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Q2 2019 Report - Gene Therapy for Rare Disease


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.

Gene Therapy for Rare Disorders: Outlook for the Sector in the Age of Zolgensma and Zynteglo

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

PH. 1

212

PH. 2

320

PH. 3

30

Clinical Trials by Technology Type

Clinical Trials by Indication

Gene Delivery: 196
Oncolytic Virus: 22
Gene Editing: 21
Other Gene Therapy: 2

 

CAR-T: 175
TCR: 53
APC or Dendritic Cell: 26
NK Cell: 25
TILs/MILs: 11
Other GMCT: 31

Companies Active in Developing Gene Therapies and Gene-Modified Cell Therapies for Rare Diseases

 

$3.1 Billion

RAISED IN H1 2019 FOR GENE THERAPIES
& GENE-MODIFIED CELL THERAPIES
FOR RARE DISEASE