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View Pipeline at MolAIcule.com

Lead Program — Retinitis Pigmentosa (RP), Extendable to AMD

A genotype-agnostic, topical small-molecule therapeutic candidate for Retinitis Pigmentosa (RP), with expansion potential to Age-related Macular Degeneration (AMD). Discovered and optimized using the Medicine.AI platform.

• Primary Indication: Retinitis Pigmentosa (RP) — progressive inherited retinal degeneration
• Expansion Opportunity: Age-related Macular Degeneration (AMD)
• Modality: Topical small molecule (eye drop delivery)
• Stage: Preclinical — lead optimization & formulation
• Mechanism: Targets common degeneration pathways (photoreceptor survival/neuroinflammation); genotype-agnostic approach
• Key Advantages: Non-invasive delivery, scalable manufacturing, designed for early-to-mid stage patients with remaining photoreceptors

Preclinical data demonstrate structural protection (ONL/OCT) and functional improvement (ERG) in retinal degeneration models. This candidate addresses a significant unmet need: no scalable pharmacologic option exists for RP today, and AMD represents a large adjacent market opportunity.