Grant Award Details

A Phase I Clinical Trial for a Lentiviral Gene Therapy Targeting the TCIRG1 Gene for Infantile Malignant Osteopetrosis (IMO)
Grant Number: 
CLIN2-12095
Project Objective: 
  • Conduct a Phase 1 trial to assess the safety and preliminary efficacy of transplanted, autologous, lentivirally transduced, TCIRG1-expressing hematopoeitic stem cells in children with Infantile Malignant Osteopetrosis.
Investigator: 
Type: 
PI
Disease Focus: 
Bone or Cartilage Disease
Human Stem Cell Use: 
Adult Stem Cell
Award Value: 
$3,728,485
Status: 
Pre-Active

Grant Application Details

Application Title: 
  • A Phase I Clinical Trial for a Lentiviral Gene Therapy Targeting the TCIRG1 Gene for Infantile Malignant Osteopetrosis (IMO)
Public Abstract: 

Therapeutic Candidate or Device

The therapeutic candidate is an ex-vivo autologous gene therapy approach for Infantile Malignant Osteopetrosis (IMO).

Indication

The target clinical indication is Infantile Malignant Osteopetrosis (IMO), a pediatric, autosomal recessive rare disease.

Therapeutic Mechanism

The therapeutic mechanism is based on an ex-vivo, lentiviral-based, patient-specific approach by genetically engineering the patient's own CD34+ selected cells with the corrected genetic sequence of TCIRG1 gene and subsequently infusing the corrected cells to deliver a potential cure.

Unmet Medical Need

Children with severe IMO face morbid conditions such as hematologic and/or neurologic deficits that worsen over time . There is a high chance of death in the first decade of life without successful allogeneic HSCT. This gene therapy will treat the underlying TCIRG1 mutation in IMO patients.

Project Objective

Phase 1 trial completed

Major Proposed Activities

  • Patient recruitment, screening, and support (by various CROs) on their clinical journey
  • Enrollment of patients at UCLA
  • Cell processing at Lonza
Statement of Benefit to California: 

Clinical studies at UCLA Mattel Children's Hospital will further provide that medical and scientific community premier access to a cutting-edge gene therapy trial. There is a possibility that the patient(s) recruited is from California, but that is to be determined.