▎Edited by the WuXi AppTec content teamOn February 4, the official website of China's National Medical Products Administration (NMPA) announced that the AAV5-hRKp.RPGR intravitreal injection solution, jointly submitted by Johnson & Johnson and MeiraGTx, has been included as a proposed breakthrough therapy for the indication ofRPGRPathogenic Variant-Associated X-Linked Retinitis Pigmentosa（RPGR-XLRP) must be determined by the treating physician to have a sufficient number of surviving retinal cells. Public information shows that this is aGene therapy drugs based on adeno-associated virus vectors areJohnson & Johnson Acquires for Over $400 Million.
Screenshot source: CDE official website
XLRP is a slowly progressive hereditary retinal dystrophy. This disease is caused byRPGRCaused by genetic mutations, patients experience night blindness during childhood, leading to peripheral vision loss and complete blindness by middle age. Currently, there are no available treatment options for patients.AAV5-hRKp.RPGR（botaretigene sparoparvovec，bota-vec）InitiallyDeveloped by MeiraGTx,Is aSpecifically Targeting the EyesRPGRTherapy for Gene Mutations。Through a one-time subretinal injection, the functional RPGR gene is delivered by adeno-associated virus (AAV) to compensate for the loss of retinal cells, which is expected to maintain and potentially restore vision in patients with XLRP.。Previously, theTherapyThe method has received Fast Track and Orphan Drug designations from the U.S. FDA, as well as PRIME and Advanced Therapy Medicinal Product designations from the European EMA.In December 2023, Johnson & Johnson reached an agreement with MeiraGTx,Acquisition of the Development Rights for the Gene Therapy Botaretigene Sparoparvovec for the Treatment of XLRP。According to the agreement, MeiraGTx will receive an upfront payment and near-term milestone payments totaling $130 million. Additionally, following the first commercial sales of the drug in the United States and the European Union, as well as the successful transfer of manufacturing technology,MeiraGTx is also expected toReceive milestone payments of up to $285 million.The Phase 1/2 clinical trial of Botaretigene sparoparvovec has been completed in adult and pediatric patients, and the Phase 3 Lumeos clinical trial completed patient enrollment in 2023. It is currently actively conducting dosing studies in patients to investigatebotaretigene sparoparvovecUsed for treatmentRPGRXLRP patients with pathogenic gene variants.Existing experimental data shows that the drugTreatmentXLRP PatientsHasAcceptable Safety, the efficacy evaluation in the proof-of-concept study showedImprovement in Retinal Sensitivity, Visual Acuity, and Functional Vision。[1] Official website of the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of China. Retrieved Feb 4, 2024, fromhttps://www.cde.org.cn/main/xxgk/listpage/9f9c74c73e0f8f56a8bfbc646055026d[2]
MeiraGTx Announces Asset Purchase Agreement Involving its AAV-RPGR
Collaboration for up to $415 million. Retrieved December 21, 2023, from
https://investors.meiragtx.com/news-releases/news-release-details/meiragtx-announces-asset-purchase-agreement-involving-its-aav[3] Michel Michaelides, Jialin Xu, Dai Wang, et al. (2022) AAV5-RPGR (botaretigene sparoparvovec) gene
therapy for X-linked retinitis pigmentosa (XLRP) demonstrates localized
improvements in static perimetry. Investigative Ophthalmology & Visual Science. https://iovs.arvojournals.org/article.aspx?articleid=2781372This article is from the content team of WuXi AppTec. Individuals are welcome to share it on their social media circles, but unauthorized reproduction by media or institutions in any form to other platforms is prohibited. For authorization to reproduce, please contact us via a message on the "MedView" WeChat Official Account. For other cooperation inquiries, please contact wuxi_media@wuxiapptec.com.
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