Escape-Resistant Oligonucleotide Therapy (ONT) for Cytomegalovirus (CMV) Disease in Hematopoietic Stem-Cell and Solid-Organ Transplant Patients

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Grant Award Details

Grant Number:
TRAN1-14062
Investigator(s):
Institution:
Type:
PI

Disease Focus:
Award Value:
$3,977,180
Status:
Active

Grant Application Details

Application Title:

Escape-Resistant Oligonucleotide Therapy (ONT) for Cytomegalovirus (CMV) Disease in Hematopoietic Stem-Cell and Solid-Organ Transplant Patients

Public Abstract:
Translational Candidate

Cytomegalovirus antiviral FD-86: DNA Oligonucleotide Therapy

Area of Impact

HSCT transplant rejection and childhood cognitive and hearing impairment caused by Cytomegalovirus

Mechanism of Action

The candidate oligonucleotide therapy, FD-86 disrupts viral IE feedback circuitry and breaks homeostatic control of cytotoxic IE proteins inducing apoptosis only in infected cells.

Unmet Medical Need

Treatment-resistant CMV is the leading cause of transplant rejection and childhood deafness and cognitive impairment. Current standard of care countermeasures (e.g. ganciclovir) are subject to escape and excessive toxicity. The proposed therapeutic is robust to escape and exhibits favorable toxicity

Project Objective

Pre-IND submission

Major Proposed Activities

  • Formulation
  • Animal Studies
  • Process Development and Scale-Up
Statement of Benefit to California:
California is ~40% Hispanic or Latinx, a population known to be at significantly increased risk of CMV infection and disease. CMV disease is a leading cause of transplant rejection and cognitive impairment in the US. An escape-resistant ONT-based intervention for CMV disease could have many advantages for California’s diverse population over the current standard of care.