![]() Schultz, MD / Ruth Works Professor of Ophthalmology Professor of Ophthalmology & Visual Sciences, Biophysics, and Cell Biology, Neurobiology and Anatomy Director, Advanced Ocular Imaging Program ![]() Natural History Study: We also have an ongoing prospective natural history study for patients with achromatopsia.įor more information about our ACHM clinical programs, please visit the MeiraGTx website or email Carroll, Ph.D. It was designed with a synthetic promoter associated with strong gene expression to account for the larger amount of protein needed to restore cone function in patients with a CNGA3 gene mutation. Investigational Gene Therapies for the Treatment of Achromatopsia (ACHM):ĪAV-CNGB3, a gene therapy treatment designed to restore cone function, is delivered via subretinal injection to the area of the eye where most of the cones in the retina are located.ĪAV-CNGA3, a gene therapy treatment designed to restore cone function, is delivered to the cone receptors at the back of the eye via subretinal injection. The portfolio expands Johnson & Johnson’s already broad ophthalmic footprint and includes a pipeline of innovative treatments for inherited retinal diseases, as well as more common retinal diseases affecting millions of people worldwide. Janssen established its Retinal Disease portfolio in 2018 to develop transformational therapies and platforms that restore and preserve vision. We are proud to have Janssen Pharmaceuticals, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson as our development partner as part of a global collaboration to develop and commercialize gene therapies for the treatment of IRDs. We are currently conducting clinical trials of investigational gene therapies for the treatment of achromatopsia (ACHM) caused by mutations in either the CNGB3 gene or the CNGA3 gene. In partnership with leading experts around the world, we are developing investigational gene therapies for the treatment of multiple inherited retinal diseases (IRDs). MeiraGTx is a clinical-stage gene therapy company focused on developing potentially curative treatments for patients living with serious diseases. This video demonstrates the effects of gene therapy in treated dogs. History of Gene Therapy: Gene therapy rescues cone function in congenital Achromatopsia: Gene Therapy in a Sheep Model of CNGA3 Achromatopsia: ARVO 2014 click here to view īanin E, Gootwine E, Obolensky A, Ezra-Elia R, Ejzenberg A, Zelinger L, Honig H, Rosov A, Yamin E, Sharon D, Averbukh E, Hauswirth WW, Ofri R.” Gene Augmentation Therapy Restores Retinal Function and Visual Behavior in a Sheep Model of CNGA3 Achromatopsia.”Mol Ther. More about this study can be found HERE ( click to read). ![]() Then you see the same sheep (ear tag shown again) after treatment (just one eye) showing its performance markedly improved, actually very nearly normal. Hauswirth at the University of Florida for sharing it with Achroma Corp.įor each animal you see the ear tag before treatment and its poor performance navigating the maze. Sheep ACH Video: This fantastic video of sheep (affected with CNGA3 mutation) that were treated with gene therapy (subretinal-injection) as a result of collaboration between University of Florida, Hadassah-Hebrew University Medical Center, The Volcani Center, and the Hebrew University of Jerusalem. Gene Therapy 101 is summarized in this article by Dr. This vector is then injected under the patient’s retina. Using Adeno-associated virus (AAV), the native (wild type) genes of the virus are removed and replaced with the gene of interest. Gene therapy involves putting a normal copy of the affected gene back into cones of patients. This page highlights some of the work of prominent researchers currently involved with gene therapy. Since our inception in 2012, Achroma Corp has provided $397,144.00* in grants to DIRECTLY support research efforts in expediting a cure.
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