Project Objective: IND
A Translating Center designed to expedite the development and delivery of high quality stem cell therapies to patients
A successful partnership, resulting in the creation of a Translating Center recognized as the leader in providing end-to-end preclinical development services (preclinical research, CMC, regulatory) to accelerate the availability of stem cell treatments to patients with unmet medical needs. The proposed Translating Center will benefit the lives of patients in CA with serious unmet medical […]
Novel Gene Therapy Targeting Multiple Pathological Drivers of Desmoplakin Associated Arrhythmogenic Cardiomyopathy
Therapeutic Candidate or Device RJB-0402 is a liver-targeting adeno-associated virus vector-based gene therapy that drives over expression of FGF21. Indication Desmoplakin-related arrhythmogenic cardiomyopathy (DSP ACM) at high risk of life-threatening ventricular arrhythmias and sudden cardiac death. Therapeutic Mechanism RJB-0402 targets hepatocytes to drive liver specific expression of FGF21 to restore cardiomyocyte function in patients with […]
Human Embryonic Stem Cell-Derived Neural Stem Cells for Severe Spinal Cord Injury (SCI)
Therapeutic Candidate or Device Human embryonic stem cell-derived neural stem cells. Indication Spinal cord injury Therapeutic Mechanism The proposed neural stem cell therapy will be transplanted into a spinal cord injury site, providing cell replacement and a new neuronal relay across the injury site in an effort to promote significant functional improvement. Unmet Medical Need […]
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 […]