Scripps Florida Awarded $2.5 Million to Advance Development of RNA-Based Therapeutics

Researchers are developing quick ways to convert information into lead drugs by using several novel and transformative technologies they've developed

Written byThe Scripps Research Institute
| 2 min read
Register for free to listen to this article
Listen with Speechify
0:00
2:00

JUPITER, FL – Scientists from the Florida campus of The Scripps Research Institute (TSRI) have been awarded a $2.5 million grant from the National Institute of General Medical Sciences of the National Institutes of Health to design precision drug candidates that target disease-associated RNAs.

Matthew DisneyMatthew Disney is a professor at the Florida campus of The Scripps Research Institute.Photo courtesy of The Scripps Research InstituteMatthew Disney, a TSRI professor, will be the principal investigator for the new four-year study.

“A major goal of genome sequencing efforts is to develop drug targets that could enable the development of patient-specific therapies,” Disney said. “In this project, we are developing quick ways to convert this information into lead drugs by using several novel and transformative technologies we developed. The new grant will keep us moving forward and allow us to tackle many new challenges in the area of precision medicine.”

Lab manager academy logo

Lab Management Certificate

The Lab Management certificate is more than training—it’s a professional advantage.

Gain critical skills and IACET-approved CEUs that make a measurable difference.

While to date large macromolecules have generally been used to target RNA, small molecules can pass through the blood-brain barrier, a critical factor in the treatment of neurological diseases and cancer that affects the brain.

The Disney lab’s untraditional approach has been broadly applicable in developing precision probes to target disease-causing RNA repeat expansions, which cause more than 30 diseases—including Huntington’s, which has no cure—affecting millions worldwide.

Disney’s successful efforts in identifying various drug-like small molecules that bind to RNA is the result of his lab’s broad, bottom-up, computational approach known as Inforna, which can deep mine information against such genome sequences and cellular RNAs.  

Related Article: Computational Changes in Next-Generation Sequencing 

The new grant will enable Disney and his team to further investigate the manipulation of microRNAs. Discovered only in the 1990s, microRNAs are short molecules that work within virtually all animal and plant cells. Typically, each one functions as a “dimmer switch” for one or more genes, binding to the transcripts of those genes and effectively keeping them from being translated into proteins.

Interested in life sciences?

Subscribe to our free Life Sciences Newsletter.

Is the form not loading? If you use an ad blocker or browser privacy features, try turning them off and refresh the page.

By subscribing, you agree to receive email related to Lab Manager content and products. You may unsubscribe at any time.

The new grant will also allow Disney to expand development of small molecules that target two miRNAs with a single small molecule and to study the cellular consequences targeting multiple disease pathways using what he calls a “designer poly-pharmacy approach.”

The number of the grant is 2R01GM097455-05.

Loading Next Article...
Loading Next Article...

CURRENT ISSUE - May/June 2025

The Benefits, Business Case, And Planning Strategies Behind Lab Digitalization

Joining Processes And Software For a Streamlined, Quality-First Laboratory

Lab Manager May/June 2025 Cover Image