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…
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…
- 1
- 2
- 3
- 4
- …
- 8
- Go to the next page
