Cell Line Generation: iPS Cell
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 […]
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 […]
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 […]
ASCENT- Advanced Stem Cell Enteric Neuropathy Therapy
Translational Candidate ASCENT – Advanced Superdonor Cellular Enteric Neuropathy Therapy, is a donor progenitor cell population that replaces the enteric nervous system. Area of Impact ASCENT would treat enteric neuropathies including Hirschsprung disease and total intestinal aganglionosis which currently have no direct therapy Mechanism of Action Our goal is to develop an allogeneic “off the […]
Phase 1/2a Dose Escalation Study of ANPD001 in Sporadic Parkinson Disease
Therapeutic Candidate or Device Autologous stem cell-derived dopaminergic neuron replacement therapy. Indication Parkinson’s disease Therapeutic Mechanism The investigational cell therapy product is being studied as an autologous neuron replacement for Parkinson’s disease. Unmet Medical Need PD is a neurodegenerative disorder, affecting 1+ million people in the US. PD causes loss of dopamine (DA) neurons in […]
Clinical Translation of Autologous Regenerative Pluripotent Stem Cell Therapy for Blindness
Therapeutic Candidate or Device The therapeutic candidate is a patient specific (autologous) induced pluripotent stem cell derived retinal pigment epithelium (AiPSC-RPE) product. Indication AiPSC-RPE cell product will be indicated for the treatment of maculopathies related to RPE atrophy. Therapeutic Mechanism The proposed therapy is an autlogous cell product and intended to be used as a […]
IND enabling development of FT516: A Natural Killer Cell Immunotherapy for Cancer Derived from a Human Inducible Pluripotent Stem Cell
Therapeutic Candidate or Device FT516: A Natural Killer Cell Immunotherapy for Cancer Derived from a Human Inducible Pluripotent Stem Cell Line Indication FT516 monotherapy for patients with advanced cancer and in combination with approved ADCC-competent monoclonal antibodies Therapeutic Mechanism FT516 drug product is comprised of natural killer (NK) cells derived from a clonal human induced […]
Human Stem-Cell Based Development of a Potent Alzheimer’s Drug Candidate
Over 6 million people in the US suffer from Alzheimer’s disease (AD). There are no drugs that prevent the death of nerve cells in AD, nor has any drug been identified that can stimulate nerve cell replacement in aged human brain. Importantly, even if nerve cells could be replaced, the toxic environment of the AD […]
A Chromatin Context Tool for Predicting iPS Lineage Predisposition and Tissue Graftability
Induced pluripotent stem (iPS) cells are cells derived from skin that closely resemble embryonic stem (ES) cells and can be coaxed into many different types of cells such as nerve cells, heart cells, liver cells, and also back to skin cells. One major bottleneck in the field is our ability to coax the cells into […]