Evidence map›Paper›PMID 41867745›Full record

ArticlebioRxiv : the preprint server for biology2026

Divalent siRNA for prion disease.

Juliana E Gentile, Taylor L Corridon, Fiona E Serack, Dimas Echeverria, Zachary C Kennedy, Corrie L Gallant-Behm, Matthew R Hassler, Garth A Kinberger, Margaret N Kelemen, Nikita G Kamath and 18 more

Abstract readPreprint
In one paragraph

Article in bioRxiv : the preprint server for biology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

28 authors.

Juliana E GentileProgram in Brain Health, Broad Institute of MIT and Harvard, Cambridge, MA, 02142, USA.
Taylor L CorridonProgram in Brain Health, Broad Institute of MIT and Harvard, Cambridge, MA, 02142, USA.ORCID 0009-0002-4167-8291
Fiona E SerackProgram in Brain Health, Broad Institute of MIT and Harvard, Cambridge, MA, 02142, USA.ORCID 0000-0001-7377-219X
Dimas EcheverriaRNA Therapeutics Institute, UMass Chan Medical School, Worcester, MA, 01655, USA.
Zachary C KennedyRNA Therapeutics Institute, UMass Chan Medical School, Worcester, MA, 01655, USA.
Corrie L Gallant-BehmAtalanta Therapeutics, Boston, MA, 02210, USA.
Matthew R HasslerAtalanta Therapeutics, Boston, MA, 02210, USA.
Garth A KinbergerAtalanta Therapeutics, Boston, MA, 02210, USA.
Margaret N KelemenProgram in Brain Health, Broad Institute of MIT and Harvard, Cambridge, MA, 02142, USA.
Nikita G KamathProgram in Brain Health, Broad Institute of MIT and Harvard, Cambridge, MA, 02142, USA.
Yuan LianProgram in Brain Health, Broad Institute of MIT and Harvard, Cambridge, MA, 02142, USA.ORCID 0009-0001-9565-2292
Katherine Y GrossRNA Therapeutics Institute, UMass Chan Medical School, Worcester, MA, 01655, USA.
Rachael MillerRNA Therapeutics Institute, UMass Chan Medical School, Worcester, MA, 01655, USA.ORCID 0000-0002-4594-0640
Kendrick DeSouza-LenzComparative Medicine, Broad Institute of MIT and Harvard, Cambridge, MA, 02142, USA.
Michael HowardComparative Medicine, Broad Institute of MIT and Harvard, Cambridge, MA, 02142, USA.
Kenia GuzmanComparative Medicine, Broad Institute of MIT and Harvard, Cambridge, MA, 02142, USA.
Nathan ChanComparative Medicine, Broad Institute of MIT and Harvard, Cambridge, MA, 02142, USA.
Vanessa LaversenneProgram in Brain Health, Broad Institute of MIT and Harvard, Cambridge, MA, 02142, USA.
Daniel T CurtisAtalanta Therapeutics, Boston, MA, 02210, USA.
Kevin FettesFTS Pharma Consulting LLC, Medfield, MA, 02052, USA.
Marc LemaitreML Consult LLC.
Aimee L JacksonAtalanta Therapeutics, Boston, MA, 02210, USA.
Ken YamadaRNA Therapeutics Institute, UMass Chan Medical School, Worcester, MA, 01655, USA.
Julia F AltermanRNA Therapeutics Institute, UMass Chan Medical School, Worcester, MA, 01655, USA.
Alissa A CoffeyProgram in Brain Health, Broad Institute of MIT and Harvard, Cambridge, MA, 02142, USA.
Eric Vallabh MinikelProgram in Brain Health, Broad Institute of MIT and Harvard, Cambridge, MA, 02142, USA.ORCID 0000-0003-2206-1608
Anastasia KhvorovaRNA Therapeutics Institute, UMass Chan Medical School, Worcester, MA, 01655, USA.
Sonia M VallabhProgram in Brain Health, Broad Institute of MIT and Harvard, Cambridge, MA, 02142, USA.ORCID 0000-0003-3824-2702

Funding

Regulatory Consulting to Support Advancement of a Divalent siRNA for Prion DiseaseU01NS132994 · NINDS · BROAD INSTITUTE, INC. · PI MINIKEL, ERIC VALLABH · 2023 to 2025
$1.7M
Development of Prion Protein-Lowering Divalent siRNA Therapy for Prion DiseaseR33NS119717 · NINDS · BROAD INSTITUTE, INC. · PI MINIKEL, ERIC VALLABH, VALLABH, SONIA MINIKEL · 2022 to 2023
$793k
Development of prion protein-lowering divalent siRNA therapy for prion diseaseR61NS119717 · NINDS · BROAD INSTITUTE, INC. · PI MINIKEL, ERIC VALLABH, VALLABH, SONIA MINIKEL · 2021 to 2021
$400k
NINDS NIH HHS R33 NS119717NINDS NIH HHS R61 NS119717NINDS NIH HHS U01 NS132994
6 · The paper itself

Abstract

Prion protein (PrP) lowering is effective in animal models of prion disease and is being tested clinically in prion disease patients, but there remains a need for more potent PrP-lowering drug candidates. Inspired by the reported potency and duration of action of divalent short interfering RNA (siRNA), a new oligonucleotide drug modality for the central nervous system, we sought to discover and develop a new PrP-lowering drug candidate. Herein we identify a mouse

Identifiers

PMID41867745
PMCPMC13001464

What OpenQuestion holds

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.