Three research teams have been selected as recipients of the 2024 Blavatnik Therapeutics Challenge Awards, an annual awards program that aims to progress promising discovery science at Harvard Medical School toward the creation of new medicines that could be life-changing for patients.
- By SUZANNE DAY
Open to faculty at the School and its 15 affiliated hospitals, the awards provide critical funding to researchers who've made fundamental discoveries with the potential for becoming powerful clinical interventions.
"We're living in a prolific time of scientific discovery, and it is our imperative to accelerate the pace of therapeutics research within the HMS community and translate more of our discoveries into new medicines," said George Q. Daley, dean of HMS.
"The journey from molecule to medicine is long and often requires coordination across several institutions and organizations," he added. "This critical funding helps us toward our goal."
Now in its fifth and concluding year, the Blavatnik Therapeutics Challenge Awards have supported 20 research projects. Of the 17 projects awarded in the first 4 years of the program, four have already progressed to either the founding of a new company or licensing of the technology as part of a start-up. Two additional projects may enter clinical trials within the next year.
The 2024 award recipients are:
Addressing Fragile X syndrome through genetics
Principal investigator Jeannie Lee, HMS professor of genetics and pathology at Massachusetts General Hospital
The challenge: The most common inherited cause of autism and intellectual disabilities worldwide, Fragile X syndrome is a genetic disorder that occurs when a single gene located on the X chromosome that is necessary for brain development shuts down. Children with Fragile X syndrome may have speech delays, learning disabilities, or intellectual challenges.
A possible solution: Lee is pursuing a novel approach to treating Fragile X syndrome by reactivating the dormant gene that underlies the condition. As Fragile X syndrome is mainly caused by an expansion of the repeating nucleotide sequence cytosine-guanine-guanine (CGG), Lee's lab aims to reduce the abnormal CGG repeats. This offers a new strategy for mitigating the syndrome's neurological impacts.
