1. A clinical-stage **Sarepta Therapeutics**, but focusing on gene therapies for inherited blindness and neurological diseases.

2. An early-stage **Vertex Pharmaceuticals**, dedicated to developing gene therapies for serious eye and brain disorders.

• Gene Therapies for Achromatopsia: Treatments targeting Achromatopsia, a form of inherited blindness, currently in Phase 1/2 clinical development.
• Gene Therapies for X-Linked Retinitis Pigmentosa: Treatments for X-Linked Retinitis Pigmentosa, an inherited retinal disease, currently in Phase 1/2 clinical development.
• Gene Therapies for RPE65-deficiency: Treatments targeting RPE65-deficiency, a cause of inherited retinal degeneration, currently in Phase 1/2 clinical development.
• Gene Therapies for Radiation-Induced Xerostomia: Treatments designed to address severe dry mouth following radiation treatment for head and neck cancers, currently in Phase 1/2 clinical development.
• Gene Therapies for Parkinson's Disease: Treatments in development for Parkinson's disease, a neurodegenerative disorder.
• Gene Therapies for Xerostomia related to Sjogren's Syndrome: Treatments in the early stages of clinical program initiation for dry mouth associated with Sjogren's syndrome.

MeiraGTx (MGTX) is a clinical-stage gene therapy company. At this stage, it does not sell commercial products directly to individuals or healthcare providers. Instead, its primary financial relationships involve collaborations with larger pharmaceutical companies for research and development.

Based on the provided information, MeiraGTx's major customer/partner is:

• Janssen Pharmaceuticals, Inc. (a subsidiary of Johnson & Johnson, symbol: JNJ)

This relationship is described as a research collaboration agreement to develop regulatable gene therapy treatment.

Alexandria Forbes, Ph.D. President and Chief Executive Officer

Dr. Alexandria Forbes has served as President, Chief Executive Officer, and a member of the board of directors of MeiraGTx since March 2015. Prior to co-founding MeiraGTx, she was the Senior Vice President of Commercial Operations at Kadmon Holdings, Inc., a biopharmaceutical company, from September 2013 to April 2015. Dr. Forbes spent eleven years as a healthcare investor, including roles at Sivik Global Healthcare from September 2000 to November 2008, where she was portfolio manager of the Sivik Global Life Science Fund, and Meadowvale Asset Management from January 2010 to June 2012, both healthcare hedge funds.

Richard Giroux Chief Operating Officer and Chief Financial Officer

Richard Giroux has served as Chief Operating Officer of MeiraGTx since March 2015 and took on the additional role of Chief Financial Officer in April 2019. He is a co-founder of MeiraGTx. Before joining MeiraGTx, Mr. Giroux was a partner at Sarissa Capital Management LP, an activist healthcare hedge fund, from March 2014 to March 2015. He was also a founding partner and healthcare portfolio manager of Meadowvale Partners, a multi-strategy hedge fund, from January 2010 to June 2012. Earlier in his career, he served as a partner at Sivik Global Healthcare (formerly Argus Partners), a healthcare hedge fund, from August 2001 to November 2008, and worked at investment banks Salomon Smith Barney and Goldman Sachs.

Stuart Naylor, Ph.D. Chief Development Officer

Dr. Stuart Naylor has served as the Chief Development Officer of MeiraGTx since April 2015. His prior experience includes serving as Chief Executive Officer of Athena Vision, a biotechnology company, from April 2015 to April 2016, and as managing director of Coltivare Ltd., a healthcare consulting company, from June 2013 to April 2015. From 2008 to 2013, Dr. Naylor was the Executive Director and Chief Scientific Officer of Oxford BioMedica plc, a gene therapy company.

Robert K. Zeldin, M.D. Chief Medical Officer

Dr. Robert Zeldin joined MeiraGTx as Chief Medical Officer in August 2020, bringing over two decades of clinical, regulatory, and industry experience. Before MeiraGTx, he served as CMO of Immunovant and Acceleron Pharma, where he played a key role in the biologics license application for luspatercept. He also served as CMO of Ablynx NV, a Belgian company, where he directed the Phase 3 development program for caplacizumab.

