Drug Target Review
JUNE 9, 2025
Alltrna’s unique approach focuses on addressing genetic diseases at a fundamental level, shifting the paradigm from a one-disease, one-therapy model to a mutation-specific strategy that could transform the treatment of hundreds of rare diseases. This will mark a critical step in validating the potential of engineered tRNA therapeutics.
Broad Institute
MARCH 11, 2025
The tRCC Project aims to address these challenges, by partnering directly with patients, caregivers, and advocates to deepen understanding of this rare disease and generate new insights that could lead to new treatments. To ensure participant privacy and security, all data will be de-identified before being shared with researchers. "By
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Broad Institute
MAY 21, 2025
Gaining access to a variety of cell types is simply put a game-changer in understanding the brain and developing therapies for human neurological disorders, said Gordon Fishell , an institute member at the Broad Institute, professor of neurobiology at Harvard Medical School, and a senior author of one of the studies.
Broad Institute
OCTOBER 30, 2024
The results pave the way for future single-cell or spatially resolved studies that look at how cells surrounding a metastatic tumor impact its progression or treatment outcomes. Eventually, we hope that these methods can help inform the development of drugs or even guide patients’ individual treatments.”
Broad Institute
AUGUST 6, 2025
Choudhary is an assistant professor of medicine at Harvard Medical School The implications of this advance are wide-ranging. CRISPR-Cas9, often likened to “molecular scissors,” allows scientists to cut DNA at targeted sites to snip, repair, or replace genes.
Broad Institute
MAY 19, 2025
The findings could shed light on the biological basis of these conditions and help scientists develop better diagnostics and treatments. Eating disorders are estimated to affect nearly 30 million Americans, although most arent receiving treatment. The team has already recruited almost 3,000 participants from all 50 states.
The Pharma Data
JUNE 12, 2025
The results come from a open-label, proof-of-concept, Phase 2 study (NCT04520451) and highlight rilzabrutinib’s potential as a disease-changing treatment option for a condition that currently has limited and non-specific treatment options and involves substantial patient suffering due to its chronic and progressive course.
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