Program Type: Clinical


Manufacturing of AS-202, an Antisense oligonucleotides for a Phase 1/2 Clinical Trial for Amyotrophic Lateral Sclerosis

Therapeutic Candidate or Device AS-202, an antisense oligonucleotide Indication Amyotrophic Lateral Sclerosis Therapeutic Mechanism AS-202 targets PIKFYVE, a lipid kinase. By inhibiting PIKFYVE activity, it induces exosomal secretion, which robustly clears misfolded proteins including C9ORF72 dipeptide repeat proteins (DPRs), TDP-43 and tau from neurons. Unmet Medical Need To date, therapeutic options for ALS have been […]

Extracellular Vesicles for Ventricular Tachycardia

Therapeutic Candidate or Device CSSHI-2003 (extracellular vesibles derived from cardiohsphere derived cells) Indication Patients afflicted with recurrent fast and irregular heart beating (a.k.a. ventricular tachycardia). Therapeutic Mechanism CSHHI-2003 (CDC exosomes) are lipid vesicles filled with bioactive materials that have anti-inflammatory, anti-fibrotic, and regenerative properties. Decreasing the fibrosis in the heart can decrease the incidence of […]

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 […]

Autologous Gene Corrected Sinus Basal Cells to Treat Serious Cystic Fibrosis Sinus Disease

Therapeutic Candidate or Device Gene corrected autologous sinus airway basal stem cells from patients with Cystic Fibrosis. Indication The proposed studies provide an innovative stem cell based approach with gene correction to treat chronic sinusitis in CF. Therapeutic Mechanism Corrected upper airway cells will produce differentiated epithelium with restored Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) […]

Treatment of the TMJ disc complex

Therapeutic Candidate or Device Tissue implant engineered using expanded, allogeneic chondroprogenitor cells Indication Defects of the temporomandibular joint disc complex Therapeutic Mechanism Though the mechanism has not been established, Hyaleon®️ may initially act as a load bearing structure that fills defects of the temporomandibular joint (TMJ) disc complex. The implant is slowly remodeled allowing for […]

Cancer Stem Cell Interception with Rebecsinib: A First-in-Class ADAR1 Inhibitor

Therapeutic Candidate or Device Rebecsinib is a novel small molecule inhibitor of ADAR1 splicing that selectively eradicates therapy-resistant cancer stem cells in blood cancers. Indication The target indication is relapsed/refractory secondary acute myeloid leukemia (sAML), or int-2 or high-risk myelofibrosis (MF). Therapeutic Mechanism Rebecsinib therapy for patients with relapsed/refractory sAML, or int-2 or HR MF […]

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 […]

Ex vivo Engineering of Autologous Hematopoietic Stem Cells for the Treatment of Hypophosphatasia

Therapeutic Candidate or Device Hematopoietic stem/progenitor cells collected from patients with hypophosphatasia and genetically modified with a lentiviral vector to release TNALP Indication Hypophosphatasia (HPP) Therapeutic Mechanism We are proposing a cell-based enzyme replacement therapy for HPP: autologous gene-modified hematopoietic stem/progenitor cells (HSPCs) will be infused into the patient and will engraft into the bone […]

Allogeneic iPSC derived Dopaminergic Drug Product for Parkinson’s disease

Therapeutic Candidate or Device Allogeneic iPSC derived dopamine progenitors delivered to the brain of Parkinson's disease patients Indication Idiopathic Parkinson's disease Therapeutic Mechanism The cellular product reconstitutes dopaminergic neuron circuits lost owing to the disease Unmet Medical Need There are currently disease modifying therapies. This approach is intended to be disease modifying. Project Objective To […]