Therapeutic/Technology: Therapeutic Approach
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 […]
Human iPSC-derived GABAergic Progenitors for Alzheimer’s Disease Treatment
Translational Candidate Human iPSC-derived GABAergic interneuron progenitors. Area of Impact Alzheimer's disease and related conditions. Mechanism of Action Transplantation of human iPSC-derived GABAergic progenitors, which will develop into mature GABAergic interneurons, to replace the lost GABAergic interneurons in the hippocampus of AD brains and related disorders. Unmet Medical Need As a complex disease that damages […]
Curing Sickle cell Disease with CRISPR-Cas9 genome editing
Translational Candidate The principal objective of this program is to bring a Cas9-based gene editing cure for sickle cell disease to the pre-IND stage of development. Area of Impact The principal barriers to transplant for SCD are lack of a donor and the toxicity of transplant, which can be overcome by the Cas9-based approach Mechanism […]
Curing Sickle cell Disease with CRISPR-Cas9 genome editing
Translational Candidate The principal objective of this program is to bring a Cas9-based gene editing cure for sickle cell disease to the pre-IND stage of development. Area of Impact The principal barriers to transplant for SCD are lack of a donor and the toxicity of transplant, which can be overcome by the Cas9-based approach Mechanism […]
Pluripotent stem cell-derived chondrocytes for articular cartilage repair
Translational Candidate We propose to develop a universal, off-the-shelf treatment for articular cartilage repair based on pluripotent stem cell (PSC)-derived chondrospheres Area of Impact The proposed therapy could treat the major cartilage lesions present in more than 10% of people under 50; which often result in pain and arthritis Mechanism of Action Untreated cartilage defects […]
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 […]
Personalized Cell Therapy for Diabetes
Translational Candidate Preclinical studies will develop patient specific stem cell-derived islets that secrete insulin & other islet hormones for regulation of blood sugar Area of Impact Genetically matched stem cell derived islets could provide treatment for diabetes without the need for immunosuppression or implantable devices. Mechanism of Action The stem cell-derived islets contain insulin-producing pancreatic […]
Personalized Cell Therapy for Diabetes
Translational Candidate Preclinical studies will develop patient specific stem cell-derived islets that secrete insulin & other islet hormones for regulation of blood sugar Area of Impact Genetically matched stem cell derived islets could provide treatment for diabetes without the need for immunosuppression or implantable devices. Mechanism of Action The stem cell-derived islets contain insulin-producing pancreatic […]
Human Embryonic Stem Cell-Derived Neural Stem Cell Transplants in Amyotrophic Lateral Sclerosis
Translational Candidate Spinal cord injections of human embryonic stem cell (hESC)-derived allogeneic neural stem cells (heNSCs) for treatment of ALS Area of Impact Treatment of Amyotrophic Lateral Sclerosis (ALS) Mechanism of Action Although the exact molecular mechanism of action is unknown, extensive research supports the concept that the behavior of defective astrocytes is key to […]
Stem Cell-Based iNKT Cell Therapy for Cancer
Translational Candidate Lenti/iNKT-sr39TK Modified Autologous Human CD34+ Hematopoietic Stem Cells (HSCs) Area of Impact The targeted area of impact for the candidate is cancer therapy, in particular cancers that are lacking existing effective treatments. Mechanism of Action The proposed candidate will generate therapeutic levels of invariant natural killer T (iNKT) cells in cancer patients, helping […]