A Pilot Feasibility Study of Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants Using a Self-Inactivating Lentiviral Vector to Transduce Autologous CD34+ Hematopoietic Cells
Phase of Trial: Phase I/II
Latest Information Update: 06 Nov 2019
Price : $35 *
At a glance
- Drugs MB 107 (Primary) ; Busulfan
- Indications Immunodeficiency disorders; X-linked genetic disorders
- Focus Adverse reactions; Registrational; Therapeutic Use
- Acronyms LVXSCID-ND
- 06 Nov 2019 According to a Mustang Bio media release, data will be presented at 61st American Society of Hematology Annual Meeting.
- 22 Aug 2019 According to a Mustang Bio media release, based on the data from this study, MB-107 has been granted the Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA).
- 18 Apr 2019 Results published in the New England Journal of Medicine