Christine Sheehy SVP, Global Integration

Christine Sheehy serves as the Senior Vice President of Global Integration at MeiraGTx, with over 25 years of experience in establishing operational, financial, and organizational infrastructures in the pharmaceutical industry. She previously held the position of SVP of Operations for Kadmon Pharmaceuticals, where she oversaw global supply chain, distribution, and IT operations. Ms. Sheehy was also a founding member of Three Rivers Pharmaceuticals, a start-up pharmaceutical company that was acquired by Kadmon in 2010.

The key risks for MeiraGTx (MGTX) are primarily associated with the inherent challenges of being a clinical-stage gene therapy company.

1. Clinical Trial Success and Regulatory Approval: As a clinical-stage company, MeiraGTx's success is heavily dependent on the positive outcomes of its ongoing and future clinical trials for its various gene therapies. There is a constant risk of catastrophic clinical trial failure or unexpected safety issues, which could halt the entire pipeline. Even with promising early data, a single unforeseen safety event in a pivotal study can derail development. Furthermore, the company faces significant risks concerning regulatory approval, as failure to obtain timely FDA or other regulatory clearances for its product candidates could lead to delays or prevent commercialization altogether.

2. Financial Viability and Funding: MeiraGTx operates with significant net losses, which is common for clinical-stage biotechnology firms, and currently generates no commercial revenue, relying entirely on capital raises and payments from partnerships. The development of gene therapies is capital-intensive, and the company has experienced sharp declines in cash, cash equivalents, and restricted cash, indicating a serious liquidity risk. Analysts have pointed to a low Piotroski F-Score and a distressing Altman Z-Score, suggesting poor business operations and a potential risk of bankruptcy within two years. The ongoing need for capital raises also carries the risk of diluting shareholder equity.

3. Intense Competition and Intellectual Property: The gene therapy sector is a highly competitive landscape with well-funded rivals such as Novartis and Roche. MeiraGTx must continually innovate and comply with regulations to maintain its market position against fierce competition. The company also faces risks related to protecting its intellectual property, which is crucial for its proprietary riboswitch technology and other gene therapy platforms.

Addressable Markets for MeiraGTx's Main Products or Services

• Ocular Diseases:
  • Achromatopsia: The global achromatopsia market across the top 7 markets (US, EU4, UK, and Japan) was valued at approximately USD 111.5 million in 2024 and is projected to reach USD 187.8 million by 2035. The United States represents the largest market for achromatopsia treatment.
  • X-Linked Retinitis Pigmentosa (XLRP): The total Retinitis Pigmentosa treatment market size in the 7MM (United States, EU4, UK, and Japan) was approximately USD 500 million in 2023. The global Retinitis Pigmentosa market size is expected to grow from USD 14.27 billion in 2024 to USD 30.69 billion by 2035. In the US, the total market size for Retinitis Pigmentosa was estimated to be nearly USD 270 million in 2023.
  • RPE65-deficiency: This genetic mutation is associated with inherited retinal dystrophies, including some forms of Retinitis Pigmentosa. Treatments for RPE65 mutations fall under the broader Retinitis Pigmentosa market.
• Xerostomia:
  • Radiation-induced Xerostomia and Xerostomia related to Sjögren's Syndrome: The global xerostomia therapeutics market was valued at approximately USD 2.13 billion in 2024 and is expected to reach USD 2.87 billion by 2032. North America dominated the market in 2024 with a share of over 36.8%. Specifically, the Sjögren's Syndrome market globally is estimated at USD 178.5 billion in 2025 and is projected to reach USD 239.9 billion by 2035. The U.S. Sjögren's Syndrome market is estimated at USD 64.8 billion in 2025 and is anticipated to reach USD 83.4 billion by 2035.
• Neurodegenerative Diseases:
  • Amyotrophic Lateral Sclerosis (ALS): The global amyotrophic lateral sclerosis treatment market size was valued at USD 713.3 million in 2023 and is projected to exhibit a 5.5% CAGR from 2024 to 2032, reaching USD 1.1 billion by 2032. North America held the largest revenue share of 37.7% in 2023.
  • Parkinson's Disease: The global Parkinson's disease treatment market size was estimated at around USD 4.3 billion in 2021 and is projected to hit around USD 12.1 billion by 2030. Another source indicates the global Parkinson's disease therapeutics market was valued at USD 6.2 billion in 2024 and is set to reach USD 13.3 billion by 2034. The Parkinson's Disease Market Size in the US was approximately USD 1,883 million in 2023.

