Therapeutic Candidate or Device Allogeneic human retinal progenitor cells (hRPC) Indication Retinitis Pigmentosa (RP) Therapeutic Mechanism The cells are intended to remain suspended in the vitreous cavity of the eye…
Therapeutic Candidate or Device The therapeutic product candidate is autologous CD34+ hematopoietic stem/progenitor cells (HSPC) transduced with the G1XCGD lentiviral vector. Indication The target indication is for the transplantation of…
Therapeutic Candidate or Device forward-oriented globin-expressing vector CD34+ HSCs Indication Severe Sickle Cell Disease (SCD) Therapeutic Mechanism Successful gene modified HSC generate RBC with effective repaired hemoglobin in 30% of…
Myelomeningocele – also known as spina bifida – is a devastating and costly defect that causes lifelong paralysis as well as bowel and bladder incontinence in newborns. It is one…
The proposed alpha clinic will bring together an outstanding team of physician-scientists with substantial clinical trials experience including stem cell and other cellular treatments of blood diseases and others. This…
The HIV-1 virus enters cells by binding to a protein called CCR5 on the cell surface. A naturally occurring mutation in CCR5, CCR5d32, has been shown to provide protection from…
Patients with end-stage type 1 diabetes (T1D) can be effectively managed by allogeneic islet transplantation. However, a severe cadaveric organ shortage greatly limits use of this promising procedure. Stem cells…
Sickle cell disease (SCD)results from an inherited mutation in the hemoglobin gene that causes red blood cells to "sickle" under conditions of low oxygen. It occurs with a frequency of…
The AIDS virus infects and destroys cells of the immune system such that the bodies of infected individuals cannot fight infections or some cancers. If untreated HIV infection leads to…
Disorders affecting the blood, including Sickle Cell Disease (SCD), are the most common genetic disorders in the world. SCD causes significant suffering and early death, despite major improvements in medical…
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