"Curing the Incurable"

Open Clinical Trials

Read more about the Bass Center and their research here

Blood Stem Cell Enginerring and Transplantation

Gene Editing

Project Name	Project State	NCT Number
Gene Correction for MPS1	Pre-Clinical
Gene Correction for Gaucher	Pre-Clinical
Gene Correction for Cystic Fibrosis	Pre-Clinical
Gene Correction for SCID-X1	Discovery
Gene Correction for FOXP3 in IPEX	Discovery
Gene Correction for IL10 deficiency	Discovery
Gene therapy for Parkinson's Disease	Discovery
Genetically Engineered HSPCs for Sustained Delivery of Anti-HIV Antibodies	Discovery
Gene Base Editing Therapy For Sickle Cell Disease With BEAM Therapeutics	Open	NCT05456880

Engineering the Immune System

*Project Name*	*Project State*	*NCT Number*
T-allo10 with MR or MMUR HSCT for Hematologic Malignancies	Open
T-allo10 after alpha beta depletion Hematologic Malignancies	Open
Phase 1 study of autologous CD4LVFOXP3 T cells in subjects with IPEX Syndrome	Open
Ortho-IL2 engineered gd T cells	Discovery
Alpha Beta Crohns disease (Auto-Inflammatory HSCT with TCR aB depletion protocol)	Pre-Clinical
Viral‐Specific T Cell Therapy	Open	NCT04364178

Tissue Regeneration

Project Name	Project State	NCT Number
hESC-derived cardiomyocytes for chronic ischemic LV dysfunction	Open	NCT05068674
Neural Stem Cells (Stroke)	Open	NCT04631406
DEBCT CRISPR-iPS corrected Skin Grafts (RDEB)	Pre-Clinical
Autologous iPSC-derived smooth muscle (urinary incontinence )	Pre-Clinical
Enhanced Autologous Pancreatic Islet Transplantation and Survival for Diabetes Mellitus Therapy	Pre-Clinical

In Vivo Gene Transfer

Transferred to Industry

Blood Stem Cell Engineering and Transplantation

Gene Editing

Engineering the Immune System

Tissue Regeneration

In Vivo Transfer

CDCM Clinical Trials Transferred to Industry