Janssen collaborates on gene therapies for retinal disorders

By Maggie Lynch

- Last updated on GMT

(Image: Getty/ Kritchanut)
(Image: Getty/ Kritchanut)

Related tags Rare disease Gene therapy Gene therapy candidate Investment Investigational new drug preclinical Preclinical contract research

Janssen enters collaboration with MeiraGTx for the development and commercialization of retinal disorder gene therapies.

The companies will collaborate on the clinical development of MeiraGTx’s inherited retinal disorder (IRD) pipeline, including its candidates for achromatopsia.

Janssen will pay MeiraGTx $100m (€87.5m) in cash as an upfront payment, with potential additional payments upon meeting development and sales milestones of up to $340m. Janssen has also agreed to fund all clinical development and commercialization costs for these programs.

IRDs are a class of rare eye conditions caused by an inherited gene mutation that are often characterized by progressive retinal degeneration, leading to vision impairment, loss, or blindness. A spokesperson for MeiraGTX told us, "There are 200,000 people with IRDs in the US, caused by 300 or more different genetic defects."

MeiraGTx’s pipeline for IRD treatments are gene therapies for conditions that develop from mutations in the CNGB3 or CNGA3 and XLRP genes.

The company currently has three ongoing clinical programs for IRD products, with a fourth program expected to enter clinical development along with its pre-clinical IRD pipeline.

Janssen will also enter a research collaboration with MeiraGTx covering the former’s pre-clinical IRD program pipeline.

Janssen will fund a significant portion of the costs and has the right to opt-in to programs coming out of the research collaboration when an investigational new drug (IND) application is submitted. Janssen has agreed to fund all clinical development and commercialization costs if it chooses to opt-in.

Additionally, the collaboration will also include the development of AAV manufacturing technology, and clinical and commercial manufacturing supply agreements for clinical and research programs.

Alexandria Forbes, CEO of MeiraGTx, said in a statement, “This important collaboration supports our leading position in gene therapy development and manufacturing and reaffirms our commitment to advancing a broad portfolio of breakthrough gene therapies that may improve the lives of patients suffering from IRDs worldwide.”

The two companies have collaborated before, having entered a research agreement in October 2018, for “regulatable gene therapy treatments”​ using MeiraGTx’s proprietary riboswitch technology.

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