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…
Targeted DOK7 gene therapy for Congenital Myasthenic Syndromes
Therapeutic Candidate or Device The therapeutic candidate to be studied under this proposal is a gene therapy product for the treatment of DOK7 Congenital Myasthenic Syndrome Indication The target indication…
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…
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…
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…
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…
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…
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…
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