Therapeutic/Technology: Therapeutic Approach
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 […]
Process development for establishing an iPSC-based therapeutic candidate for Canavan disease
Translational Candidate Genetically-corrected patient iPSC-derived neural progenitor cells, which have demonstrated efficacy to correct disease phenotype in a CD mouse model. Area of Impact This candidate has the potential to develop into a therapy for Canavan disease, a fatal neurological disease that has no cure or standard treatment. Mechanism of Action The proposed candidate is […]
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 […]
Overexpression of HexA/HexB by lentivector expression in blood cells to treat Tay-Sachs and Sandhoff disease
Translational Candidate Autologous hematopoietic stem cells transduced with a lentiviral vector expressing wild type human HexA and HexB. Area of Impact The therapeutic candidate would halt disease progression in Tay-Sachs and Sandhoff disease patients who have no curative or ameliorating treatment. Mechanism of Action Wild type HexA and HexB will be delivered to affected neurons […]