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SGMO Pipeline - all you need to know From their website: Ex Vivo Gene-Edited Cell Therapy Beta thalassemia is a rare blood disorder caused by a mutation in the beta-globin gene that results in greatly impaired production of healthy red blood cells. ST-400 is a gene-edited cell therapy that uses our ZFN genome editing technology to modify a patient’s own (autologous) hematopoietic stem cells (HSCs) to produce functional red blood cells using fetal hemoglobin. Sickle cell disease is a blood disease caused by a mutation in the beta-globin gene that causes the red blood cells to form an abnormal sickle or crescent shape. The cells are fragile and deliver less oxygen to the body’s tissues resulting in pain and irreversible organ damage. BIVV-003 is a gene-edited cell therapy that uses our ZFN genome editing technology to modify a patient’s own (autologous) hematopoietic stem cells (HSCs) to produce normal-shaped, functional red blood cells using fetal hemoglobin. TX-200 is a CAR-Treg cellular therapy in development for the prevention of chronic rejection after organ transplantation. TX-200 is composed of HLA-A2 CAR-Treg cells, regulatory T cells (Tregs) engineered with a Chimeric Antigen Receptor (CAR) designed to bind to the HLA-A2 protein expressed on the graft. This cell therapy approach is designed enable tolerance and long-term protection of the grafted organ. Sangamo has a worldwide collaboration and license agreement with Kite, a Gilead company, to develop next-generation ex vivo cell therapies for the treatment of cancer. Kite will use Sangamo’s ZFN genome editing technology to modify genes within T-cells and NK cells to develop CAR, TCR and NKR cell therapies for autologous (self) and allogeneic (universal) use. In Vivo Genome Editing MPS I (Scheie, Hurler-Scheie and Hurler syndromes) is caused by a deficiency of the enzyme alpha-L iduronidase (IDUA) and results in the toxic buildup of glycosaminoglycans (GAGs). SB-318 uses ZFN-based genome editing to insert a normal functioning copy of the IDUA gene under control of the strong albumin promoter in the patient’s liver. This approach is designed to enable the liver to produce and secrete active IDUA into the bloodstream to be taken up by other tissues. MPS II (Hunter syndrome) is caused by a deficiency of the enzyme iduronate-2-sulfatase (IDS) that results in the toxic buildup of glycosaminoglycans (GAGs). SB-913 uses ZFN-based genome editing to insert a normal functioning copy of the IDS gene under control of the strong albumin promoter in the patient’s liver. This approach is designed to enable the liver to produce and secrete active IDS into the bloodstream to be taken up by other tissues. Hemophilia B is a rare bleeding disorder, caused by a deficiency of clotting Factor IX protein, in which blood does not clot or stop bleeding when blood vessels are injured. SB-FIX uses ZFN genome editing to permanently insert a new therapeutic copy of the Factor 9 gene under control of the strong albumin promoter in the patient’s liver. This approach is designed to enable the liver to produce and secrete functionally active Factor IX protein into the bloodstream. In Vivo Gene Regulation Tauopathy disorders, including Alzheimer’s disease and other neurodegenerative diseases, are associated with intracellular tau protein aggregation and/or the formation of neurofibrillary tangles in neurons, leading to neuronal loss and atrophy. Sangamo is using our ZFP-TF gene regulation technology to develop potential gene therapies for tauopathy disorders designed to selectively repress the tau gene in neurons with the goal of reducing all forms of tau protein globally across the CNS and potentially ameliorating or reversing disease progression. Amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD) are part of a spectrum of neurodegenerative disorders linked to mutations in the C9ORF72 gene that involve hundreds of additional repetitions of a six base pair sequence of DNA. This ultimately leads to the deterioration of motor neurons, in the case of ALS, or neurons in the frontal and temporal lobes, in the case of FTLD. In collaboration with Pfizer, we are developing a gene therapy, using our ZFP-TF gene regulation technology, designed to specifically downregulate the mutant C9ORF72 allele to treat ALS and FTLD linked to mutations of the C9ORF72 gene. Huntington’s disease is an inherited, progressive neurologic disease caused by a mutation in the HTT gene characterized by greater than 39 additional repetitions of a three base pair sequence of DNA. Most patients inherit one normal and one mutant copy of the HTT gene, which ultimately leads to deterioration of muscle control, cognition and memory, onset in adulthood. In collaboration with Shire, we are developing a gene therapy using our ZFP-TF gene regulation technology, designed to selectively repress the expression of the mutant HTT allele while leaving expression levels of the normal gene unchanged. |
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Msg # | Subject | Author | Recs | Date Posted |
136576 | Re: SGMO Pipeline - all you need to know | nightowl | 2 | 11/17/2019 6:33:28 PM |
136594 | Re: SGMO Pipeline - all you need to know | MaxPowerNGS | 1 | 11/18/2019 10:30:36 AM |