Pre-Clinical To Clinical Gene Therapy Development For CMT4J
Grant Award Details
Grant Type:
Grant Number:
CLIN1-14945
Investigator(s):
Disease Focus:
Award Value:
$3,930,964
Status:
Active
Grant Application Details
Application Title:
Pre-Clinical To Clinical Gene Therapy Development For CMT4J
Public Abstract:
Therapeutic Candidate or Device
AAV9 Gene Therapy For An Ultra-Rare Disease Called CMT4J
Indication
CMT4J or the FIG4 Gene
Therapeutic Mechanism
Gene Therapy through an Intrathecal One-Time Injection
Unmet Medical Need
CMT4J is an ultra-rare disorder that presently lacks any available treatment options and represents an underserved orphan population.
Project Objective
Successful Filing of an IND
Major Proposed Activities
AAV9 Gene Therapy For An Ultra-Rare Disease Called CMT4J
Indication
CMT4J or the FIG4 Gene
Therapeutic Mechanism
Gene Therapy through an Intrathecal One-Time Injection
Unmet Medical Need
CMT4J is an ultra-rare disorder that presently lacks any available treatment options and represents an underserved orphan population.
Project Objective
Successful Filing of an IND
Major Proposed Activities
- Produce the therapeutic agent to support the proposed preclinical research activities.
- Evaluate the safety profile of the therapeutic in a animal model
- Assemble the preclinical data to submit an Investigational New Drug (IND) application.
Statement of Benefit to California:
This project will positively impact California and its residents by being managed by a California-based company, collaborating with local companies and consultants when feasible, and administering treatments at a California-based hospital, ultimately serving patients within the state and beyond.