Therapeutic For Dry Age-Related Macular Degeneration and Stargardt Disease

Technology Description

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. 

Advantages

• 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 identified therapeutic 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 therapeutic than what is 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

Application

• 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 thereapeutic 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

Data Availability

Under CDA

Patent Status

Patent Pending

Other Information

Published application: WO 2023/220136