Adrenoleukodystrophy – Pipeline Review, H2 2011
Adrenoleukodystrophy – Pipeline Review, H2 2011
Reference Code: GMDHC1617IDB
Publication Date: December 2011
Adrenoleukodystrophy – Pipeline Review, H2 2011 GMDHC1617IDB / Pub December 2011
© Global Markets Direct. This report is a licensed product and is not to be photocopied Page(1)
Adrenoleukodystrophy – Pipeline Review, H2 2011
Table of Contents
Table of Contents ............................................................................................................................................................................... 2
List of Tables ................................................................................................................................................................................. 3
List of Figures ................................................................................................................................................................................ 4
Introduction......................................................................................................................................................................................... 5
Global Markets Direct Report Coverage ........................................................................................................................................ 5
Adrenoleukodystrophy Overview ........................................................................................................................................................ 6
Therapeutics Development................................................................................................................................................................. 7
An Overview of Pipeline Products for Adrenoleukodystrophy ........................................................................................................ 7
Adrenoleukodystrophy Therapeutics under Development by Companies .......................................................................................... 9
Adrenoleukodystrophy Therapeutics under Investigation by Universities/Institutes ......................................................................... 10
Late Stage Products ......................................................................................................................................................................... 11
Comparative Analysis .................................................................................................................................................................. 11
Mid Clinical Stage Products.............................................................................................................................................................. 12
Comparative Analysis .................................................................................................................................................................. 12
Adrenoleukodystrophy Therapeutics – Products under Development by Companies ...................................................................... 13
Adrenoleukodystrophy Therapeutics – Products under Investigation by Universities/Institutes ....................................................... 14
Companies Involved in Adrenoleukodystrophy Therapeutics Development ..................................................................................... 15
GENETIX Pharmaceuticals, Inc................................................................................................................................................... 15
Adrenoleukodystrophy – Therapeutics Assessment......................................................................................................................... 16
Assessment by Monotherapy Products ....................................................................................................................................... 16
Assessment by Combination Products ........................................................................................................................................ 17
Assessment by Route of Administration ...................................................................................................................................... 18
Assessment by Molecule Type .................................................................................................................................................... 20
Drug Profiles..................................................................................................................................................................................... 22
Lenti-D - Drug Profile ................................................................................................................................................................... 22
Product Description................................................................................................................................................................. 22
Mechanism of Action ............................................................................................................................................................... 22
R&D Progress ......................................................................................................................................................................... 22
Campath + Clofarabine + Melphalan + Hematopoietic Stem Cell Transplantation + Radiation Therapy - Drug Profile ............... 24
Product Description................................................................................................................................................................. 24
Mechanism of Action ............................................................................................................................................................... 24
R&D Progress ......................................................................................................................................................................... 25
Campath-1H + Cyclophosphamide + Busulfan + Allogeneic Stem Cell Transplantation - Drug Profile ....................................... 26
Product Description................................................................................................................................................................. 26
Mechanism of Action ............................................................................................................................................................... 26
R&D Progress ......................................................................................................................................................................... 26
Busulfan + Cyclophosphamide + Antithymocyte Globulin + Hematopoietic Stem Cell Transplantation - Drug Profile ................ 28
Product Description................................................................................................................................................................. 28
Mechanism of Action ............................................................................................................................................................... 28
R&D Progress ......................................................................................................................................................................... 28
Campath + Busulfan + Cyclophosphamide + Stem Cell Transplantation - Drug Profile .............................................................. 30
Product Description................................................................................................................................................................. 30
Adrenoleukodystrophy – Pipeline Review, H2 2011 GMDHC1617IDB / Pub December 2011
© Global Markets Direct. This report is a licensed product and is not to be photocopied Page(2)
Adrenoleukodystrophy – Pipeline Review,