About gene therapy
At Opus Genetics, our mission begins and ends with you—the patients and families living with inherited retinal diseases (IRDs). We believe sight is worth fighting for, and everyone deserves the chance to see what makes life beautiful. Together with scientists, doctors, and most importantly, you, we are developing gene therapies that have the potential to transform lives.
Maci, NMNAT1 patient with Mom Jenna
Braydon, RHD12 Patient with Mom Kelly
Why gene therapy
IRDs are caused by genetic mutations that prevent the production or proper function of proteins essential for vision. Gene therapy works by delivering a healthy copy of the affected gene to retinal cells, aiming to restore function and slow or stop disease progression.
Why the eye is an ideal target for gene therapy:
- Small dose required
- Ability to precisely place the dose in the subretinal space
- Built-in defense against inflammation
- Ability to monitor safety and efficacy with non-invasive imaging and functional tests
We’re thrilled to be a part of the launch of Opus Genetics to establish a patient-first priority and build capabilities to bring life-altering treatments for the people who need them most.
– Paul Manning, Manning Family Foundation
Our programs
OPGx-LCA5
For Leber congenital amaurosis type 5 (LCA5).
OPGx-BEST1
For Best Vitelliform Macular Dystrophy (BVMD) and Autosomal Recessive Bestrophinopathy (ARB).
Preclinical Pipeline
We are also developing therapies for additional IRDs, including RDH12, MERTK, RHO, CNGB1, and NMNAT1
Patient advocacy & community
Opus Genetics was founded in partnership with the Foundation Fighting Blindness. We continue to partner with advocacy groups including, Hope in Focus, Eyes on the Future, RDH12 Fund for Sight, and Foundation Fighting Blindness.
Interested in sharing your story or partnering together?
Contact usPatient Resources
We understand there is great interest within the patient community to advance the science, and we are committed to developing transformative retinal gene therapy treatments for patients.
Opus’ Expanded Access (EAP) policy applies to selected programs and sets forth the general guidelines that apply to ensure that requests for access to our investigational medicines are processed in accordance with applicable legal, medical, and regulatory requirements, as well as in alignment with Opus’ policies and procedures.
Patients and families can find more details on Opus’ EAP program at the Reagan-Udall’s Expanded Access Patient Navigator (Opus Genetics, Inc. | Expanded Access Navigator) and Clinicaltrials.gov (Study Details | Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) | ClinicalTrials.gov).