Janssen and MeiraGTx Link on Optical Gene Therapy

Janssen, the pharmaceuticals arm of Johnson & Johnson, is once again collaborating with US clinical-stage gene therapy company MeiraGTx to develop and commercialize treatments for inherited retinal diseases (IRDs).

This is Janssen’s second partnership with the New York-based company, having entered into a research collaboration in October 2018 to develop gene therapy treatments using MeiraGTx’s proprietary riboswitch technology.

Under the latest agreement, the companies will work together on MeiraGTx’s leading IRD pipeline, including product candidates for achromatopsia (ACHM) caused by mutations in either CNGB3 or CNGA3 and X-linked retinitis pigmentosa (XLRP).

MeiraGTx will receive $100 million in cash up front and is eligible to receive additional payments for achieving development and sales milestones related to its CNGB3, CNGA3 and XLRP programs of up to $340 million. In addition it is eligible for untiered royalties of 20% of annual net sales for the CNGB3, CNGA3 and XLRP programs. Janssen has agreed to fund all clinical development and commercialization costs for the programs.

In addition, both companies will enter into a research collaboration to develop a pipeline of IRD gene therapy candidates, with Janssen paying a “significant” portion of the costs.

MeiraGTx currently has three ongoing clinical programs in IRDs, with a fourth expected to enter clinical development in the first half of 2019, as well as a deep pipeline of pre-clinical IRD programs for a variety of gene mutations.

A third collaboration will see the two companies further develop AAV manufacturing technology as well as entering into clinical and commercial manufacturing  supply agreements for the clinical and research programs.

“By combining Janssen’s extensive clinical, regulatory and commercial expertise and global reach with MeiraGTx’s deep experience in gene therapy development and manufacturing, we aim to accelerate the development of our pipeline of potential IRD gene therapies to address the needs of patients globally,” said Alexandria Forbes, president CEO of MeiraGTx.

Janssen has the right to opt-in to programs coming out of the research collaboration at the time of clearance of an Investigational New Drug (IND) application by the US Food and Drug Administration (FDA).

The transaction, which remains subject to customary closing conditions, is expected to close in the first quarter of 2019.

IRDs are a group of rare eye conditions caused by an inherited gene mutation that are often characterized by progressive retinal degeneration, which leads to severe vision impairment, loss or blindness.