Escape-Resistant Oligonucleotide Therapy (ONT) for Cytomegalovirus (CMV) Disease in Hematopoietic Stem-Cell and Solid-Organ Transplant Patients
Grant Award Details
Grant Type:
Grant Number:
TRAN1-14062
Investigator(s):
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
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.