Johnson & Johnson's Ophthalmic Gene Therapy AAV5-hRKp.RPGR (JNJ-5340) Nominated for Breakthrough Therapy Designation in China

On February 4, the CDE website showed that Johnson & Johnson's AAV5-hRKp.RPGR intravitreal injection solution is proposed to be included in the breakthrough therapy designation for the treatment of patients diagnosed with RPGR pathogenic variant-associated X-linked retinitis pigmentosa (RPGR-XLRP).

AAV5-hRKp.RPGR (JNJ-5340, botaretigene sparoparvovec) is a gene therapy drug based on an adeno-associated virus vector, designed to treat XLRP caused by variations in the open reading frame 15 (RPGR ORF15) of the eye-specific RPGR gene. It is currently in Phase III clinical trials for the treatment of X-linked retinitis pigmentosa.

In 2019, Johnson & Johnson signed a collaboration with MeiraGTx to develop gene therapies for inherited eye diseases, including JNJ-5340, as well as two other gene therapy drugs for achromatopsia, JNJ-5288 and JNJ-5301. By the end of 2023, Johnson & Johnson acquired the development rights for botaretigene sparoparvovec in a deal worth up to $415 million.

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