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Recently, MeiraGTx announced Johnson & JohnsonFor up to $415 million, acquiring its gene therapy botaretigene sparoparvovec for treating X-linked retinitis pigmentosa (XLRP)(bota-vec, previously known as AAV-RPGR).MeiraGTx to Receive $130 Million in Upfront and Near-Term Milestone Payments, Plus up to $285 Million in Additional Funds After the First Commercial Sales of Bota-Vec in the U.S. and EU, as Well as Manufacturing Technology Transfer.Bota-vec is a gene therapy based on adeno-associated virus (AAV) vector., used to treat patients with XLRP caused by mutations in the eye-specific RPGR gene (RPGR 0RF15).Bota-vecAimed to deliver a functional copy of the RPGR gene to the subretinal space to improve and maintain visual function.At the 2020 Virtual Annual Meeting, 6-month data from the Phase I/II trial of AAV-RPGR was announced: interim data showed lowLow and medium doses of AAV-RPGR were well tolerated and showed significant improvement in vision.。Bota-vec Phase 3 Lumeos Clinical Trial Completed Patient Enrollment in 2023.According to the data from this study, the European Medicines Agency (EMA) granted Bota-vec PRIME designation and orphan drug status, and the U.S. FDA also granted it Fast Track designation and orphan drug status.MeiraGTx CEO, Dr. Alexandria ForbesMeiraGTx President and CEO, Dr. Alexandria Forbes, said: "This collaboration has provided us with a significant amount of cash, a $130 million upfront payment, plus the $30 million investment we received from Sanofi in October.Will extend our cash runway to mid-2026。”MeiraGTx Holdings is a vertically integrated clinical-stage gene therapy company with four ongoing clinical programs and a range of preclinical and research programs. It has core competencies in viral vector design and optimization, gene therapy manufacturing, and potential transgene regulation technologies.https://investors.meiragtx.com/news-releases/news-release-details/meiragtx-announces-asset-purchase-agreement-involving-its-aav
