Date: 2013-11-22
Type of information: Production agreement
Compound: gene therapy
Company: MolMed (Italy) GSK (UK)
Therapeutic area: Rare diseases - Genetic diseases
Type agreement: manufacturing
production
Action mechanism:
Disease: ADA-SCID (Adenosine Deaminase Deficiency – Severe Combined Immune Deficiency)
Details: * On August 5, 2011, MolMed has signed an agreement with GSK under which MolMed will develop a production process for an investigational gene therapy for ADA-SCID (Adenosine Deaminase Deficiency – Severe Combined Immune Deficiency). This gene therapy, which is in late stage clinical trials, has been developed by the San Raffaele Telethon Institute for Gene Therapy (HSRTIGET) and was in-licensed by GSK to develop and commercialise the therapy.
Financial terms: MolMed will receive up to € 5.5 million in revenues over a two-year period.
Latest news: * On November 22, 2013, MolMed has entered into an agreement with GSK under which MolMed will be responsible for the manufacture of GSK’s investigational gene therapy for compassionate use in patients with Adenosine Deaminase Deficiency – Severe Combined Immune Deficiency (ADA-SCID) in the potential event that an investigator led named patient compassionate use application. Under the original agreement announced in August 2011, MolMed developed the production process for the investigational gene therapy for ADA-SCID. MolMed previously produced on behalf of Telethon the investigational gene therapy where the correct form of the ADA gene is inserted into the patients’ own bone marrow derived stem cells. GSK is now continuing the clinical development of the ADA-SCID gene therapy in collaboration with the San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET) from which GSK has in-licensed the rights to develop and commercialize the therapy.
Clinical trials testing the gene therapy are now complete and for patients lacking suitable therapy referred to San Raffaele Hospital, following ethical approval, GSK will support compassionate use named patient through product manufacture at MolMed.
