Stage of Program: Clinical Stage Programs (2.0 and relevant 1.0 projects)
Phase 2 Study of Hematopoietic Stem Cell Gene Transfer Inducing Fetal Hemoglobin in Sickle Cell Disease
Boston Children’s Hospital is conducting a gene therapy clinical trial for sickle cell disease (SCD). This project is part of an agreement between CIRM and the National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health, to co-fund cell and gene therapy programs under the NHLBI’s “Cure Sickle Cell” Initiative. The […]
A phase I/II study of human placental hematopoietic stem cell derived natural killer cells (CYNK-001) for the treatment of adults with COVID-19
This trial will use blood stem cells obtained from the placenta to generate natural killer (NK) cells, a type of white blood cell that is a vital part of the immune system, and administer them to patients with COVID-19. NK cells play an important role in defense against cancer and in fighting off viral infections. […]
A Phase I Clinical Trial for a Lentiviral Gene Therapy Targeting the TCIRG1 Gene for Infantile Malignant Osteopetrosis (IMO)
Rocket Pharmaceuticals is conducting a clinical trial using a gene therapy for infantile malignant osteopetrosis (IMO), a rare and life-threatening disorder that develops in infancy. IMO is caused by defective bone cell function, which results in blindness, deafness, bone marrow failure, and death very early in life. The trial will use a gene therapy that targets IMO caused […]
Anti-HIV duoCAR-T cell therapy for HIV infection
The University of California San Francisco (UCSF) is conducting a clinical trial that modifies a patient’s own immune cells in order to treat and potentially cure HIV. Current treatment of HIV involves the use of long-term antiretroviral therapy (ART). However, many people are not able to access and adhere to long-term ART. The team will […]
Evaluation and Characterization of SARS-CoV-2 Antibody in Convalescent Volunteer Plasma Donors for Potential Therapeutic Use
Plasma is a component of blood that carries proteins called antibodies that are usually involved in defending our bodies against viral infections. Blood plasma from patients that have recovered from COVID-19, referred to as convalescent plasma, contain antibodies against the virus that can be used as a potential treatment for COVID-19.
Mesenchymal Stromal Cells for ARDS (COVID positive and COVID negative)
Acute respiratory distress syndrome (ARDS) is a life-threatening lung injury that occurs when fluid leaks into the lungs. As a result, breathing becomes difficult and oxygen cannot get into the body. ARDS is one of the most serious and lethal consequence of COVID-19. This clinical trial will use Mesenchymal Stromal Cells (MSCs) for the treatment of ARDS, […]
A Phase 1 Study of ECT-001 Expanded Cord Blood and Myeloablative Regimen with Reduced Toxicity in Patients with Severe Sickle Cell Disease
Sickle Cell Disease (SCD) is an inherited blood disorder caused by a single gene mutation that results in the production of “sickle” shaped red blood cells. It affects an estimated 100,000 people, mostly African American, in the US and can lead to multiple organ damage as well as reduced quality of life and life expectancy. […]
Safety and Feasibility of Cultivated Autologous Limbal Stem Cells for Limbal Stem Cell Deficiency
Limbal stem cell deficiency (LSCD) is a blinding corneal disease. LSCD is caused by a decrease in the number and/or function of limbal stem cells (LSCs), a type of stem cell that is needed to continuously regenerate tissue of the cornea, the clear front surface of the eye that refracts light entering the eye and is […]
Clinical Study to Assess Safety and Efficacy of Subretinal Injection of Human Neural Progenitor Cells for Treatment of Retinitis Pigmentosa
Retinitis pigmentosa is a blinding eye disease that affects approximately 150,000 individuals in the US and 1.5 million people around the world. It is caused by the destruction of light-sensing cells in the back of the eye known as photoreceptors. This leads to gradual vision loss and eventually blindness. For this trial, human neural progenitor […]
A Phase 2 Study of the Safety of Repeat Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects with Retinitis Pigmentosa
Retinitis pigmentosa is blinding eye disease that affects approximately 150,000 individuals in the US and 1.5 million people around the world. It is caused by the destruction of light-sensing cells in the back of the eye known as photoreceptors. This leads to gradual vision loss and eventually blindness. The approach for this trial involves injecting […]