The Center for Drug Evaluation (CDE) website has indicated that Johnson & Johnson (J&J, NYSE: JNJ)’s AAV5-hRKp.RPGR (JNJ-5340, botaretigene sparoparvovec) intraocular injectable solution is on track to receive breakthrough therapy designation (BTD). This product, an adeno-associated virus vector (AAV) based gene therapy co-developed with MeiraGTx Holdings plc, is aimed at treating X-linked retinitis pigmentosa (RPGR-XLRP) in patients diagnosed with a pathogenic variant of the RPGR gene. It is essential that the treating physician confirms the presence of sufficient surviving retinal cells for treatment.
Johnson & Johnson and MeiraGTx entered into an agreement in 2019 to develop genetic therapies for hereditary eye diseases, including JNJ-5340 alongside two other gene therapies for total color blindness, JNJ-5288 and JNJ-5301. At the end of 2023, J&J acquired the development rights to JNJ-5340 for a sum of USD 415 million.- Flcube.com
