Opus Genetics has received $1.7 million in project-based funding from the Foundation Fighting Blindness (FFB) to support two preclinical inherited retinal disease (IRD)-based programs.
How about a quick refresh on Opus Genetics?
Launched in 2021, the clinical-stage gene therapy company is headquartered in Raleigh, North Carolina, and funded by a venture arm of FFB: the Retinal Degeneration (RD) Fund.
- Note: The RD Fund provides investments in therapeutic companies working to prevent, treat, and cure retinal degenerative diseases.
The company’s focus: Addressing unmet needs found in the IRD space via an adeno-associated virus (AAV)-based gene therapy portfolio positioned to leverage novel orphan drug manufacturing scale and efficiencies.
The targeted indications:
- Leber congenital amaurosis (LCA)
- Vitelliform macular dystrophy
- Retinitis pigmentosa (RP)
- Most common IRD, impacing 1.5 million patients across the globe.
- Mutations in 80 different genes are associated with non-syndromic RP (disease without systemic abnormalities, accounting for 70-80% of all patients)
- Syndromic RP (disease with other non-ocular syndromes) accounts for 20-30% of all patients
- Most common IRD, impacing 1.5 million patients across the globe.
Didn’t the company also acquire new assets last year?
Sort of ... back in December 2022. Two pre-clinical gene therapy candidates, to be exact—from Iveric Bio, Inc.— targeting the treatment of:
Read our coverage here.
Are either of these candidates receiving this funding?
As a matter of fact, yes: The gene therapy targeting RHO-adRP.
RHO-adRP is considered one of the most common IRDs and affects an estimated one in 50,000 individuals (+6,000 in the U.S. alone).
The company received $1 million from FFB’s Translational Research Acceleration Program (TRAP) Award—which accelerates preclinical research for potential IRD and dry age-related macular degeneration (AMD) therapies—to conduct a preclinical safety study on this gene therapy vector.
Give me the details on this study.
The preclinical safety study will be conducted using an “established canine animal model at the University of Pennsylvania School of Veterinary Medicine.”
Its significance: Opus Genetics plans for the study to be the last preclinical trial needed for the gene therapy to move into the clinical trial stage.
And the remaining $700,000 in funding?
That (approximate) amount was granted to the company for project-based and operational funding to advance the preclinical development of a specific novel viral vector for:
- RP due to mutations in the proto-oncogene tyrosine-protein kinase MER (MERTK) gene
What’s the plan for this?
Per the company, Opus Genetics will partner with FFB to launch “IND-enabling studies for a newly-designed (AAV) viral vector to replace mutated MERTK genes in the retinal pigmented epithelial (RPE) cells of the retina.”
To start: The initial $700K in funding will be used for resources to test the vector within an animal model of the disease and conduct early safety assessments in larger animals, Optus Genetics reported.
Why zero in on MERTK mutations?
These types of mutations are associated with severe autosomal recessive retinal dystrophies among the Royal College of Surgeons (RCS) rats—a strain of rats that model for IRDs.
Plus: They’re known to lead to rod-cone dystrophy with early macular atrophy—with RP being the most common retinal phenotype.
And the bigger picture with these preclinical studies?
Per Opus Genetics CEO Ben Yerxa, PhD, the funding for this research will make “a significant impact to help accelerate the development of these two preclinical candidates”—with the potential to advance clinical studies to human trials and identify treatments for IRD patients.