TRACK RECORD
CELL THERAPY WITH GENETIC MANIPULATION
TK , MolMed’s proprietary cell therapy - based on genetically engineered donor T cells - that opens the door of bone marrow transplantation to leukaemic patients lacking a fully compatible donor, currently in Phase III clinical development:
• Production of retroviral vectors in bioreactor.
• Transduction of donor T cells and expansion of transduced cells in closed systems.
ADA-SCID, a “one shot “ gene therapy to cure severe combined immunodeficiency (SCID) caused by deficiency of adenosine deaminase (ADA), produced on behalf of the San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET, Italy)
• Production of retroviral vectors.
• Purification and transduction of CD34+ cells from patient bone marrow.
Wiskott-Aldrich Syndrome (WAS) and Metachromatic Leucodystrophy (MLD), “one shot“ gene therapies to cure these two rare genetic diseases, produced on behalf of the San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET, Italy)
• Production and purification of lentiviral vectors.
• Purification and transduction of CD34 + cells from patient bone marrow or circulating blood.
Hematopoietic stem cells for transplantation, produced on behalf of San Raffaele Foundation, Italy.
• Positive selection of donor CD34 + hematopoietic cell progenitors derived from peripheral blood after mobilisation from bone marrow.
Duchenne Muscular Dystrophy, produced on behalf of San Raffaele Foundation, Italy.
• Development of large-scale production of human mesangioblasts from muscle tissue.
• Development of analytical methods for cell characterization.
• Production in vitro of mesangioblasts for clinical use.
• Regulatory advise and support in facing the international authorities and drafting documentation for the application for clinical trials at the Italian Regulatory Authority (IMPD).
Melanoma, produced on behalf of San Raffaele Foundation, Italy
• Extraction of tumor-associated peptides, naturally processed from a specific melanoma cell line.
• Production in vitro dendritic cells isolated from melanoma patients and stimulated with tumour-associated peptides.
Refractory angina pectoris produced on behalf of San Raffaele Foundation, Italy
• positive selection of human CD34 + hematopoietic progenitor cells promoting re-vascularisation, to be infused in patients with refractory angina pectoris.
Immunotherapy with IL-10, produced on behalf of San Raffaele Foundation, Italy
• Treatment in vitro of allogeneic T cells with IL-10 for patients undergoing hematopoietic stem cells transplantation.
European Commission Seventh Framework Programme (FP7)
• ATTRACT – project number 238778.
• PERSIST – project number 222878.
• OPTISTEM – project number 223098.
European Commission Seventh Framework Programme (FP6)
• SKINTHERAPY - project number 512073.
• CONSERT - project number 005242.
