RP
Retinitis Pigmentosa (RP) is a group of inherited diseases that cause progressive degeneration of the photoreceptor cells in the retina, the light-sensing cells at the back of your eye. It affects approximately 2 million people worldwide and is one of the leading causes of inherited blindness.
RP is not a single disease but a family of related retinal disorders caused by mutations in more than 100 different genes. Despite this genetic diversity, all forms of RP share a common final pathway: the progressive death of photoreceptor cells, particularly rod cells first, then cone cells.
Rod cells are responsible for peripheral and night vision. Cone cells provide central vision and color perception. As rods die first, early RP typically causes difficulty seeing in dim light (night blindness) and loss of peripheral vision.
Over years or decades, as more photoreceptors die, patients develop tunnel vision. Eventually, central vision may also be affected, leading to legal blindness. The rate of progression varies widely between individuals and genetic subtypes.
RP can be inherited in several patterns: autosomal dominant, autosomal recessive, or X-linked. In some cases, there is no family history (simplex RP).
Early Stage
Middle Stage
Advanced Stage
The honest reality is that approved, disease-modifying treatments for RP are extremely limited.
Spark Therapeutics / Roche
Only treats patients with mutations in the RPE65 gene, fewer than 1% of all RP patients (~650 eligible US patients). Costs $850,000 per patient. Commercial uptake has been deeply disappointing, with $20.5M in 2024 revenue (down 59% YoY).
Various
Some studies suggest high-dose vitamin A palmitate may slow rod degeneration in certain patients, but evidence is limited and it does not halt progression.
Various
Helps patients adapt to vision loss but does not treat the underlying disease.
The bottom line for RP patients
For 99% of RP patients, those without the specific RPE65 mutation, there is no approved treatment that modifies the course of the disease. Management focuses on monitoring, adaptive strategies, and protecting remaining vision. This represents one of the most significant unmet needs in ophthalmology.
Rather than targeting a specific gene mutation, a neuroprotective therapy aims to protect photoreceptor cells from the apoptosis (programmed cell death) pathway that all forms of RP converge on, regardless of genetic cause.
About SBC003 in RP
SunRegen is investigating SBC003, an oral compound that induces expression of neuroglobin, a protein that protects neurons from apoptotic signals, via post-transcriptional regulation. In a murine model of RP, oral SBC003 at 50 mg/kg significantly preserved the outer nuclear layer (p<0.001) and photoreceptor nuclei row count compared to vehicle, as confirmed by OCT imaging.
SBC003 is currently in preclinical development. It has not been approved and is not yet available to patients outside of clinical trials.
Foundation Fighting Blindness
Largest private funder of RP research; patient community and trial finder
Retina International
Global network of retinal degeneration patient organizations
ClinicalTrials.gov: RP
Search for open RP clinical trials
American Academy of Ophthalmology
Find a retinal specialist and patient education
Pioneering first-in-class orally bioavailable lipid therapeutics to halt neurodegeneration and restore vision. Clinical-stage. Basel, Switzerland.
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