Pre-Clinical To Clinical Gene Therapy Development For CMT4J

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Grant Award Details

Grant Number:

Award Value:

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


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.