Grant Award Details
- 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.
Grant Application Details
- A Phase I Clinical Trial for a Lentiviral Gene Therapy Targeting the TCIRG1 Gene for Infantile Malignant Osteopetrosis (IMO)
Therapeutic Candidate or Device
The therapeutic candidate is an ex-vivo autologous gene therapy approach for Infantile Malignant Osteopetrosis (IMO).
The target clinical indication is Infantile Malignant Osteopetrosis (IMO), a pediatric, autosomal recessive rare disease.
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.
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
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.