Therapeutic/Technology: Gene-modified, donor cell therapy
Human Neural Progenitors Secreting Glial Cell Line-Derived Neurotrophic Factor (CNS10-NPC-GDNF) for the Treatment of Amyotrophic Lateral Sclerosis
Therapeutic Candidate or Device CNS10-NPC-GDNF – a neural progenitor cell secreting GDNF Indication ALS Therapeutic Mechanism This therapy will replace damaged astrocytes. The new astrocytes will release paracrine factors. As the cells have been modified to release GDNF they will also provide this factor to dying motor neurons. Unmet Medical Need There is no treatment […]
Neural stem cell delivered CRAd-S-pk7 oncolytic viro-immunotherapy for ovarian cancer
Therapeutic Candidate or Device A clinically tested tumor-tropic neural stem cell (NSC) platform for effective distribution of oncolytic virotherapy to ovarian cancer metastases. Indication Chemo-resistant, metastatic ovarian cancer. Therapeutic Mechanism CRAd-S-pk7 is a tumor specific replication-competent adenovirus driven by survivin, which is highly expressed in ovarian cancer cells. We will use our tumor-tropic NSC platform […]
TRX103 for prevention of GvHD in patients receiving HLA mismatched related or unrelated allogeneic HSCT for the treatment of hematologic malignancies.
Therapeutic Candidate or Device An allogenic, off the shelf, engineered regulatory T cell product that mimics the function of T regulatory Type 1 (Tr1) cells. Indication Prevention of acute and chronic Graft versus Host Disease (GvHD) in patients undergoing mismatched stem cell transplant. Therapeutic Mechanism GvHD is driven by the reaction of allogenic donor T […]
MRI Guided Delivery of Neural Progenitor Cells Secreting GDNF for the Treatment of Parkinson’s disease
Therapeutic Candidate or Device CNS10-NPC-GDNF is a neural progenitor cell line transfected with glial cell line derived neurotrophic factor (GDNF) Indication Mid-stage Parkinson's disease (UPDRS stage III or lower) Therapeutic Mechanism Degeneration of dopaminergic neurons that project from the substantia nigra to the striatum causes the primary motor symptoms of Parkinson's disease. CNS10-NPC-GDNF cells will […]
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 […]
Programming Human ESC-derived Neural Stem Cells with MEF2C for Transplantation in Stroke
The goal of this project is to produce a stem cell-based therapy for stroke (also known as an ischemic cerebral infarct). Stroke is the third leading cause of death in the USA, and a leading cause of disability among adults. Currently, there are no effective treatments once a stroke has occurred (termed completed stroke). In […]
Programming Human ESC-derived Neural Stem Cells with MEF2C for Transplantation in Stroke
The goal of this project is to produce a stem cell-based therapy for stroke (also known as an ischemic cerebral infarct). Stroke is the third leading cause of death in the USA, and a leading cause of disability among adults. Currently, there are no effective treatments once a stroke has occurred (termed completed stroke). In […]
Progenitor Cells Secreting GDNF for the Treatment of ALS
This project aims to use a powerful combined neural progenitor cell and growth factor approach to treat patients with amyotrophic lateral sclerosis (ALS or Lou Gehrig’s Disease). ALS is a devastating disease for which there is no treatment or cure. Progression from early muscle twitches to complete paralysis and death usually happens within 4 years. […]
Phase I study of IM Injection of VEGF-Producing MSC for the Treatment of Critical Limb Ischemia
Critical limb ischemia (CLI) represents a significant unmet medical need without any approved medical therapies for patients who fail surgical or angioplasty procedures to restore blood flow to the lower leg. CLI affects 2 million people in the U.S. and is associated with an increased risk of leg amputation and death. Amputation rates in patients […]
MSC engineered to produce BDNF for the treatment of Huntington’s disease
One in every ten thousand people in the USA has Huntington’s disease, and it impacts many more. Multiple generations within a family can inherit the disease, resulting in escalating health care costs and draining family resources. This highly devastating and fatal disease touches all races and socioeconomic levels, and there are currently no cures. Screening […]