A novel therapeutic composition to restore function in Retinitis Pigmentosa Type 10
Key Advantages
- Selective inhibition of IMPDH1.
- Validated in a relevant new mouse model for RP10.
- Therapeutic potential to delay or prevent photoreceptor degeneration.
- Repurposing potential using clinically validated drugs.
Goal
Seeking a license agreement, but other collaborations may be considered.
Intellectual Property
EP filed – Priority date: April 17th 2026
Reference
UBTT0548
Contact
Marc Salazar
Email: msalazar@fbg.ub.edu
A novel therapeutic composition to restore function in Retinitis Pigmentosa Type 10
Executive summary
A research group, with comprehensive research experience in retinal biochemistry and physiology has developed a new approach for the treatment of Retinitis Pigmentosa 10 (RP10) by employing AMPK activators to inhibit IMPDH1 activity.
Description
This technology provides a novel therapeutic strategy based on AMPK-activating compounds to inhibit IMPDH1 activity in the retina in vivo. This approach restores purine nucleotide balance and prevents photoreceptor cell death and retinal degeneration, directly addressing the metabolic root cause of degeneration.
In addition, a first-in-class genetically engineered mouse model carrying a clinically relevant IMPDH1 mutation has been developed. This model faithfully replicates RP10 disease progression and represents a valuable tool for preclinical validation and drug development in retinal degeneration.
Why does it matter?
Inherited retinal diseases (IRDs) affect approximately 1 in 2.000-3.000 individuals and are a leading cause of irreversible blindness in children and working-age adults.
Retinitis Pigmentosa (RP) is the most common of IRD, affecting ~1 in 4.000 individuals and causing progressive degeneration of photoreceptors, eventually leading to blindness.
RP10, caused by mutations in IMPDH1, represents 0.2–2.5% of RP cases and represents a rapidly progressive form with onset in childhood and severe vision loss by early adulthood. Currently, no approved disease-modifying therapy exists.
In Spain, more than 6,000 patients have been clinically diagnosed with IRDs, with nearly 4.000 genetically confirmed cases, which highlights the critical unmet need for targeted therapies.
Current stage of development
- Identify the optimal drug or drug combination and dosing regimen.
- Confirm robust efficacy and target engagement in vivo.
- Assess Safety & Tolerability.
- Position lead candidate for regulatory consultation and development.