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Scientists create next generation of tools in battle against brain disease

Broad Institute

Scientists create next generation of tools in battle against brain disease By Corie Lok May 21, 2025 Breadcrumb Home Scientists create next generation of tools in battle against brain disease The findings contained in eight studies could lead to targeted gene therapies for brain disorders. Adapted from an Allen Institute press release.

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Count Me In launches new patient-partnered research project for translocation renal cell carcinoma

Broad Institute

"Translocation renal cell carcinoma (tRCC) is a rare and aggressive form of kidney cancer that poses significant treatment challenges, said Srinivas Viswanathan, a physician at Dana-Farber Cancer Institute and an assistant professor of medicine at Harvard Medical School.

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New tRNA tech aims to rewrite rare disease treatment

Drug Target Review

“This experience underscored the gaps in rare disease innovation, motivating me to lead Alltrna, a company pioneering engineered tRNA therapies to address a broad range of genetic diseases in a profound way unlike any other genetic modality.”

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The biotech mapping thousands of hidden therapeutic clues

Drug Target Review

By mapping known genetic mutations to these novel proteins, ProFound can identify those with clear associations to disease – significantly increasing the probability that targeting them could lead to breakthrough therapies. From theory to therapy This approach is already delivering promising insights across key therapeutic areas. .

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Oncology advances through the lens of women in STEM

Drug Target Review

Getting into medical school was very competitive with thousands of candidates vying for a handful of positions, but I was fortunate enough to succeed. My inclination toward biological sciences led me to medical school to pursue a future in STEM. I opted for medicine due to my love for biological sciences.

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Alnylam Promotes Pushkal Garg to Chief R&D Officer

The Pharma Data

Dr. Garg expressed a bold vision for the future of RNAi therapy, underscoring Alnylam’s goal of delivering gene-silencing drugs to every major tissue in the body. By bridging the gap between discovery and clinical translation, Alnylam is strengthening its ability to deliver breakthrough therapies more efficiently and with greater precision.

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Rilzabrutinib Cuts Flares in IgG4-Related Disease; Fast Track Granted in US

The Pharma Data

John Stone, MD, MPH , Professor of Medicine at Harvard Medical School, the Edward A. This design was meant to enable clinician-researchers to gauge the ability of rilzabrutinib to control disease activity after the withdrawal of standard therapy.