MeiraGTx is at the forefront of genetic medicine, utilizing adeno-associated virus (AAV) vectors to deliver therapeutic genes for various diseases. Their innovative approach includes the development of riboswitch technology, which allows for precise control of gene expression through small molecules. This technology aims to enhance the dynamic range of gene expression, enabling tailored dosing and potentially improving treatment outcomes for conditions such as Parkinson's disease and various inherited retinal disorders. The company has received multiple designations from regulatory bodies, including Rare Pediatric Disease Designations from the FDA, highlighting their commitment to addressing unmet medical needs in rare diseases.

In addition to their gene therapy platforms, MeiraGTx is advancing a robust pipeline of clinical programs targeting conditions like xerostomia, neurodegenerative diseases, and genetic obesity. Their product candidates, such as AAV-GAD for Parkinson's disease and AAV-AQP1 for xerostomia, are currently in various stages of clinical development, demonstrating promising efficacy and safety profiles. The company’s focus on optimizing vector design, including capsid and promoter engineering, further distinguishes their approach in the competitive landscape of genetic therapies, positioning them to deliver next-generation treatments for complex diseases.

United States

MeiraGTx is a clinical-stage gene therapy company focused on developing potentially curative treatments for patients living with serious diseases. 

Cancer

Central Nervous System (CNS)

Eye Diseases

Genetic Diseases

MeiraGTx

Eye disease

Genetic disorder

X-linked retinitis pigmentosa

AAV-RPGR is designed to treat the most common form of X-linked retinitis pigmentosa (XLRP) caused by mutations in the eye-specific form of the RPGR gene called RPGR open reading frame 15 (RPGR ORF15). Both rods and cones photoreceptors require RPGR ORF15 to function.
<a href="https://meiragtx.com/research-development/pipeline/">Source</a>

Bota-vec (Botaretigene sparoparvovec) (AAV-RPGR)

Retinal dystrophy

AAV-RPE65 is a gene therapy product candidate in which expression of a codon-optimized RPE65 gene is driven by a novel synthetic retinal pigment epithelium (a thin layer of cells at the back of the eye) cell-specific promoter. The RPE65 protein is essential for rod function because it recycles the light sensing machinery in rod photoreceptors. The codon and vector optimization resulted in a gene therapy that is 100 to 1,000 times more potent than the first generation therapy.
We are currently conducting a Phase 1/2 clinical trial of AAV-RPE65 in both adult and pediatric patients with RPE65-deficiency.
The FDA and EMA each granted orphan status to AAV-RPE65 for the treatment of Leber’s Congenital Amaurosis (LCA). The FDA also granted AAV-RPE65 rare pediatric disease designation for the treatment of inherited retinal dystrophy due to biallelic RPE65 mutations
Gene therapy

AAV-RPE65

Achromatopsia

AAV-CNGA3, a gene therapy treatment designed to restore cone &nbsp;function, is delivered to the cone receptors at the back of the eye via &nbsp;subretinal injection. It was designed with a synthetic promoter &nbsp;associated with strong gene expression to account for the larger amount &nbsp;of protein needed to restore cone function in achromatopsia (ACHM) &nbsp;patients with a&nbsp;CNGA3&nbsp;gene&nbsp;mutation.
<a href="https://meiragtx.com/research-development/pipeline/">Source</a>
Gene therapy

AAV-CNGA3

Autosomal recessive congenital ichthyosis

Skin disease/disorder

AAV-CNGB3, a gene therapy treatment designed to restore cone function, is delivered via subretinal injection to the area of the eye where most of the cones in the retina are located.&nbsp;MeiraGTx is currently conducting a Phase 1/2 clinical trial of AAV-CNGB3 in both adult and pediatric patients with achromatopsia (ACHM) due to mutations in the CNGB3 gene.
AAV-CNGB3 was granted Orphan Drug, Rare Pediatric Disease and Fast Track designations by the FDA, and Orphan Drug and PRIME designations by the EMA, for the treatment of ACHM caused by mutations in the CNGB3 gene.
Gene therapy

AAV-CNGB3

Salivary gland

MeiraGTx is developing AAV-AQP1 to treat radiation-induced xerostomia (RIX) by increasing water conduction in the salivary glands damaged by radiation therapy. Our gene therapy product candidate works by introducing a water conducting channel into the remaining epithelial cells of these damaged glands.
Gene therapy

AAV2-hAQP1

Parkinson's disease

The objective of the AAV-GAD trial (<a href="https://www.clinicaltrials.gov/ct2/show/NCT05603312" rel="nofollow" target="_blank">NCT05603312</a>) is to evaluate the safety and tolerability of AAV-mediated delivery of glutamic acid decarboxylase (GAD) gene transfer into the subthalamic nuclei (STN) of participants with Parkinson's disease.
Gene therapy

MeiraGTx

Company Details