MeiraGTx is a clinical-stage gene therapy company developing potentially curative treatments for patients living with serious diseases. In partnership with leading experts around the world, we are developing gene therapies for the treatment of multiple inherited retinal diseases (IRDs). We are currently conducting clinical trials of investigational gene therapies that were developed for the treatment of X-Linked Retinitis Pigmentosa (XLRP) caused by mutations of the RPGR gene, achromatopsia (ACHM) caused by mutations in either the CNGB3 gene or the CNGA3 gene and retinal dystrophy associated with biallelic RPE65 mutations1. Additionally, MeiraGTx manufactured and released for compassionate use under an MHRA Specials License an investigational gene therapy for the treatment of Leber Congenital Amaurosis 4 (LCA4) caused by mutations in the AIPL1 gene2.
We are proud to have Janssen Pharmaceuticals, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson as our development partner for our XLRP and ACHM programs as part of a global collaboration to develop and commercialize gene therapies for the treatment of IRDs. Janssen established its Retinal Disease portfolio in 2018 to develop transformational therapies and platforms that restore and preserve vision. The portfolio expands Johnson & Johnson’s already broad ophthalmic footprint and includes a pipeline of innovative treatments for inherited retinal diseases, as well as more common retinal diseases affecting millions of people worldwide.
For more information about our clinical programs, please visit the MeiraGTx website https://meiragtx.com/patients-families or email patients@meiragtx.com
Investigational Gene Therapies
Treatment of XLRP
AAV-RPGR is an investigational gene therapy treatment designed to treat XLRP caused by mutations in the eye-specific form of the RPGR gene (RPGR-ORF 15) delivered via subretinal injection. Both rod and cone photoreceptors require RPGR-ORF 15 to function. We are currently conducting a Phase 1/2 clinical trial of AAV-RPGR in adult and pediatric patients. For more information about the clinical trial, please visit: https://www.clinicaltrials.gov/ct2/show/NCT03252847
Treatment of Achromatopsia
AAV-CNGB3 is an investigational gene therapy treatment designed to restore cone function in patients with achromatopsia caused by mutations in the CNGB3 gene. AAV-CNGB3 is delivered via subretinal injection to the area of the eye where most of the cones in the retina are located. We are currently conducting a Phase 1/2 clinical trial of AAV-CNGB3. For more information about the clinical trial, please visit: https://www.clinicaltrials.gov/ct2/show/NCT03001310
AAV-CNGA3 is an investigational a gene therapy treatment designed to restore cone function in patients with achromatopsia caused by mutations in the CNGA3 gene. AAV-CNGA3 is delivered via subretinal injection to the area of the eye where most of the cones in the retina are located. It was designed with a synthetic promoter associated with strong gene expression to account for the larger amount of protein needed to restore cone function in patients with a CNGA3 gene mutation.
We are currently conducting a Phase 1/2 clinical trial of AAV-CNGA3. For more information about the clinical trial, please visit: https://clinicaltrials.gov/ct2/show/NCT03758404
Treatment of RPE65-Associated Retinal Dystrophy1
AAV-RPE65 is an investigational gene therapy treatment in which expression of a codon-optimized RPE65 gene is driven by a novel synthetic retinal pigment epithelium cell-specific promoter. We have completed a Phase 1/2 clinical trial and are exploring the pathway forward for the program with regulatory agencies.
Trial details can be found here : https://www.clinicaltrials.gov/ct2/show/NCT02781480
Treatment of Leber Congenital Amaurosis 4 (LCA4)2
AAV-AIPL1 is an investigational gene therapy manufactured by MeiraGTx and released for compassionate use under an MHRA Specials License in the UK. This compassionate use program allows physicians to prescribe a treatment of AAV-AIPL1 for LCA4 patients they deem appropriate. MeiraGTx plays no role in the physician’s treatment decision.
Please contact Patients@meiragtx.com for inquiries.
1-Treatment wholly owned by MeiraGTx and not part of the Janssen collaboration
2-AAV-APIL1 not part of the MeiraGTx/Janssen collaboration
