Our Commitment to Patients

At MeiraGTx, patients and their families are the cornerstone of everything we do. We are continually asking how our research and our therapies can potentially improve the lives of patients living with serious diseases. Each day, we work to improve the lives of patients through cutting-edge science designed to treat their specific disease with the best treatment possible.

To help us with this mission, we have established, and continue to build, strong relationships with patient advocacy groups around the world to understand how to best serve patients and their families. The clear lines of communication that flow to and from our patient advocates actively help inform and guide our research and clinical development decisions. These relationships also help patients and their families stay up-to-date on our clinical trial progress.

Find out more about our clinical trials by visiting www.clinicaltrials.gov and searching ‘MeiraGTx’ or by contacting us at patients@meiragtx.com, and learn more about our expanded access program by clicking here.


Inherited Retinal Disease

We currently have four ongoing Phase 1/2 clinical programs in inherited retinal diseases (IRDs), along with a compassionate use program for those with Leber congenital amaurosis (LCA) due to mutations in the AIPL1 gene. IRDs are a group of rare eye disorders caused by an inherited gene mutation that can result in individuals experiencing severe vision loss, impairment or blindness. Our four ongoing Phase 1/2 clinical stage programs include: Achromatopsia (ACHM) due to mutations both in in the CNGB3 and CNGA3 genes, X-Linked Retinitis Pigmentosa (XLRP) and RPE65-deficiency. Both our AAV-RPE65 and AAV-CNGB3 Phase 1/2 clinical trials were completed in 2019.

MeiraGTx Clinical Trials

Learn more about MeiraGTx’s ocular programs and determine if you may be eligible to participate in one of our IRD gene therapy clinical trials or ongoing natural history studies:

To learn more about our compassionate use program for those with LCA due to mutations in the AIPL1 gene, please email patients@meiragtx.com.

IRD Patient Resources

To find out more about genetic testing for IRDs, please visit the My Retina Tracker website provided by the Foundation Fighting Blindness.

For additional information about IRDs, please visit the following organizations to find resources and support:

Parkinson’s Disease

More than 10 million people worldwide are living with Parkinson’s disease. Parkinson’s disease is a lifelong, neurodegenerative disease that occurs when brain cells that make dopamine, a chemical that coordinates movement, stop working. The disease is associated with a progressive loss of motor control (e.g., shaking or tremor at rest and lack of facial expression), as well as non-motor symptoms (e.g., depression and anxiety). Each year, approximately 60,000 Americans are diagnosed with Parkinson’s disease.

We are currently developing AAV-GAD, an investigational gene therapy designed to deliver the glutamic acid decarboxylase (GAD) gene to the brain in order to normalize motor circuits and improve symptoms associated with Parkinson’s disease without affecting other areas of the brain.

Patient Resources

For additional information about Parkinson’s disease, please visit the following organizations to find resources and support:

Radiation-Induced Xerostomia

Xerostomia is a chronic and debilitating disorder of the salivary glands in which saliva production is impaired. Xerostomia has a number of different potential causes but may result from radiation therapy for head and neck cancer or be caused by certain autoimmune diseases such as Sjogren’s Syndrome.

We are developing AAV-AQP1 to treat radiation-induced xerostomia (RIX). AAV-AQP1 is an investigational gene therapy that was designed to increase water conduction in salivary glands that have been damaged by radiation therapy. The gene therapy introduces a water conducting channel into the damaged glands. We are currently conducting a Phase 1 clinical trial in patients who have lived cancer free for five or more years following treatment for head and neck cancer and are suffering from grade 2 or 3 RIX.

MeiraGTx Clinical Trials

MeiraGTx is currently recruiting patients to participate in the following salivary gland gene therapy clinical trials:

Patient Resources

For additional information about xerostomia, please visit the following organizations to find resources and support:

Rare Diseases

It’s estimated there are more than 7,000 different types of rare diseases. In the U.S., a rare disease is defined as a condition that affects fewer than 200,000 people. The exact cause of many rare diseases is unknown, however the majority are thought to be genetic, directly caused by changes in genes or chromosomes, while others may occur randomly. Rare diseases are estimated to affect 25 to 30 million Americans. While individual patient numbers of specific diseases may be small, the community as a whole is large, and MeiraGTx is proud to be a part of the millions of people working towards finding cures to these currently untreatable diseases.

Rare Disease Patient Resources

For additional information about rare diseases, please visit the following organizations to find resources and support: