Researchers focus on regenerative treatments for age-related conditions, including AMD
Scientists have identified several small molecule drugs that selectively expand RPEs to regenerate a functional RPE layer.
(Image credit: AdobeStock/Lorant)
Scientists at Scripps Research and its nonprofit drug development arm, the Calibr-Skaggs Institute for Innovative Medicines, are working to advance regenerative medicines capable of repairing tissues damaged by age-related disease. According to the company, these first-in-class small molecule drugs act on endogenous stem cells in the body to control their fate. They are being developed to treat osteoarthritis, inflammatory bowel disease, lung diseases, heart failure, macular degeneration, and more.1
In the press release1, Peter Schultz, PhD, President and CEO of Scripps Research shared why age-related conditions are a focus, saying, “Current therapies for these chronic conditions slow disease progression or treat the symptoms, but don’t fully stop or reverse the resulting damage. In contrast, the regenerative medicine pipeline at Scripps Research represents a new frontier in healthy aging, with disease-modifying therapies that repair damaged tissues and thereby restore function in patients.”
Among the conditions the company researching and developing potential treatments for is repairing the retina in age-related macular degeneration (AMD). Scientists have identified several small molecule drugs that selectively expand RPEs to regenerate a functional RPE layer, which are being evaluated in laboratory and animal studies to determine which of several mechanisms would be most amenable as an AMD therapy. If successful, the envisioned therapy would be a one-time injection of a long-acting drug to replenish the RPE layer in people with early-stage AMD.1
Michael Bollong, PhD, associate chemistry professor and the Early Career Endowed Roon Chair for Cardiovascular Research at Scripps Research, who led the discovery of several of the regenerative programs, along with Schultz, who is also a professor of Chemistry, notes that the Scripps Research/Calibr-Skaggs team’s approach focuses on the development of small molecule drugs designed to be more accessible and easily administered than stem cell therapies.1
The idea being that conventional complexity, poor efficacy, and high costs have stalled cell. Small molecules may provide a path forward for more effective regenerative medicines. “Our ultimate goal,” says Bollong, “is to expand to other organs to repair disease-related injury or even to rejuvenate healthy tissues as we age.”1
Schultz adds, “If we are able to show these therapies work in a disease setting, the next question will be to ask if they reverse age-related damage in otherwise healthy aging adults.”1
