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OPGx-RDH12 · 1 trial · 2 indications
| Arm | Type | Description |
|---|---|---|
| OPGx-RDH12 | EXPERIMENTAL | Administration of OPGx-RDH12 will occur via a cannula into the subretinal space, using the standard technique for delivery of other adeno-associated virus (AAV) therapies including Luxturna®. A dose of 1E11 vg/eye will be injected sub-retinally one time into the treatment eye. The treatment eye will be the eye with the worst visual function (as determined by visual acuity, full-field sensitivity testing \[FST\] and kinetic perimetry) or the non-dominant eye in cases of bilateral symmetric disease. |
| Name | Type | Description |
|---|---|---|
| OPGx-RDH12 | DRUG | Experimental gene therapy |
Inclusion Criteria: * Age ≥18 years (adult participants) or 12-17 years (adolescent participants) at the time of consent/assent. * Provide written informed consent and/or assent prior to any study procedures. * Willing to adhere to the clinical protocol and follow directions of the Investigator reg...
OPGx-RDH12 is an investigational small molecule being developed for Leber Congenital Amaurosis (LCA), a rare inherited retinal disease. It is designed to address retinal degeneration associated with LCA. The drug is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
OPGx-RDH12 targets the RDH12 gene, which is implicated in Leber Congenital Amaurosis. By targeting this gene, the therapy aims to address the underlying genetic cause of the retinal degeneration seen in LCA patients. The drug is a small molecule designed to modulate this genetic target.
OPGx-RDH12 is being developed by Opus Genetics, Inc., a biopharmaceutical company. The company is conducting clinical trials for this investigational therapy. Opus Genetics is focused on advancing treatments for rare genetic diseases, including inherited retinal disorders like Leber Congenital Amaurosis.
OPGx-RDH12 is currently in Phase 1 clinical development. It is an investigational drug and has not yet received FDA approval. The Phase 1 trial is designed to evaluate the safety and tolerability of the drug in patients with Leber Congenital Amaurosis.
OPGx-RDH12 is being studied in a Phase 1 clinical trial with the identifier NCT07681778. The trial is titled 'Safety and Tolerability of Subretinal OPGx-RDH12-1001 for LCA-Associated Inherited Retinal Degeneration (LCA-IRD)' and is expected to enroll 10 participants in the United States. The trial is not yet recruiting.