MeiraGTx (MGTX) has several anticipated drivers for future revenue growth over the next 2-3 years, primarily stemming from advancements in its late-stage clinical pipeline and strategic collaborations:

1. Commercialization and milestone payments from bota-vec: MeiraGTx is eligible to receive up to $285 million upon the first commercial sales of bota-vec in the US and EU, along with revenue from a commercial supply agreement for bota-vec manufacturing with Johnson & Johnson Innovative Medicine.
2. Advancement and potential commercialization of AAV-GAD for Parkinson's disease: The strategic collaboration with Hologen AI provides $200 million upfront, with an additional $230 million committed to a joint venture to fund the development of the AAV-GAD program through commercialization. A Biologics License Application (BLA) filing for this program is anticipated by 2027.
3. Potential regulatory approval and launch of AAV-AIPL1 for Leber Congenital Amaurosis type 4 (LCA4): MeiraGTx is pursuing expedited approval pathways for this therapy in the UK (Marketing Authorization Application under exceptional circumstances) and is in discussions with the FDA for a similar pathway in the US, based on positive clinical data.
4. Potential regulatory approval and launch of AAV-hAQP1 for radiation-induced Xerostomia (RIX): The company has gained alignment with the FDA on the ongoing Phase 2 AQUAx2 study, which is designed to support a potential BLA filing, with data expected in late 2026.
5. Partnership revenue from its riboswitch gene regulation technology platform: MeiraGTx is progressing its riboswitch programs into the clinic, initially focusing on metabolic diseases. This proprietary platform has the potential to generate revenue through collaborations and milestone payments from partners like Janssen Pharmaceuticals.

Share Repurchases

• In December 2025, MeiraGTx repurchased 2.3 million ordinary shares from Perceptive Life Sciences Master Fund for $18.2 million.

Share Issuance

• In October 2023, Sanofi made a $30 million strategic investment by purchasing 4.0 million ordinary shares.

Inbound Investments

• In November 2025, MeiraGTx entered a strategic collaboration with Eli Lilly and Company in ophthalmology, receiving an upfront payment of $75 million and eligibility for over $400 million in total milestone payments.
• In March 2025, a strategic collaboration with Hologen AI was announced, including a $200 million upfront payment to MeiraGTx and up to an additional $230 million in committed funding to a joint venture for the AAV-GAD program for Parkinson's disease.
• In December 2023, MeiraGTx entered an asset purchase agreement with Janssen Pharmaceuticals for its X-linked Retinitis Pigmentosa (XLRP) gene therapy (bota-vec) for up to $415 million, which included $130 million in upfront and near-term milestone payments.

Outbound Investments

• In July 2025, MeiraGTx Holdings completed a merger/acquisition with Smart Immune.

Capital Expenditures

• MeiraGTx maintains a vertically integrated manufacturing platform with two Good Manufacturing Practice (GMP) viral vector production facilities and a GMP Quality Control facility, reflecting significant investment in infrastructure.
• The company's research and development expenses increased to $32.5 million in the third quarter of 2025, up from $26.2 million in the same period of 2024, indicating accelerated investment in clinical programs.
• As of March 2025, the company projected it would have sufficient capital, including proceeds from strategic collaborations, to fund operating expenses and capital expenditure requirements into 2027.