The Generative Brain

UMass Chan Medical School (Khvorova lab)

UMass Chan Medical School, home of the Khvorova lab that invented the divalent siRNA scaffold used in the PRiSM drug; it licenses oligonucleotide programs to nonprofit foundations and hosts the RTI Nucleic Acid Chemistry Center.

Key facts

  • Divalent siRNA invented by Anastasia Khvorova (UMass Chan) developed with the Broad team as di-RNA-2439s4: as low as 17 percent residual whole-hemisphere PrP after a single 348 microgram dose and 15 percent with repeat dosing, sustained up to six months.
  • Anastasia Khvorova is professor in the RNA Therapeutics Institute at UMass Chan and founded the RTI Nucleic Acid Chemistry Center, described as the only non-profit center in North America able to synthesize complex RNAs at scale.
  • The prion collaboration began after a 2019 Broad talk; the lab screened Prnp and PRNP sequences, filed patent WO2021173984 and early development was funded by an NINDS IGNITE grant.
  • In January 2026 UMass Chan licensed an allele-specific siRNA for PLN-R14del cardiomyopathy to PLaN Therapeutics, a nonprofit owned by the PLN Foundation, which keeps exclusive product rights.
  • Khvorova received the 2025 Else Kroner Fresenius Prize (2.5 million euros) and said it would fund RNA molecules against toxic brain proteins.

Timeline

  1. 2019PRNP collaboration with the Broad begins.
  2. 2025Else Kroner Fresenius Prize announced.
  3. 2026PLN-R14del siRNA license to a nonprofit.
  4. 2026Divalent siRNA for prion disease published.
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