Development of a Gene Therapy for the Treatment of WWOX related epileptic encephalopathy (WOREE)
Grant Award Details
Grant Type:
Grant Number:
CLIN1-14825
Investigator(s):
Award Value:
$4,000,000
Status:
Pre-Active
Grant Application Details
Application Title:
Development of a Gene Therapy for the Treatment of WWOX related epileptic encephalopathy (WOREE)
Public Abstract:
Therapeutic Candidate or Device
MZ-9138 is an AAV9 gene therapy
Indication
WWOX-related epileptic encephalopathy
Therapeutic Mechanism
MZ-9138 delivered to the brain may transduce neurons to create functioning WWOX protein and ultimately improve the phenotype of patients with WOREE
Unmet Medical Need
WOREE is a severe epileptic disorder resulting in dramatically shortened survival of patients. There are no therapies approved for WOREE and most patients do not respond to anti-epileptic drugs. MZ-9138 may improve symptoms and would be the first disease-modifying treatment for WOREE syndrome.
Project Objective
Phase 1-2 trial initiated
Major Proposed Activities
MZ-9138 is an AAV9 gene therapy
Indication
WWOX-related epileptic encephalopathy
Therapeutic Mechanism
MZ-9138 delivered to the brain may transduce neurons to create functioning WWOX protein and ultimately improve the phenotype of patients with WOREE
Unmet Medical Need
WOREE is a severe epileptic disorder resulting in dramatically shortened survival of patients. There are no therapies approved for WOREE and most patients do not respond to anti-epileptic drugs. MZ-9138 may improve symptoms and would be the first disease-modifying treatment for WOREE syndrome.
Project Objective
Phase 1-2 trial initiated
Major Proposed Activities
- Completion of GLP studies in mouse and nonhuman primate
- Manufacture of cGMP gene therapy to support the Phase 1-2 study
- Clinical activities including Phase 1-2 start up and the initiation of a natural history study.
Statement of Benefit to California:
It's estimated that 1:160,000 children are born with WOREE syndrome. To date, with the help of the WWOX Foundation, Mahzi has identified 4 in California. Mahzi, a California company, will collaborate with partner organizations & vendors in our state, including Rady Children's Consortium for newborn screening and will select clinical trial sites in California (UCSF). Our efforts will support identification and inclusion of California families in the pursuit of a therapy.