Strong Partnerships
Strategic investments from J&J & Sanofi and a partnership with Hologen AI, validate MeiraGTx's integrated approach and financial strength.
An overview of the main reasons to invest and the key risks involved.
Strategic investments from J&J & Sanofi and a partnership with Hologen AI, validate MeiraGTx's integrated approach and financial strength.
Proprietary Riboswitch technology offers precise, controlled gene therapy delivery.
Advanced clinical programs targeting high unmet needs in retinal and neurodegenerative diseases like Parkinson's.
Long, uncertain approval process could delay or block therapies.
Trial failures or adverse events could derail progress.
High development costs may strain finances without additional funding or revenue.
MeiraGTx, an emerging leader in genetic medicine, boasts Johnson & Johnson and Sanofi as shareholders, a partnership with Hologen AI – an Eric Schmidt (former Google CEO) company – and has a number of late-stage drugs in its clinical pipeline using its first-gen gene technology. MeiraGTx is pioneering future genetic medicines with its next-gen Riboswitch technology that broadens the application of genetic medicine to a wider range of disease areas.
The company has received two FDA RMAT designations, for its Parkinson’s and Xerostomia programs, further strengthening its regulatory momentum.
Overview of buy and sell case of the business.
Key pieces of information about the business that you need to know about.
Major pharmaceuticals, J&J and Sanofi, have made strategic investments into MeiraGTx, validating its unique pipeline and technology. Plus, the company recently entered a JV with Hologen AI - a venture co-founded by highly regarded individuals including former Google CEO, Eric Schmidt.
MeiraGTx has multiple clinical-stage programs focused on diseases with limited or no treatment options. These include conditions such as X-linked Retinitis Pigmentosa, Inherited Retinal Diseases, Salivary Gland Disorders, and Parkinson’s Disease.
Its gene therapy for Parkinson’s (AAV-GAD), now supported by FDA RMAT designation, aims to slow or stop disease progression — a potential breakthrough. Separately, J&J’s bota-vec gene therapy for vision loss missed its main trial goal but showed meaningful improvements on several secondary measures, and may still advance toward approval.
The key events that could drive investment opportunities and shift markets.
Phase 3 initiation for AAV-GAD (Parkinson’s) – 2025:
A pivotal trial supported by RMAT status and full funding through Hologen AI.
BLA filing for AAV-AQP1 (Xerostomia) – 2026:
RMAT-designated therapy with promising Phase 2 data addresses a significant unmet need in radiation-induced dry mouth.
Expansion of Riboswitch platform into new indications:
Progress in metabolic disease and autoimmune applications could lead to new preclinical partnerships or INDs.
BLA submission for AAV-GAD (Parkinson’s) – 2027:
If successful, could be the first disease-modifying gene therapy for Parkinson’s.
Pre-IND progress across core pipeline:
ALS, Stargardt’s Disease, AMD, and Glaucoma programs represent diversified opportunities in high-need areas.
Next-generation cell and gene therapy innovation:
Advances in in vivo CAR-T and Riboswitch-controlled therapies targeting cancer and autoimmune disease.
Marketing authorization for AAV-AIPL1 (LCA4):
Planned submission to the UK’s MHRA under exceptional circumstances based on vision gains in 11 legally blind children. No further trials required.
Regulatory engagement for AAV-GAD (Parkinson’s):
Following RMAT designation and strong clinical data, FDA interactions may fast-track development and approval discussions.
Manufacturing expansion and monetization:
Ongoing scale-up of MeiraGTx’s GMP viral vector and plasmid production may unlock near-term efficiencies and potential third-party revenue.
Key pieces of information about the business risks that you need to know about.
The path to approval for gene therapies can be long and fraught with regulatory challenges, including the risk of trial delays or rejections that could significantly impact the company’s timeline and financial standing.
With gene therapies still in the clinical phase, there are inherent risks related to trial outcomes. Negative results or adverse events could delay or derail progress.
The capital-intensive nature of gene therapy development requires substantial funding. If MeiraGTx fails to secure additional financing or its therapies do not generate the expected revenue, it could face liquidity challenges
Quickly navigate key insights from industry experts and leverage their knowledge and market intelligence.
Access the most recent investor updates published by the company.
- Announced strategic collaboration with Hologen AI, including a $200 million cash upfront payment to MeiraGTx and the formation of a joint venture, Hologen Neuro AI Ltd, with a further $230 million in capital committed to initially focus on expediting Phase 3 clinical development of AAV-GAD for
Building a Broad-based Gene Therapy Company: From Molecular Genetics to Synthetic Biology. Presented by Dr. Alexandria Forbes of MeiraGTx.A Science in the Ne...
Our Pipeline We currently have multiple programs in clinical development, including the Phase 3 Lumeos clinical study for X-Linked Retinitis Pigmentosa (XLRP), Phase 1/2 clinical stage programs in Achromatopsia (ACHM), and RPE65-Deficiency, Phase 2 clinical study for radiation-induced xerostomia (RIX) and a Parkinson’s program that has completed a Phase 2 trial with published data.
