Therapeutic/Technology: Biologic
Clinical Translation of hESC-derived protein therapy that positively regulates the regenerative capacity of post-natal muscle for treating DM1
Translational Candidate We engineered a human embryonic stem cell-secreted signaling protein into a biologic for treatment of skeletal muscle disorders. Area of Impact Skeletal muscle disorders (including DM1 and sarcopenia) remain major unmet needs that require treatments restoring muscle strength and function. Mechanism of Action Our animal data demonstrate an endocrine stimulation by our biologic […]
PRPE-SF, polarized hESC-derived RPE Soluble Factors, as a Therapy for Early Stage Dry Age-related Macular Degeneration
Translational Candidate PRPE-SF is a preparation of soluble factors from polarized retinal pigment epithelial cells, to support survival of photoreceptors in dry AMD (dAMD). Area of Impact dAMD with early geographic atrophy (RPE dysfunction/photoreceptor degeneration) that does not involve the fovea, with visual acuity better than 20/80. Mechanism of Action PRPE-SF is composed of multiple […]
Injectable pro-regenerative scaffold for treating symptomatic peripheral artery disease
Translational Candidate Injectable biomaterial derived from the natural scaffolding of porcine muscle Area of Impact Improving the quality of life of patients with symptomatic peripheral artery disease. Mechanism of Action The proposed mechanism of action is through recruitment of blood vessels and recruitment and differentiation of muscle stem cells. The injected material forms a new […]
An autologous somatic stem cell therapy for the treatment of osteonecrosis
Translational Candidate An autologous somatic stem cell therapy for the treatment of osteonecrosis. Area of Impact Osteonecrosis is a painful, progressive disease for which there is no treatment, save replacing the dead bone with a metal implant. Mechanism of Action Autografts contain skeletal stem cells. In young patients, these stem cells differentiate and give rise […]
2nd Generation Vaccine for the Treatment of Glioblastoma
Translational Candidate It is the peptide LEEKKYNYVVVTDHC conjugated to KLH and used as an anti-cancer vaccine. Area of Impact This is a better optimized, more robust vaccine that aspires to greatly improve glioblastoma patient survival over the current vaccine. Mechanism of Action The vaccine stimulates B cell and T cells. We have found this may […]
Phase 2b Clinical Study of KPI-012 Topical Ophthalmic Human Mesenchymal Stem Cell Secretome for the Treatment of Persistent Corneal Epithelial Defect
Therapeutic Candidate or Device KPI-012 is a human bone marrow mesenchymal stem cell secretome formulated as a topical ophthalmic therapeutic Indication Treatment of persistent corneal epithelial defect, a cornea defect refractory to conventional treatments that can lead to blindness Therapeutic Mechanism KPI-012 is intended to restore normal corneal wound healing, providing a curative solution to […]
Reduced intensity conditioning with JSP191 prior to TCRαβ+ T-cell/CD19+ B-cell depleted hematopoietic stem cell transplant for Fanconi Anemia patients
Therapeutic Candidate or Device αβdepleted-HSC grafts and a reduced-intensity preparative conditioning regimen containing JSP191 Indication Treatment of patients with all genetic forms of Fanconi Anemia (FA) with evidence of cytopenias Therapeutic Mechanism Fanconi anemia (FA) patients undergoing HCT have heightened sensitivity to current conditioning regimens and short and long-term sensitivity to graft versus host disease […]
Phase 1 Clinical Development of IO-202, A First-in-Class Antibody Targeting LILRB4, for the Treatment of AML with Monocytic Differentiation and CMML
Therapeutic Candidate or Device IO-202, a first-in-class antibody targeting leukocyte immunoglobulin-like receptor B4 (LILRB4), an immune inhibitory receptor Indication Acute myeloid leukemia (AML) with monocytic differentiation and chronic myelomonocytic leukemia (CMML) Therapeutic Mechanism IO-202 is the first T-cell activator for AML. Preclinical studies showed that IO-202 can convert a “don’t kill me” to “kill me” […]
A monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants
Therapeutic Candidate or Device CD34+CD90+ hematopoietic stem cells (HSC) in combination with AMG 191, a humanized anti-CD117 monoclonal antibody Indication Severe Combined Immunodeficiency Therapeutic Mechanism AMG 191 is being utilized as a conditioning agent for selectively eliminating endogenous stem cells in pediatric SCID patients prior to CD34+CD90+ hematopoietic stem cell transplantation for repopulation of the […]
A monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants
Therapeutic Candidate or Device CD34+CD90+ hematopoietic stem cells (HSC) in combination with AMG 191, a humanized anti-CD117 monoclonal antibody Indication Severe Combined Immunodeficiency Therapeutic Mechanism AMG 191 is being utilized as a conditioning agent for selectively eliminating endogenous stem cells in pediatric SCID patients prior to CD34+CD90+ hematopoietic stem cell transplantation for repopulation of the […]