Patients & Families
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:
MeiraGTx is focused on developing potentially curative treatments for patients living with serious diseases. Our goal is to provide access to our product candidates at the appropriate time and in the correct manner for patients.
Expanded access refers to the use of a product candidate outside the auspices of a clinical trial when the primary purpose is to diagnose, prevent, or treat a serious condition in a patient for which no alternative treatments are available. This is different from a clinical trial, where more comprehensive safety and efficacy data are collected. Expanded access use is usually requested by physicians on behalf of their patients for whom participating in a clinical trial is difficult or impracticable. We understand the need for expanded access programs, and we recognize the importance of having an appropriate policy.
A number of factors consistent with the policies of the U.S. Food and Drug Administration (FDA) and guidelines of international regulatory agencies should be taken into account when considering expanded access. They include:
- The illness must be serious or life-threatening
- There are no other viable treatment options (such as approved products or enrolling clinical trials)
- There is sufficient evidence that the potential benefit to the patient would likely outweigh the potential risks based on safety and efficacy information available at the time
- Ability to provide a product in a fair and equitable manner, so that there is adequate manufacturing capacity for ongoing programs
- Whether granting expanded access would potentially compromise the scientific validity of broader development programs, or interfere with or delay the conduct of current clinical trials or regulatory submissions designed to make the product candidate available to many more patients
At this time, MeiraGTx believes that participation in one of our clinical trials is the most appropriate way to access our product candidates. If you have additional questions, please speak with your physician or contact patients@meiragtx.com. We anticipate acknowledging receipt of requests sent to this e-mail within five business days.
Pursuant to the 21st Century Cures Act, MeiraGTx may revise this policy at any time.
Dated: February 2021
