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NIHNATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE

Therapeutic Editing to Lower PrP in Prion Disease

Active
Institution
BROAD INSTITUTE, INC., MA
Principal investigator
Sonia Minikel Vallabh
Funding
$1.0M
Duration
2023–2028
Open portfolio

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Abstract

PROJECT SUMMARY The overarching goal of the parent grant is to file an Investigational New Drug (IND) application with FDA to enable a first in human trial of PRNP-AAV-CHARM, a PrP-lowering epigenetic editor therapeutic, in prion disease. The goal of this administrative supplement is to provide funding to produce clinical-grade drug product at a scale that will support our first-in-human clinical trial in N=10 prion disease patients. This clinical study will very likely be the first time either CHARM or the BI-hTfRv2 viral vector are tested in humans. If this human proof-of-concept is successful, both technologies stand to be of use against a wide range of other CNS diseases.

Research team

  • Benjamin E DevermanPI
  • Sonia Minikel VallabhPI

LOADING MOONBASE SCORE

NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKEU19Special Emphasis Panel[ZRG1(70)-R]prioneditorviral vectorind applicationprpdrug indparent grantsummary overarching

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