MeiraGTx Holdings plc has agreed to purchase all interests in Johnson & Johnson’s investigational candidate for X-linked retinitis pigmentosa (XLRP): botaretigene sparoparvovec (bota-vec).
First, let’s get a look at MeiraGTx.
Operating out of London and New York, the clinical-stage genetic medicines company was founded in 2015 as a collaboration between University College London (UCL) and Moorfields Eye Hospital.
Since then: It has developed a pipeline of late-stage clinical programs targeting five therapeutic areas (including ophthalmology).
- And in the ophthalmic space, its disease focus includes: XLRP, Stargardt’s disease, wet age-related macular degeneration (AMD), dry AMD / geographic atrophy (GA), and Leber congenital amaurosis (LCA4; AIPLI-associated congenital blindness).
And what technology are these candidates based on?
That would be MeiraGTx’s roboswitch in-vivo delivery platform, designed to enable the broad application and in-vivo delivery of any therapeutic protein.
- Learn more about this here.
Now to this XLRP therapeutic.
Bota-vec is an investigational gene therapy utilizing an adeno-associated virus (AAV5) vector to deliver genetic material into retinal cells.
In the context of XLRP: The therapeutic carries a functional version of the RPGR gene, which is defective in most XLRP cases.
- Its mode of administration: Is via a subretinal injection, enabling it to reach retinal photoreceptor cells to target XLRP.
- Its regulatory status: Though investigational, bota-vec has already received two FDA designations: Fast Track and Orphan Drug.
And what do we know about its development?
The drug was initially developed by J&J’s Janssen unit in alignment with MeiraGTx.
In fact: Bota-vec was originally included in a 2019 deal between the two companies worth up to $440 million, in which they agreed to a global collaboration and license agreement to develop, manufacture, and commercialize a portfolio of inherited retinal disease (IRD) drugs.
As such: MeiraGTx and J&J have partnered on bota-vec’s clinical development and investigation since the phase 1 stage (though J&J took the lead after fully acquiring the rights in 2023).
So what has this entailed?
The global, randomized phase 3 LUMEOS study (NCT04671433) evaluated bota-vec’s bilateral treatment as a single low or high dose on vision-guided mobility among XLRP patients.
The findings: As reported in May 2025, the trial failed to meet its primary endpoint.
- However, secondary endpoint data were reported to be “very strong, with clinically meaningful and statistically significant improvements demonstrated in each of the three domains of vision.”
- See here for those numbers.
And check out a full rundown on the study’s “meaningful improvements across multiple outcome measures with 10-and 15-letter gains.”
And where does that leave this drug?
In announcing its acquisition, MeiraGTx stated that it has heard from “numerous investigators about the clinically meaningful benefit that bota-vec has afforded a significant number of patients” who participated in the LUMEOS study.
In fact: Following that phase 3 reporting last year, the Foundation Fighting Blindness actually issued a public letter of support for the filing (and eventual approval) of a bota-vec to treat XLRP across the globe.
- Read the full statement here.
And more recently, FFB CEO Jason Menzo noted: “The data from the LUMEOS phase 3 study of bota-vec, reflected in both objective measures and patient-reported outcomes, point to real improvements in vision.”
Does this mean an FDA submission is in the works?
The plan is for MeiraGTx to “expeditiously file for approval” a biologics license application (BLA) in the United States—as well as in the European Union and Japan, according to the CEO—with a target launch date of 2027.
- According to President and CEO Alexandria Forbes, PhD, from a regulatory chemistry, manufacturing, and control (CMC) standpoint: “We have completed PPQ (process performance qualification) with the CMC datasets for filing with global regulators.”
- To note: PPQ is critical for obtaining regulatory approval.
And is this approval part of bota-vec’s acquisition deal?
Indeed, it is. While MeiraGTx will pay J&J a $25 million upfront cash repayment for the drug, the agreement also includes a one-time regulatory and commercial milestone tied to:
- Its (potential) U.S. approval and U.S. sales performance
- A high double-digit royalty on global net sales, starting in 2029
So when can we expect this FDA submission?
That’s currently to be determined … so stay tuned!