First FDA-Approved Mesenchymal Stromal Cell Therapy

The Food and Drug Administration (FDA) approved the first mesenchymal stromal cell (MSC) therapy, RYONCIL®, indicated for steroid-refractory acute graft-versus-host disease (SR-aGVHD) in children two months and older. This milestone in regenerative medicine was developed and patented by Osiris Therapeutics (Osris), a venture founded by Kevin Kimberlin and Dr. Arnold Caplan, and first backed by Spencer Trask & Co.

Kimberlin, who previously collaborated with Dr. Jonas Salk to develop the first FDA-approved cell-based immunotherapy, partnered with Dr. Caplan to conduct the first human clinical trials with mesenchymal stromal cells. This work was instrumental in establishing the safety and feasibility of MSC therapies, directly contributing to the development and eventual FDA approval of treatments like RYONCIL. To complete the necessary Phase 3 trials, Osiris licensed the intellectual property in exchange for milestone payments and royalties, and sold the product, originally called Prochymal, to Mesoblast Limited (Mesoblast), creating a powerhouse at the forefront of regenerative medicine.