This trial proposes to replace SCID patients' dysfunctional immune cells with healthy ones using a safer form of bone marrow transplant (BMT). Current BMT procedures must use toxic chemotherapy to…
Retinitis pigmentosa (RP) is a severe form of blindness that runs in families with an incidence of 1:4000. A team at UC Irvine, is using cells called retinal progenitor cells…
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…
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…
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…
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…
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…
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