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Related to monogenic disease: Genetic diseases, autosomal dominant disorders, single gene disorder
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These forward-looking statements include, without limitation, the potential of ZFNs to treat a broad range of human monogenic diseases, including beta-thalassemia, hemophilia and LSDs, research and development of novel ZFP TFs and ZFNs, therapeutic applications of Sangamo's ZFP technology platform, including IVPRP, in indications such as hemophilia and LSDs, the anticipated timing and the number of IND filings and initiation and completion of clinical trials.
Pre-marital counselling and screening is one method of decreasing the likelihood of children being born with monogenic diseases.
Most diagnostic exomes on the market today have not been optimized, and have sensitivities of between 20% and 50% for monogenic disease.
Sangamo is developing the IVPRP as a generally applicable strategy to provide a one-time, permanent genetic cure for monogenic diseases that are currently managed by protein replacement therapy, which involves costly repeat infusions over the lifetime of the patient.
In addition, the mRNA delivery method could be used in the development of second generation ZFP Therapeutics to broaden the patient group that can be treated using Sangamo's In Vivo Protein Replacement Platform (IVPRP) which is being developed as a generally applicable strategy to provide a permanent genetic cure for certain monogenic diseases.
Sangamo's novel zinc finger protein (ZFP) technology is being developed for various therapeutic applications such as HIV/AIDS and several monogenic diseases including sickle cell disease and beta-thalassemia, hemophilia, lysosomal storage disorders and Huntington's disease.