Gene therapy may be “one shot stop” for rare bone disease

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by injection three to six times each week.

HPP—also known as soft bone disease—is a rare, inherited disorder that causes abnormal development of bones and premature loss of teeth. HPP ranges in severity. Milder cases put affected adults at greater risk of breaking bones. Severe forms of the condition cause life-threatening disease in approximately one per 100,000 live births.

In a paper published on January 12, 2025, in the Journal of Bone and Mineral Research, José Luis Millán, PhD, and collaborators added additional weight to prior preclinical evidence of the safety and effectiveness of a gene therapy for HPP.

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Make a big impact with a future gift

Generosity comes in many forms, and it’s often the best way for you to support important causes that matter most to you. When you give to Sanford Burnham Prebys, you help us make a difference. We will work with you to find a charitable plan that is best for you and your family while providing vital support to our mission.

If you let us know about your intended bequest or other planned gift, we will be able to thank you and recognize you as a member of our Legacy Circle, which honors those who have made a planned gift to Sanford Burnham Prebys.

For more information, please contact Sandy Liarakos, vice president of Philanthropy, at sliarakos@sbpdiscovery.org.

Learn more about our Legacy Circle »

Curebound awards two grants to Sanford Burnham Prebys scientists

Curebound recently announced the awarding of 17 grants in December 2024 for a total of $8.25 million in funding to advance cancer research in 2024. The San Diego–based philanthropic organization has awarded $43 million in cancer research to date.

Two new grants will support cancer research conducted by scientists at Sanford Burnham Prebys. Since 2014, 32 Curebound grants have supported projects that included scientists at the Institute.

Brooke Emerling, PhD, and her collaborators will optimize compounds that break down lipid enzymes known to affect the growth of cancers with a common mutation. The group’s goal is to develop cancer drugs that are strong candidates for future clinical trials.

Michael Jackson, PhD, and Changlu Liu, PhD, will work with Pandurangan Vijayanand, MD, PhD, at the La Jolla Institute for Immunology to identify agents that boost tumor immune responses. The research team’s work has the potential to identify a new class of immunotherapy drugs for patients with lung cancer.

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Theo Tzaridis named 2024 recipient of Eric Dudl Endowed Scholarship

Theo Tzaridis, MD, was named the 2024 recipient of The Eric Dudl Endowed Scholarship at Sanford Burnham Prebys.

He officially joined Sanford Burnham Prebys on January 6, 2025.

The scholarship fund was established at the Institute to remember Eric Dudl, a postdoctoral researcher whose life was tragically cut short by cancer at the age of 33. Since 2007, 17 postdoctoral scientists have received support for their research from the endowed scholarship fund.

Tzaridis is a postdoctoral fellow in the lab of Peter Adams, PhD. Tzaridis studies ways to enhance immunotherapy for diffuse intrinsic pontine glioma, the deadliest brain tumor in children.

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San Diego researchers face big losses if court allows Trump funding cuts

David Brenner, MD, president and CEO of Sanford Burnham Prebys, was interviewed about cuts to indirect cost payments at the National Institutes of Health. He described indirect cost reimbursements as “critical and unavoidable expenses,” and noted that the 15% cap is “arbitrary and ill conceived, with immediate and long-term repercussions.”

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We invite you to read the annual Impact Report

with highlights from 2024. It showcases significant progress and achievements and pays tribute to our faculty, staff, and, most especially, friends like you whose support helps turn ideas and concepts into realities and gives substance to our mission.

Our deepest appreciation for your partnership. Thanks to you, we can continue to take on the toughest health challenges and find new ways

to treat debilitating diseases.

Through philanthropy we translate science

into health.

Click here to read the report.