Discovery opens doors to improving diagnostics and developing new therapy for majority of ALS patients

Summary

• HSCI-funded research using human pluripotent stem cell models and tissue from ALS patients found that the protein TDP-43 regulates a gene called Stathmin2 (STMN2).
   
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Biotech company Moderna, co-founded by HSCI scientist Derrick Rossi, is set to bring a new class of treatments to patients.

Summary

• Derrick Rossi’s research into using mRNA as a new kind of therapeutic was initially supported by HSCI seed funding, and gave rise to biotech company Moderna Therapeutics.
• Moderna’s public offering of $600 million is the largest ever of a venture-capital-backed biotechnology company, reflecting the potential of its new technology: mRNA therapeutics
• Moderna’s success in creating therapies that will benefit patients reflects the crucial role of HSCI in enabling early-stage, high-risk research.

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Zebrafish models could help develop precision cancer therapies