A curated collection of third-party content relevant to the company and sector to help inform your investment decision.
Meira GTx, in Shoreditch, is working on potentially revolutionary treatments for a wide array of illnesses
New data from mouse and rat studies show that MeiraGTx’s gene therapy for amyotrophic lateral sclerosis, or ALS, can repair a key cell-killing process that’s common in both genetic and sporadic for | New data from mouse and rat studies show that MeiraGTx’s ALS gene therapy repairs a key cell-killing process that’s common in both genetic and sporadic forms of the disease.
The MeiraGTx founder and CEO, now on her third career, didn’t take a typical path.
Making therapies feasible from biological, regulatory, and commercial standpoints drives scalable genetic medicine.
Making therapies feasible from biological, regulatory, and commercial standpoints drives scalable genetic medicine.
-- Manufacturing facilities built to accelerate development and delivery of advanced medicines to patients using state-of-the-art technology at scale, with quality appropriate for commercialization -- Site reflects MeiraGTx’s unique, end-to-end approach to gene therapy manufacturing to expedite
Meira GTx, in Shoreditch, is working on potentially revolutionary treatments for a wide array of illnesses
- This RMAT designation is based on data from 3 clinical studies demonstrating the potential benefit of AAV-GAD as a one-time treatment for Parkinson’s disease - RMAT designation includes the benefits of the Fast Track and Breakthrough Therapy designations, allows frequent regulatory interactions
- MeiraGTx to receive $200 million in upfront cash consideration - MeiraGTx and Hologen will form a joint venture, Hologen Neuro AI Ltd, with an additional $230 million committed capital from Hologen to fund 100% of the development of AAV-GAD for Parkinson’s disease through to commercialization, as
As disclosed in the paper, 4 out of 4 young children with the AIPL1-related retinal dystrophy, LCA4, benefited substantially from unilateral subretinal administration of rAAV8 .hRKp.AIPL1 with improved visual acuity, functional vision, and protection against progressive retinal degeneration
MeiraGTx has recently received Rare Pediatric Disease Designation (RPDD) for four inherited retinal diseases (IRDs) reflecting the transformative therapeutic potential of the Company’s proprietary technology platforms LONDON and NEW YORK, Jan. 22, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings Plc
MeiraGTx Holdings plc (NASDAQ: MGTX) on Thursday announced an asset purchase agreement with Janssen Pharmaceuticals Inc, a Johnson & Johnson (NYSE: JNJ) compan
The venture aims to advance MeiraGTx’s Parkinson’s gene therapy through Phase III trials with up to $430m in funding from Hologen.
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MeiraGTx’s $200 million upfront deal with Hologen AI (an AI-driven biotech firm backed by high-profile figures, including ex-Google CEO Eric Schmidt) plus $230 million in committed funding, fully finances AAV-GAD, its gene therapy for Parkinson’s disease, through commercialization. This non-dilutive capital strengthens MeiraGTx’s cash position, alleviating investor concerns over future funding needs.
The partnership uniquely integrates AI into a clinical-stage program, optimizing trial design and patient selection to potentially accelerate approval and enhance efficacy. Hologen’s minority stake in MeiraGTx’s manufacturing subsidiary further scales gene therapy production, a key commercialization hurdle.
With J&J and Sanofi as existing backers, Hologen’s involvement solidifies MeiraGTx’s leadership in AI-driven genetic medicine. Investors view this as a transformational deal, boosting confidence in MeiraGTx’s long-term market potential in a multi-billion-dollar Parkinson’s market.
In 2019, MeiraGTx and Janssen, a subsidiary of Johnson & Johnson, entered an agreement for the clinical development of MeiraGTx’s leading IRD pipeline. MeiraGTx received an upfront payment of $100 million and the potential of additional milestone payments of up to $340 million, along with untiered royalties.
In 2023, MeiraGTx sold full rights to bota-vec to J&J for $130 million upfront, with the potential for $285 million in milestone payments. However, J&J’s recent Phase 3 data missed the primary endpoint, casting uncertainty over future payments. That said, MeiraGTx is not reliant on bota-vec revenues, with its Parkinson’s candidate fully financed by a separate $430 million deal with Hologen AI.
Rare Pediatric Disease Designations (RPDDs) are special designations granted by the FDA to drugs intended to treat serious or life-threatening rare diseases that primarily affect children. MeiraGTx has four RPDDs (Two for Retinal Dystrophy, one for Bardet-Biedl syndrome (BBS) and one for Leber congenital amaurosis).
If a therapy has an RPDD then it can qualify for the FDA's Rare Pediatric Disease Priority Review Voucher (PRV) program which incentivizes the development of treatments for serious or life-threatening rare pediatric diseases .These vouchers can significantly accelerate regulatory review (from 10 months to 6 months), helping to reduce development costs and expedite market entry. PRVs are also a valuable financial asset, as they can be sold for hundreds of millions of dollars, further bolstering MeiraGTx’s cash reserves.
NASDAQ:MGTX
$6.926.79%
$521.00m
-3.11
458k
Pricing delayed 15 mins. Jul 3, 2025 4:00 AM
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