Janssen and MeiraGTx to Develop Gene Therapy Programs for Inherited Retinal Diseases

Originally Published by Johnson & Johnson.

The Janssen Pharmaceutical Companies of Johnson & Johnson announced a worldwide collaboration and license agreement with MeiraGTx Holdings plc (NASDAQ:MGTX), a clinical-stage gene therapy company, to develop, manufacture and commercialize its clinical stage inherited retinal disease portfolio, including leading product candidates for achromatopsia (ACHM) caused by mutations in either CNGB3 or CNGA3, and X-linked retinitis pigmentosa (XLRP). Further, the companies have formed a research collaboration to explore new targets for other inherited retinal diseases and further develop adeno-associated virus (AAV) manufacturing technology.


“Janssen is excited to expand our portfolio with the addition of innovative assets that have the potential to improve sight or prevent the progression to blindness in inherited retinal diseases which currently have no treatment options,” said James List, M.D., Ph.D., Global Therapeutic Area Head, Cardiovascular & Metabolism, Janssen Research & Development, LLC. “We look forward to partnering with MeiraGTx to develop these assets and explore new potential targets for inherited retinal diseases.”

Under the terms of the agreement, MeiraGTx will grant Janssen an exclusive worldwide license to certain clinical assets in MeiraGTx’s inherited retinal disease portfolio. The companies have also formed a research collaboration to develop a pipeline of products addressing novel gene targets, giving Janssen the exclusive option to license new treatments for other inherited retinal diseases.

“This collaboration builds on Janssen’s long-standing heritage of addressing the unmet needs of patients around the world,” said Mathai Mammen, M.D., Ph.D., Global Head, Janssen Research & Development, LLC. “Through this collaboration we look forward to deepening our expertise in gene therapy and leveraging our breadth of research and development expertise to help bring forward new treatment options for people living with inherited retinal diseases.”

About Achromatopsia, AAV-CNGB3, and AAV-CNGA3

Achromatopsia is an inherited retinal disease that prevents cone photoreceptors from functioning. Patients are legally blind from birth and usually suffer from severely reduced visual acuity of 20/200 or worse; a disabling sensitivity to light, or photophobia; total color blindness; and involuntary back and forth eye movements, or nystagmus. There are currently no approved treatments for achromatopsia.

AAV-CNGB3 and AAV-CNGA3, gene therapy candidates designed to restore cone function, are delivered via subretinal injection to the area of the eye where most of the cones in the retina are located. AAV-CNGB3 was granted orphan drug designation (ODD), rare pediatric disease and Fast Track designations by the U.S. Food and Drug Administration (FDA), and orphan medicinal product and PRIME designations by the European Medicines Agency (EMA) for the treatment of achromatopsia caused by mutations in the CNGB3 gene. A Phase 1/2 clinical trial of AAV-CNGB3 in both adult and pediatric patients is underway.

About X-Linked Retinitis Pigmentosa and AAV-RGPR

X-linked retinitis pigmentosa (XLRP) represent some of the most severe forms of RP, resulting in early onset in childhood and rapid progression to blindness by the time patients reach 20 to 30 years old. In XLRP, both rods and cones function poorly, leading to degeneration of the retina and total blindness. There are currently no approved treatments for XLRP.

AAV-RGPR is designed to treat the most common form of XLRP caused by mutations in the RPGR gene. Both rod and cone photoreceptors require RPGR to function. AAV-RPGR has received Fast Track designation and ODD from the FDA and orphan medicinal product designation from the EMA. A Phase 1/2 clinical trial of AAV-RPGR in adult and pediatric patients is underway.

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