This report provides industry specific statistics compiled from cell therapy, gene therapy, tissue engineering, and other regenerative medicine companies active in developing therapies for rare disease and provides context for the potential of regenerative medicine to improve upon the current standard of care.
In Q2 2019, the gene therapy sector experienced two major approvals: AveXis / Novartis’s Zolgensma (onasemnogene abeparvovec-xioi), an in vivo AAV gene therapy for the treatment of spinal muscular atrophy (SMA) type 1 in infants under the age of two, and bluebird bio’s Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene), an ex vivo lentiviral gene therapy for the treatment of transfusion-dependent beta thalassemia (TDT).
As the number of approved gene therapies grows, it imperative that stakeholders continue to convene to identify strategies to ensure patients can access these lifechanging therapies.
562+
Gene therapy and gene-modified therapy clinical trials underway
worldwide in rare disease as of the end of H1 2019
212
320
30
