Zoledronic Acid As a Therapeutic For Dry Age-Related Macular Degeneration and Stargardt Disease
Tech ID: 32906 / UC Case 2022-156-0
The invention is a therapeutic intended to prevent the onset and progression of age-related macular degeneration (MD) as well as Stargardt disease-related vision loss.
UCSF/University of Wisconsin researchers have identified that abnormal activation of acid sphingomyelinase (ASM) in the retinal pigment epithelium (RPE) serves as a pathological trigger in dry age-related MD and Stargardt’s disease. Activation of ASM results in increased ceramide, which makes the RPE (the tissue that is responsible for nourishment and support of light-sensing photoreceptors) susceptible to damage. The RPE is the primary site of damage for both Stargardt inherited and age-related MD.
- Potential preventative treatment for age-related MD and Stargardt disease
- ~ 35 million people worldwide suffer from age-related MD
- Stargardt disease affects ~1 in 8,000 children
- Treatment with bisphosphonate zoledronic acid (the only known structural inhibitor of ASM) can prevent ceramide accumulation and preserve the RPE
- In vivo efficacy in mouse models of MD is observed with a 2,000-fold lower dose than that used for functional ASM inhibitors and 15-fold lower dose of ZA than that currently used to treat osteoporosis.
- Currently, there are no approved therapies for dry age-related MD (which is characterized by focal loss of the RPE) or Stargardt disease
- Therapy to prevent
- age-related MD onset or progression
- vision loss due to Stargardt disease
- Potential therapy for any condition associated with increased ceramide.
- Excess ceramide has been implicated in many diseases, including Alzheimer’s and Parkinson’s disease.
Stage of Development
Target validation and proof of concept translational studies.
Treatment with an ultra-low dose of Zoledronic acid has been shown to prevent ceramide accumulation while preserving RPE health and function in cell-based models of AMD and in vivo in the Abca4-/- mouse model of Stargardt inherited MD
Looking for Partners
To further develop the technology