MeiraGTx Holdings plc
MGTX evidence brief
What we can confirm
- BioSniper Score:
- 4.1 / 10
- Market Cap:
- $1.23B
- Lead Asset:
- aav-aipl1 (Preclinical)
What it means
What to watch
Prepared research question
What matters most for MGTX—catalysts, financial risk and supporting evidence?
Traceable citations · Unknowns marked
BioSniper AI · MGTX
MeiraGTx Holdings plc (MGTX) is a vertically integrated, clinical-stage genetic medicines company with a broad pipeline of late-stage programs targeting severe unmet needs, including radiation-induced xerostomia, Parkinson's disease, and inherited retinal diseases.
Key risk: No product revenue yet
BioSniper Score
Based on 4 of 6 signals
Market Cap
$1.23B
Cash Runway
—
Lead Asset
aav-aipl1 (Preclinical)
Next Catalyst
—
Financial data as of Jun 30, 2026.
Tracking 4 pipeline candidates for MeiraGTx Holdings plc.
MeiraGTx Holdings plc (MGTX) is a vertically integrated, clinical-stage genetic medicines company with a broad pipeline of late-stage programs targeting severe unmet needs, including radiation-induced xerostomia, Parkinson's disease, and inherited retinal diseases.
- Cash & equivalents of $143.2M as of 2026-06-30 [sec].
- Total revenue of $321.4M and net income of $160.7M as of 2026-06-30 [sec].
- Received FDA Breakthrough Therapy Designation for AAV2-hAQP1 for grade 2/3 radiation-induced xerostomia [news · published 2026-08-13].
- Completed acquisition of all rights to botaretigene sparoparvovec (bota-vec) for XLRP from Johnson & Johnson for $25 million [news · published 2026-08-13].
- Entered strategic collaboration with Eli Lilly on AAV-AIPL1 for LCA4, receiving $75 million upfront [sec_mda · as of 2026-03-30].
What works
- Vertically integrated with two GMP viral vector production facilities, internal plasmid production, and in-house Quality Control hub, enabling IND through commercial supply [sec_mda · as of 2026-03-30].
- Strong cash position of $143.2M as of 2026-06-30, bolstered by $100 million equity financing concurrent with recent acquisition [sec] [news · published 2026-08-13].
- Multiple late-stage clinical programs with FDA Breakthrough Therapy Designation for AAV2-hAQP1 and positive three-year data from Phase 1 AQUAx study [sec_mda · as of 2026-08-13].
- Strategic partnerships with Janssen and Eli Lilly providing upfront payments and milestone potential, including over $400 million in total milestones from Lilly [sec_mda · as of 2026-03-30].
What to weigh
- Company is clinical-stage and has not generated any product revenues to date [sec_mda · as of 2026-05-14].
- Since inception, the company has incurred significant accumulated deficits, though the exact figure is not provided in the chunks [sec_mda · as of 2026-05-14].
- Dependence on regulatory and commercial milestones for bota-vec, with potential royalties beginning only in mid-2029 [sec_mda · as of 2026-08-13].
- XLRP program faces competition and no currently approved treatment options, with a small patient population of over 20,000 in U.S. and EU [sec_mda · as of 2026-08-13].
From the filings
Quoted directly from source documents.
FDA Breakthrough Therapy Designation for AAV2-hAQP1
“Received FDA Breakthrough Therapy Designation for AAV2-hAQP1”
News · August 13, 2026
Acquisition of bota-vec from J&J for $25 million
“Completed the acquisition of all interests and rights to botaretigene sparoparvovec (bota-vec) for the treatment of X-linked retinitis pigmentosa (XLRP) from Johnson & Johnson* (J&J) for $25 million”
News · August 13, 2026
Strategic collaboration with Eli Lilly on AAV-AIPL1 for LCA4
“We entered into a strategic collaboration with Lilly, granting Lilly worldwide exclusive rights to the AAV-AIPL1 program for Leber congenial amaurosis 4 (LCA4) and access to additional ocular and gene regulation assets. Under the terms of the agreement, we received an upfront payment of $75 million”
SEC MD&A · March 30, 2026
Risks & what to watch (4)›
Key risks
- No product revenue yet — Company is clinical-stage and has not generated any product revenues to date [sec_mda · as of 2026-05-14].
- Dependence on milestones — Revenue heavily reliant on milestone payments from partners like Lilly and Janssen, with uncertain timing [sec_mda · as of 2026-03-30].
- Small patient populations — Programs target rare diseases (e.g., XLRP with >20,000 patients in U.S./EU), limiting commercial scale [sec_mda · as of 2026-08-13].
- Regulatory and commercial risk — Bota-vec royalties begin only mid-2029, contingent on U.S. approval and sales performance [sec_mda · as of 2026-08-13].
What to watch
- FDA decisions on Breakthrough Therapy Designation for AAV2-hAQP1 and potential regulatory submissions [news · published 2026-08-13].
- Progress of Phase 3 LUMEOS study for bota-vec in XLRP and any updates on approval timelines [sec_mda · as of 2026-08-13].
- Initiation of clinical trials for trigeminal neuralgia program under Hologen collaboration [sec_mda · as of 2026-03-30].
- IND filing for Ribo-Leptin program in metabolic disease and subsequent clinical development [sec_mda · as of 2026-08-13].
Sources: News · SEC MD&A · Generated October 11, 2026 · How we verify sources →
Run MeiraGTx Holdings plc through your own thesis
Use the pipeline, catalyst, financial, FDA, and market facts on this page as inputs to a protocol you control.
Your protocol can:
- Turn available MeiraGTx Holdings plc facts into screen rules or deeper checks
- Show cited findings, blockers, and unresolved evidence together
- Save the logic and rerun it as the company evidence changes
Evidence & data
Score breakdownBased on 4 of 6 signals · derived from public data
Cash runway data unavailable
Net income $161M
Lead: Phase 1
4 candidates
No upcoming catalysts tracked
0 buys · 6 sells (180d)
Pipeline
Financials
Short Interest
as of May 21, 2026- % of float
- 8.8%
- Days to cover
- 5.1
- Shares short
- 5,731,677
- Daily short volume
- -
Short percent of float and days-to-cover reflect reported short positioning. Daily short volume is the exchange-reported short share of a single day's trading volume and is noisier. See the most-shorted biotech ranking →
MGTX short interestGovernance
Frequently asked questions
What is the AI investment thesis for MeiraGTx Holdings plc?
MeiraGTx Holdings plc (MGTX) is a vertically integrated, clinical-stage genetic medicines company with a broad pipeline of late-stage programs targeting severe unmet needs, including radiation-induced xerostomia, Parkinson's disease, and inherited retinal diseases.
What is MeiraGTx Holdings plc's lead drug candidate?
MeiraGTx Holdings plc's most advanced tracked candidate is aav-aipl1 (Preclinical) for Leber congenital amaurosis 4 (LCA4).
Who is the CEO of MeiraGTx Holdings plc?
Dr. Alexandria Forbes Ph.D. is the chief executive officer of MeiraGTx Holdings plc (MGTX).
Where is MeiraGTx Holdings plc headquartered?
MeiraGTx Holdings plc (MGTX) is headquartered in New York, NY, United States.
What therapeutic areas does MeiraGTx Holdings plc focus on?
MeiraGTx Holdings plc develops therapies across Neurology, Ophthalmology.
The AI overview and BioSniper Score are neutral, data-derived signals built from public SEC filings, ClinicalTrials.gov and FDA records — not investment advice.