Stage of Program: Clinical Stage Programs (2.0 and relevant 1.0 projects)


Development of a therapeutic monoclonal antibody for the treatment of myocardial infarction and heart failure

Therapeutic Candidate or Device Fully Humanized monoclonal antibody targeting human ectonucleotide pyrophosphatase/phosphodiesterase (ENPP1) Indication Heart Disease: To prevent the development of heart failure after heart attacks Therapeutic Mechanism After myocardial infarction, myofibroblast progenitors express ENPP1. ENPP1 is a type II transmembrane protein that hydrolyzes extracellular ATP and hydrolytic products generated by ENPP1 initiate an inflammatory […]

Chimeric TGFB Signaling Receptor (CTSR) Enabled Anti-B7H3 CAR T-cell Therapy in Children and AYA with Recurrent Solid Tumors

Therapeutic Candidate or Device Engineered CAR T-cells are enhanced to allow their survival in solid tumors Indication Children, adolescents and young adults with variety of solid tumors but focus on sarcomas and neuroblastomas Therapeutic Mechanism Engineered T-cells are expanded and infused back to the patients. The additional mechanisms engineered in these cells allows them to […]

A 1XX-enhanced and fully non-viral BCMA CAR T cell therapy for Relapsed and Refractory Multiple Myeloma (UCCT-BCMA-1)

Therapeutic Candidate or Device Cryopreserved autologous TRAC locus 1XX BCMA-CAR T cells Indication Relapsed and Refractory Multiple Myeloma Therapeutic Mechanism Engineered T cells target and kill BCMA+ Myeloma cells Unmet Medical Need No durable treatments are available for RRMM and only ~30% of patients can access current BCMA CAR therapies. This product can improve product […]

Pre-Clinical To Clinical Gene Therapy Development For CMT4J

Therapeutic Candidate or Device AAV9 Gene Therapy For An Ultra-Rare Disease Called CMT4J Indication CMT4J or the FIG4 Gene Therapeutic Mechanism Gene Therapy through an Intrathecal One-Time Injection Unmet Medical Need CMT4J is an ultra-rare disorder that presently lacks any available treatment options and represents an underserved orphan population. Project Objective Successful Filing of an […]

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 […]

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 […]

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 […]