Publications by authors named "Matthew Tonini"

Article Synopsis
  • TNG908 is a clinical-stage inhibitor targeting PRMT5, utilizing a unique binding mechanism that exploits the loss of the MTAP gene commonly found in various cancers.
  • It specifically inhibits PRMT5 in cancer cells lacking MTAP, which occurs in 10-15% of human cancers and could lead to more effective treatments compared to earlier drugs.
  • Ongoing Phase I/II trials are investigating the effectiveness of TNG908 in patients with MTAP-null tumors, including glioblastoma, suggesting a promising future for this therapy in multiple cancer types, particularly those affecting the brain.
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Article Synopsis
  • PRMT5 inhibitors can target cancer cells with a specific gene deletion by utilizing the buildup of a substrate called MTA, leading to selective cell death.* -
  • TNG908 is a newly discovered and potent inhibitor that binds to the PRMT5·MTA complex, demonstrating a significant ability to kill MTAP-null cells more effectively than normal cells.* -
  • TNG908 shows promise as an oral treatment in mouse models for various tumors, including those in the central nervous system, because it can cross the blood-brain barrier.*
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Mucopolysaccharidosis type IIIA (MPS IIIA) is a neurodegenerative lysosomal storage disorder that results from a deficiency of sulfamidase (N-sulfoglucosamine sulfohydrolase), with consequential accumulation of its substrate, partially degraded heparan sulfate. Conventional doses (e.g.

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