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Alnylam Pharmaceuticals

Alnylam is an RNAi therapeutics company and Regeneron's partner in a 2019 collaboration on eye and CNS targets. Its link to prion disease is indirect on the public record. ALN-6457, also called ALN-PRNP, carries an Alnylam style code, but Regeneron alone sponsors the trial, the registry names no collaborator, and Alnylam's pipeline page lists no prion program. No prion term was found in SEC filings of either company. So the working inference that the drug came out of this collaboration remains an inference. It should not be read as a confirmed Alnylam development role.

Key facts

  • Regeneron is the only sponsor of the ALN-6457 trial, the record lists no collaborators, and Alnylam is not named in it.
  • The registry gives the other name ALN-PRNP and describes a Phase 1/2 open label study in adults with sporadic CJD or genetic prion disease, with 60 estimated participants and no sites listed.
  • Alnylam's pipeline page has no prion, PRNP or ALN-6457 entry; its neuroscience list includes mivelsiran, ALN-SOD, brezosiran, ALN-5288 and ALN-SNCA.
  • ALN-PNP on the Alnylam pipeline sits under metabolic programs and should not be confused with ALN-PRNP.
  • SEC full text search found no use of the word prion in Alnylam or Regeneron filings, and no filing with the strings ALN-PRNP or ALN-6457; control terms worked.
  • A Europe PMC search found no prion paper with an Alnylam affiliation.
  • The CJD Foundation says Regeneron is developing divalent siRNA therapies against PRNP and does not mention Alnylam.
  • An automated aggregator labels ALN-6457 an siRNA with Regeneron as originator and lists zero matched asset deals; it never names Alnylam.
  • The 2019 deal gave Alnylam USD 400 million upfront and a USD 400 million equity purchase, with up to USD 200 million in early clinical milestones on eye and CNS programs.
  • CNS leadership alternates, the lead party keeps global responsibility, and both sides may opt at lead candidate selection to share costs and profits equally; otherwise the lead party is licensee and pays milestones up to USD 150.0 million per product.
  • The initial research term ended in May 2026, which ends general exclusivity in eye and CNS discovery, although nominated targets stay exclusive if lead candidates are designated within two years.
  • Alnylam describes C16 as a delivery approach for CNS and eye tissue, advanced in the Regeneron collaboration including for its Huntington program.

Timeline

  1. 2019Regeneron and Alnylam announce the eye and CNS RNAi collaboration.
  2. 2026Initial Research Term of the collaboration expires.
  3. 2026ALN-6457 trial record first submitted by Regeneron.
  4. 2026ALN-6457 trial record first posted.
  5. 2026Estimated trial start; the record read on 2026-10-31 still showed not yet recruiting.
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