Opus Genetics Announces FDA Clearance of IND Application for OPGx-RDH12 Gene Therapy for Inherited Childhood Blindness
Key Highlights
- ➤FDA cleared IND for OPGx-RDH12 in RDH12-associated childhood blindness
- ➤Opus plans to initiate a Phase 1/2 trial this quarter
- ➤Global RDH12 Alliance committed up to $1.6 million toward development
- ➤RDH12-LCA affects an estimated 30,900 people globally
- ➤Opus advances its third gene therapy program into clinical development
Expert Statements
George Magrath, Chief Executive Officer of Opus Genetics
“We are pleased to advance our third gene therapy pipeline program into clinical development and plan to initiate a Phase 1/2 trial this quarter.”
George Magrath, Chief Executive Officer of Opus Genetics
“RDH12-LCA causes progressive vision loss beginning at a very young age, underscoring the urgency of developing treatments that may preserve vision before further deterioration occurs.”
George Magrath, Chief Executive Officer of Opus Genetics
“Receiving IND clearance for OPGx-RDH12 represents an important step toward bringing a potentially transformative gene therapy to children and families affected by this devastating disease.”
George Magrath, Chief Executive Officer of Opus Genetics
“We are grateful for the collaboration and commitment of our partners at the Global RDH12 Alliance, which has been instrumental in advancing this program into the clinic.”
Mathew Pletcher, Board member of the RDH12 Fund for Sight
“Bringing a treatment to the members of the RDH12-LCA community has been the stated goal of The RDH12 Fund for Sight since its founding more than a decade ago”
Mathew Pletcher, Board member of the RDH12 Fund for Sight
“From the foundational work, funded by patient dollars, in the lab of Dr. Jean Bennett through today’s announcement, the RDH12-LCA community has been collaborating with partners to ensure the delivery a potentially vision-saving therapy to the clinic.”
Silvia Cerolini, Chief Executive Officer of Eyes on the Future
“This is what true co-development looks like.”
Silvia Cerolini, Chief Executive Officer of Eyes on the Future
“Our RDH12 patient organizations and Opus have pooled resources, capabilities and risk to bring this rare gene therapy into the clinic, combining venture philanthropy, scientific innovation and a patient-driven sense of urgency.”
Silvia Cerolini, Chief Executive Officer of Eyes on the Future
“We have been working toward this moment for many years, and we are grateful to Opus for being a true partner.”
Silvia Cerolini, Chief Executive Officer of Eyes on the Future
“We believe this model can become a blueprint for bringing gene therapies to rare disease communities, and one that other patient organizations can follow.”
FDA clearance enables clinical development of OPGx-RDH12 for RDH12-associated Leber congenital amaurosis, a rare inherited retinal disease that causes progressive vision loss beginning in early childhood
Strategic collaboration with the Global RDH12 Alliance supports the development of a potential treatment for this disease with high unmet need
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