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To strengthen Opus Genetics\u2019 gene therapy program focused on RDH12 gene mutations, the company announced its partnership with Global RDH12 Alliance.1 According to a press release, the Alliance serves as a collaborative platform that unites various inherited retinal disease (IRD) advocacy groups such as \u201cRDH12 Fund for Sight\u201d in the US and \u201cEyes on the Future\u201d in the UK.1<\/p>\n
This partnership allows for the continual development of OPGx-RDH12, Opus\u2019 gene therapy program targeting the RDH12 gene mutation for the potential treatment of Leber congenital amaurosis (RDG12-LCA).1 To deliver a functional copy of the RDH12 gene directly to the retina\u2019s photoreceptors, OPGx-RDH12 uses an adeno-associated virus (AAV) vector.1<\/p>\n
\u201cPreclinical studies in cell and mouse models have shown restoration of RDH12 activity and functional improvements,\u201d Jean Bennett, MD, PhD, Opus Genetics scientific advisor and Board of Directors member, said in the press release.<\/p>\n
Through the partnership, which includes a risk-sharing structure and performance-based milestones, the Alliance will provide up to $1.6 million toward the development of Opus\u2019 OPGx-RDH12 program.1 The companies’ goal is to file an investigational new drug (IND) application with the US Food and Drug Administration (FDA) by late 2025.<\/p>\n
\u201cSince founding the RDH12 Fund for Sight more than a decade ago, our goal has always been to bring a treatment to the RDH12-LCA community,\u201d Mathew Pletcher, PhD, board member of the RDH12 Fund for Sight, said in the release. Pletcher is also the parent of an adult child living with the condition.<\/p>\n
RDH12-LCA affects several thousand people globally. Mutations of the RDH12 gene impair protein function in the retina, which leads to early visual decline by age 2 and rapid progression during the second decade of life.<\/p>\n
\u201cThis partnership represents a significant step forward. By combining our patient community\u2019s unique, first-hand perspectives on RDH12-LCA and resources with Opus\u2019 gene therapy expertise, we can accelerate the transition of this promising therapy out of the laboratory and into the clinic,\u201d Pletcher said.1<\/p>\n
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