Johnson & Johnson's Ophthalmic Gene Therapy AAV5-hRKp.RPGR (Botaretigene Sparoparvovec) Designated for Breakthrough Therapy in China for RPGR-XLRP
Johnson & Johnson
Healthcare Product Manufacturers, Health Service Providers
On February 4, the CDE website showed that Johnson & Johnson'sAAV5-hRKp.RPGR Intravitreal Injection Solution (AAV-RPGR）Proposed inclusion in the breakthrough therapy designation for the treatment of patients diagnosed with RPGR pathogenic variant-associated X-linked retinitis pigmentosa (RPGR-XLRP).
AAV-RPGR (JNJ-5340, botaretigene sparoparvovec) is aAAV-RPGR, a gene therapy currently under research, is designed to treat patients with XLRP caused by mutations in the eye-specific RPGR gene (RPGR ORF15). AAV-RPGR aims to deliver a functional copy of the RPGR gene to the subretinal space to improve and maintain visual function. AAV-RPGR is jointly developed by Johnson & Johnson and MeiraGTx Holdings for treating XLRP patients with RPGR ORF15 mutations.A Phase III clinical trial for the treatment of X-linked retinitis pigmentosa is currently underway.
In January 2019, Janssen entered into a global collaboration and licensing agreement with MeiraGTx Holdings plc, a clinical-stage gene therapy company, to develop, manufacture, and commercialize its clinical-stage portfolio of inherited retinal disease products. The AAV-RPGR gene therapy was developed under this collaboration and licensing agreement. In addition to forming a research collaboration to explore new targets for other inherited retinal diseases, Janssen also partnered with MeiraGTx to build core capabilities in viral vector design, optimization, and manufacturing.
In addition to AAV-RGRP, the two parties are also developing two gene therapies for the treatment of achromatopsia: AAV-CNGB3 and AAV-CNGA3. These two gene therapies are delivered to the area of the eye where most cone cells are located in the retina through subretinal injection, in order to restore the function of cone cells.
By the end of 2023, Johnson & Johnson acquired the R&D rights to botaretigene sparoparvovec in a deal worth up to $415 million.
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