediatric HIV/AIDS using bone marrow stem cells. 

His blood stem cell gene therapy method involves collecting some of a patient’s own blood stem cells and either adding a healthy copy of or fixing a gene that has a disease-causing mutation. Patients then receive a transplant of their own corrected stem cells, which will ideally produce a lifelong supply of healthy blood and immune cells. This method eliminates the risk of rejection associated with receiving a bone marrow transplant from a different person.

Kohn is applying similar blood stem cell gene therapy techniques in four additional clinical trials that are showing promising early results. This includes treatments for the rare immune disorders X-linked SCID,  leukocyte adhesion deficiency-I, X-linked chronic granulomatous disease and the genetic blood disorder sickle cell disease. He is continuing to adapt this therapeutic platform to treat a wider range of diseases, developing methods to make gene therapies more efficient and affordable and working with novel gene editing approaches such as CRISPR/Cas9 and base editing. 

A physician-scientist for over 30 years, Kohn has also been recognized for his mentorship and his outstanding contributions to the fields of cell and gene therapy. He is committed to training new investigators in the treatment of blood diseases in an inclusive, safe and supportive research environment.