Advertisement Janssen, MeiraGTx collaborate on gene therapy programs for inherited retinal diseases


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Janssen, MeiraGTx sign $440m deal to develop gene therapy programs for IRDs

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Janssen Pharmaceuticals and MeiraGTx have signed an agreement worth $440m to develop gene therapy programs for inherited retinal diseases (IRDs).

MeiraGTx has five programs in clinical development and a pipeline of preclinical and research programs.

As part of a worldwide collaboration and license agreement, the partnership will develop, manufacture and commercialize clinical stage inherited retinal disease products, including major product candidates for achromatopsia (ACHM) caused by mutations in either CNGB3 or CNGA3 and X-linked retinitis pigmentosa (XLRP).

The firms have also established a research collaboration to assess new targets for other inherited retinal diseases and further develop adeno-associated virus (AAV) manufacturing technology.

Janssen Research & Development cardiovascular and metabolism global therapeutic area head Dr James List said: “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.

“We look forward to partnering with MeiraGTx to develop these assets and explore new potential targets for inherited retinal diseases.”

As per terms of the deal, Janssen will secure an exclusive worldwide license from MeiraGTx for certain clinical assets under its inherited retinal disease portfolio.

The partnership will also engage in the development of various products to address novel gene targets. It will provide Janssen with exclusive option to license new treatments for other inherited retinal diseases.

Achromatopsia is an inherited retinal disease that inhibits cone photoreceptors from functioning. AAV-CNGB3 and AAV-CNGA3, gene therapy candidates have been designed to restore cone function, and delivered through subretinal injection to the area of the eye where most of the cones in the retina are located.

The US Food and Drug Administration (FDA) granted orphan drug, rare pediatric disease and fast track designations for AAV-CNGB3, while the European Medicines Agency (EMA) granted orphan medicinal product and PRIME designations to treat achromatopsia caused by mutations in the CNGB3 gene.

Janssen Research & Development global head Dr Mathai Mammen said: “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.”


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