Treatment Database
Autologous genetically modified CD34+ hematopoietic cells transduced with a lentiviral vector (LV-EFS-TCIRG1-WPRE) that encodes for the human TCIRG1 transgene
Trade Name not avaiable
Orphan Indication Infantile malignant osteopetrosis
USA Market Approval
USA Designation Date 28-02-2019
Sponsor Rocket Pharmaceuticals, Inc.
The Empire State Building, 350 Fifth Avenue, Suite 7530
New York, New York, 10118
More about Albers-Schonberg disease
Free Newsletter