Autologous Gene Corrected Sinus Basal Cells to Treat Serious Cystic Fibrosis Sinus Disease
Grant Award Details
Grant Type:
Grant Number:
CLIN1-14770
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Award Value:
$6,000,000
Status:
Pre-Active
Grant Application Details
Application Title:
Autologous Gene Corrected Sinus Basal Cells to Treat Serious Cystic Fibrosis Sinus Disease
Public Abstract:
Therapeutic Candidate or Device
Gene corrected autologous sinus airway basal stem cells from patients with Cystic Fibrosis.
Indication
The proposed studies provide an innovative stem cell based approach with gene correction to treat chronic sinusitis in CF.
Therapeutic Mechanism
Corrected upper airway cells will produce differentiated epithelium with restored Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene function. This restored function will enable improved muco-ciliary clearance which will resolve chronic sinusitis in CF patients and dramatically improve quality of life.
Unmet Medical Need
Small molecule modulators for CF cannot treat all patients. Previous attempts using viral and non-viral gene therapies have been unsuccessful. CRISPR/Cas9 genome editing enabling the precise correction of CF causing mutations in airway stem cells offers a durable autologous cell therapy to treat CF.
Project Objective
Filing of IND application with the FDA
Major Proposed Activities
Gene corrected autologous sinus airway basal stem cells from patients with Cystic Fibrosis.
Indication
The proposed studies provide an innovative stem cell based approach with gene correction to treat chronic sinusitis in CF.
Therapeutic Mechanism
Corrected upper airway cells will produce differentiated epithelium with restored Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene function. This restored function will enable improved muco-ciliary clearance which will resolve chronic sinusitis in CF patients and dramatically improve quality of life.
Unmet Medical Need
Small molecule modulators for CF cannot treat all patients. Previous attempts using viral and non-viral gene therapies have been unsuccessful. CRISPR/Cas9 genome editing enabling the precise correction of CF causing mutations in airway stem cells offers a durable autologous cell therapy to treat CF.
Project Objective
Filing of IND application with the FDA
Major Proposed Activities
- Develop process for Patient Scale Manufacturing Runs and complete three runs that meet release specifications
- Completion of nonclinical feasibility, safety and biodistribution studies
- Submit clinical protocol to IRB and file an IND with the FDA
Statement of Benefit to California:
Cystic Fibrosis (CF) is one of the most common genetic diseases in California. There is no curative therapy for CF and CF patients spend a lifetime focused on mitigating the symptoms of their disease. Moreover, the costs of treating a single CF patient are enormous. Thus, the benefit to California if this proposal is successful is that it would improve the lives of its citizens (both patients and family members) while simultaneously decreasing the societal costs associated with this disease.