Autologous lentiviral WAS stem cell therapy

Unassigned

New Medicines

Wiskott Aldrich syndrome

Information

Advanced therapy medicinal product (ATMP)
Orchard Therapeutics
Orchard Therapeutics

Development and Regulatory status

None
Phase II Clinical Trials
None
Yes
Yes

Category

An ex vivo stem cell therapy that uses lentiviral vector to transfer functional WAS genes into autologous haematopoietic stem cells
The underlying mutation is in the gene for the Wiskott-Aldrich syndrome protein (WASP) on the X-chromosome at Xp11.22-23. WASP is needed for normal antibody function, T-cell responses and platelet production. The incidence of the classic syndrome is estimated to be between one and ten in one million individuals, although it is likely to be higher [1].
Wiskott Aldrich syndrome
Intravenous infusion

Wiskott Aldrich syndrome

Information

Advanced therapy medicinal product (ATMP)
Genethon
Genethon

Development and Regulatory status

Phase II Clinical Trials
Phase II Clinical Trials
Phase II Clinical Trials
Yes

Category

WAS gene therapy based on ex vivo gene transfer, where a lentiviral vector carring the therapeutic gene is inserted into the haematopoietic stem cellsof the patient (autologous CD34+ cells)
The underlying mutation is in the gene for the Wiskott-Aldrich syndrome protein (WASP) on the X-chromosome at Xp11.22-23. WASP is needed for normal antibody function, T-cell responses and platelet production. The incidence of the classic syndrome is estimated to be between one and ten in one million individuals, although it is likely to be higher [1].
Wiskott Aldrich syndrome
Intravenous infusion