5 European Projects Found
Searched on 125080 European Projects
FINISHED
Clinical development of a dendritic-cell vaccine therapy for acute myeloid leukaemia (AML-VACCiN)
Start date: Jan 1, 2016, End date: Mar 31, 2020,
Eradicating minimal residual disease (MRD) in patients with acute myeloid leukaemia (AML) is an area of high unmet medical need. AML is a deadly rare disease that affects both children and adults. Approximately 45% of younger AML patients who are treated will be cured, and in older patients (>60 years of age), 85% will relapse and die within 2 years.Immunotherapy has great potential for treating M ...
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12
Partners Participants
FINISHED
Gene Therapy for X-linked Chronic Granulomatous Disease (CGD) (NET4CGD)
Start date: Dec 1, 2012, End date: May 31, 2018,
This project is focused on the clinical development of a new orphan drug that can rapidly become a new treatment option for patients with the X-linked form of chronic granulomatous disease (X-CGD). This rare primary immune deficiency of phagocytes is caused by mutations in the gp91phox gene. Affected patients are highly-susceptible to infections and develop inflammatory granulomas. Several member ...
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11
Partners Participants
FINISHED
Phase I/II ex vivo gene therapy clinical trial for recessive dystrophic epidermolysis bullosa using skin equivalent grafts genetically corrected with a COL7A1-encoding SIN retroviral vector (GENEGRAFT)
Start date: Mar 1, 2011, End date: Dec 31, 2017,
RDEB is one of the most severe rare genetic skin diseases of children and adults characterized by skin blistering resulting from lack of expression of type VII collagen. There is no treatment for this life-threatening disease. In March 2009, we obtained the orphan drug designation for the Medicinal product: “Skin equivalent graft genetically corrected with a COL7A1-encoding SIN retroviral vecto ...
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10
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FINISHED
Advanced Cell-based Therapies for the treatment of Primary ImmunoDeficiency (CELL-PID)
Start date: Nov 1, 2010, End date: Apr 30, 2016,
Primary immune deficiencies (PID) are inherited disorders of the adaptive and innate immune system marked by severe infections, autoimmunity and high risk of cancer. Treatment entails hematopoietic stem cell (HSC) transplantation from allogeneic donors, however in the absence of an HLA compatible donor, HSCT outcome is limited by delayed or suboptimal reconstitution and complications. SCID-X1 and ...
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20
Partners Participants
FINISHED
Persisting Transgenesis (PERSIST)
Start date: Jan 1, 2009, End date: Jun 30, 2013,
"For many disabling or fatal diseases, there is pre-clinical or clinical evidence of the potential therapeutic efficacy of gene therapy and, yet, the limitations of current gene transfer technologies have prevented success or even caused serious adverse events leading to termination of trials. PERSIST will explore the use of highly innovative gene-modifying and delivery technologies and capitalize ...
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