Click on any gene therapy product in the interactive pipeline below to learn more about hemophilia gene therapies in development
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Adeno-associated virus (AAV) vectors
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A vector is a vehicle to carry genetic material, such as the F8 or F9 gene, into a cell where it can be expressed. Viruses have been identified as efficient delivery vehicles. Some have evolved to infect humans and have been tested as gene delivery vehicles. Adeno-associated viruses (AAV) can be genetically engineered to be used as vectors for gene delivery. AAV vectors are the most commonly used vectors for gene therapy for hemophilia. AAV vectors are safe, efficient, and non-pathogenic. There are many different types of AAVs: think of them as cousins or siblings to one another depending on how similar they are to one another.
Gene transfer can be achieved by directly injecting the AAV vector containing the new gene into the body. This is most often done intravenously, where much of the AAV is directed to the liver. In the liver it enters cells, leading to Factor VIII or Factor IX production.
Gene transfer can be achieved by directly injecting the AAV vector containing the new gene into the body. This is most often done intravenously, where much of the AAV is directed to the liver. In the liver it enters cells, leading to Factor VIII or Factor IX production.
Lentiviral vectors
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A vector is a vehicle to carry genetic material, such as the F8 or F9 gene, into a cell where it can be expressed. Lentiviruses can be genetically engineered to be used as a vector for gene delivery. Their use in gene therapy for hemophilia is currently under investigation. To date (April 2020), no patients with hemophilia have started treatment with lentiviruses.
Gene transfer can be achieved by directly injecting the lentiviral vector containing the new gene into the body or removing stem cells from a patient. If stem cells are removed, the lentivirus is incubated with the cells in the laboratory and they are returned to the patient. This is a complicated process that involves some risk since some of the bone marrow needs to be destroyed to make room for the new cells.
Gene transfer can be achieved by directly injecting the lentiviral vector containing the new gene into the body or removing stem cells from a patient. If stem cells are removed, the lentivirus is incubated with the cells in the laboratory and they are returned to the patient. This is a complicated process that involves some risk since some of the bone marrow needs to be destroyed to make room for the new cells.
Cell Therapy
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Cell therapy is the transfer of cells containing genetic material of interest to a patient. A specific gene, such as the F8 or F9 gene, is first introduced into cells. Gene transfer can then be achieved by injecting the cells containing the new gene into the body. This has not yet been done in persons with hemophilia, but laboratory studies are making progress (April 2020).
Long Term Follow-Up Studies
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Long-term follow up (LTFU) studies are used to monitor participant’s health outcomes for a duration of time after they complete a clinical trial. These LTFU studies are important to gather information about longer term effects of treatments, including safety and efficacy.
Real World Evidence / Observational Studies
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Real-world data (RWD) are data that come from sources other than traditional randomized, controlled trials (RCTs). RWD can come from many sources, including registries and observational studies. Registries and observational studies are a fundamental part of epidemiological research where the investigators observe individuals without manipulation or intervention. RWD can be used to provide real-world evidence.