Johnson & Johnson has made its biggest move into gene therapy, signing a $100m deal with UK-headquartered biotech MeiraGTx for rights to two genetic eye diseases programmes.
J&J is licensing candidates for two inherited retinal disorders – achromatopsia (ACHM) caused by mutations in either CNGB3 or CNGA3, and X-linked retinitis pigmentosa (XLRP) – which are in phase 1/2 clinical trials.
ACHM is a non-progressive condition characterised by partial or total absence of colour vision, while XLRP, caused by mutations in the RPGR gene, leads to progressive vision loss.
In addition to the $100m upfront payments, J&J’s Janssen division is proffering up to $340m in additional payments if the programs progress towards the market, plus royalties on any future sales, and has agreed to fund clinical trials.
In return it has claimed worldwide rights to the gene therapies, which represent a sizeable chunk of MeiraGTx’ clinical pipeline, as well as taking an option on preclinical-stage follow-ups for inherited retinal disorders. MeiraGTx is also working on gene therapies for salivary gland problems and neurodegenerative diseases.
The companies will also work together on new targets for other inherited retinal diseases and on adeno-associated virus (AAV) manufacturing technology.
J&J has made some small investments in gene therapies over the last few years, but this is its biggest move to date, and comes after it started working with MeiraGTx last year on a project looking at using the biotech’s riboswitch technology – essentially an on-off switch for delivered genes – to engineer “regulatable” gene therapies.
J&J’s earlier forays into this area include a collaboration with the University of Pennsylvania on an Alzheimer’s programme, announced in early 2018.
The deal comes at a time when gene therapies finally seem to be about to fulfil their promise, after decades of research.
In Europe, the first approved gene therapy – UniQure’s Glybera – was launched way back in 2012 but was withdrawn five years later for commercial reasons. GlaxoSmithKline launched its Strimvelis product in 2016, but sold it to Orchard Therapeutics last year after opting to exit the rare disease category.
The US market developed a little later, with Spark Therapeutics’ Luxturna (voretigene neparvovec) for biallelic RPE65-mediated inherited retinal disease claiming FDA approval at the end of 2017, with a green light in Europe – where it will be sold by Novartis – coming through last year.
Another gene therapy, Novartis’ Zolgensma for spina muscular atrophy, is heading for regulatory decision in the US this year and in Europe in 2020.