Therapeutic/Technology: Therapeutic Approach

Development of an AAV gene therapy immunotherapy for the treatment of glioblastoma

Translational Candidate SRN-101, an experimental AAV gene therapy for treating glioblastoma Area of Impact Cancer – solid tumors Mechanism of Action Following delivery with an AAV, engineered cytokines are expressed from within the tumor to kill the tumor from the inside out, they are then further secreted to stimulate local immune cells to kill remaining […]

Pro-regenerative infusible ECM biomaterial for treating acute myocardial infarction

Translational Candidate Injectable biomaterial derived from the natural scaffolding of pig hearts Area of Impact Improving the quality of life of patients with heart attacks Mechanism of Action The proposed mechanism of action is through recruitment of the body's own stem cells and reducing inflammation to heal the heart. Unmet Medical Need The prevalence of […]

Hematopoietic Stem Cell Gene Editing for X-linked Agammaglobulinemia (XLA)

Translational Candidate Autologous CD34+ hematopoietic stem and progenitor cells (HSPC) with BTK gene insertion for treatment of X-linked agammaglobulinemia (XLA). Area of Impact The candidate will provide improved outcomes for patients with XLA, by allowing autologous transplantation with reduced intensity conditioning Mechanism of Action The Drug Product has biological activity of hematopoietic stem cells (HSC) […]

Adenine Base Editing for Autologous Hematopoietic Stem Cell Gene Therapy of CD3δ SCID

Translational Candidate The translational candidate is Autologous Hematopoietic Stem and Progenitor Cells from CD3δ SCID Patients Corrected by Adenine Base Editing Area of Impact The candidate will provide treatment for a fatal inborn error of immunity (CD3δ SCID) affecting a genetically-isolated population. Mechanism of Action Autologous Hematopoietic Stem and Progenitor cells from CD3δ SCID Patients […]

Development of a Gene Therapy for the Treatment of Arginase Deficiency – Translating from Proof of Concept to Support Pre-IND Meeting

Translational Candidate Adeno-associated viral vector serotyped for hepatic tropism to express Arginase 1 in hepatocytes. Area of Impact Developing a new therapy for Arginase Deficiency, where present day this is minimally effective at best. Mechanism of Action The proposed clinical candidate is a virus that has been altered to carry the gene for & produce […]

Ex Vivo Modified Hematopoietic Stem Cells to Treat Danon Disease

Translational Candidate The candidate is CD34+ HSPCs transduced ex vivo with a LAMP2 lentiviral vector. Area of Impact Danon Disease, Lysosomal Storage Diseases, Drug Development for Rare Disease Mechanism of Action Engrafted HSPC progeny will supply normal LAMP2B to the heart, liver, muscle, and brain via lysosomal cross-correction. Specifically, macrophages transfer lysosomes containing LAMP2B to […]

Targeting multiple myeloma with BCMA-CAR NK cells expressing a GPRC5D-NKG2D bispecific antibody

Translational Candidate s15.BsAb.BCMA-CAR NK cells derived from CD34(+) umbilical cord blood hematopoietic stem cells Area of Impact patients with multiple myeloma Mechanism of Action s15.BsAb.BCMA-CAR NK cells are umbilical cord blood-derived CD34+ hematopoietic stem cells that are engineered to include two components of the BCMA-CAR and the anti-NKG2D-anti-GPRC5D BsAb. s15.BsAb.BCMA-CAR NK cells can launch dual […]

Hematopoietic Stem Cell Gene Therapy for Wiskott Aldrich Syndrome

Translational Candidate Human hematopoietic stem cells that have been modified to express a functional WAS gene to treat patients with Wiskott Aldrich Syndrome (WAS) Area of Impact These studies will bring stem cell gene therapy for WAS closer to the clinic especially for those without an HLA match or disease too severe for HSCT Mechanism […]

AAV Gene Therapy for Treating Congenital Hereditary Endothelial Dystrophy associated with Biallelic SLC4A11 Mutations

Translational Candidate Therapeutic candidate rAAV8-EF1α-hSLC4A11 is a recombinant AAV vector with single-stranded cDNA encoding the wild-type human SLC4A11 protein. Area of Impact The candidate is for treatment of congenital hereditary endothelial dystrophy, an orphan disease associated with congenital corneal opacification. Mechanism of Action Therapeutic candidate rAAV8-EF1α-hSLC4A11 introduces normal copies of human SLC4A11 gene into the […]

Development of Autologous Cell Replacement Therapy for Parkinson’s Disease: Path to Personalized Treatment

Translational Candidate autologous iPSC-derived dopaminergic progenitor cells Area of Impact Parkinson's Disease Mechanism of Action Autologous iPSC-derived dopaminergic progenitor cells represent a promising strategy to replace the nigrostriatal cells which are lost in Parkinson's Disease (PD). While approaches using fetal tissue / allogeneic stem cells show great promise, they are not sufficiently personalized to provide […]