- Johnson & Johnson's Janssen signed a collaboration and licensing deal with MeiraGTx for its inherited retinal disease portfolio, boosting the biotech's stock value almost 30%.
- The deal gives Janssen an exclusive worldwide license to clinical-stage assets including candidates for achromatopsia caused by mutations in either CNGB3 or CNGA3, as well as X-linked retinitis pigmentosa.
- The two companies will collaborate to explore new targets for other inherited retinal diseases and further develop adeno-associated virus (AAV) manufacturing technology. Janssen gains an exclusive option to license candidates from the deal.
Inherited retinal diseases (IRDs) affect around 1 in 2000 people and are the leading cause of vision loss in people between 15 and 45 years of age.
They are also appealing targets for gene therapy companies. The eye is easy to access for dosing, and is immune privileged, meaning the risk of a therapy setting off an immune response is lower.
MeiraGTx's IRD pipeline includes three gene therapies in Phase 1/2 development, including one for RPE65-deficiency that's not a part of the deal with Janssen.
RPE65-mediated IRD is the target of Spark Therapeutics' Luxturna (voretigene neparvovec-rzyl), which became in late 2017 the first gene therapy OK'd in the U.S. for an inherited disorder.
Its approval, in the U.S. and also in Europe, has paved a way for other gene therapies for these types of disorders. While Luxturna has yet to see much in the way of commercial sales, its success helps validate the field and other biotechs working in it.
U.K.-based Nightstar Therapeutics, for example, saw a boost in its share price following Meira's deal with Janssen. Nighstar lists two IRD gene therapies in clinical development, including one for X-lined retinitis pigmentosa that could soon read out fresh data.
Anlaysts viewed the Meira deal as a positive signal of Nightstar's chances of finding a pharma partner or being taken out, indicating forthcoming data could serve to de-risk the company's pipeline.
Joseph Schwartz, an analyst at Leerink, believes that the trend towards acquiring gene therapy companies — seen in buyouts of AveXis and Bamboo Therapeutics — is a "sign of the evolution and maturation of gene therapy as a promising modular therapy."