Therapeutic Approach – Page 4

Transplantation of CRISPR-CAS9 Corrected Hematopoietic Stem Cells (CRISPR_SCD001) in Patients with Severe Sickle Cell Disease

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Post published:January 25, 2026
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Therapeutic Candidate or Device CRISPR corrected blood stem cells are manufactured from persons with severe sickle cell disease and returned by transplant to the same person Indication Persons with sickle…

A Phase 1 Study of ECT-001 Expanded Cord Blood and Myeloablative Regimen with Reduced Toxicity in Patients with Severe Sickle Cell Disease.

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Post published:January 25, 2026
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Therapeutic Candidate or Device ECT-001 graft contains more stem and immune cells than conventional cord blood graft, leading to prompt recovery and better outcomes for patients. Indication Severe Sickle Cell…

University of California, San Francisco (UCSF) CIRM Alpha Stem Cell Clinic

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Post published:January 25, 2026
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We propose a cellular trial operation at UCSF that will focus on childhood hereditary hematological and immunologic disorders and in anti-inflammatory lung cellular therapies. This alpha clinic operation at UCSF…

University of California, San Francisco (UCSF) CIRM Alpha Stem Cell Clinic

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Post published:January 25, 2026
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We propose a cellular trial operation at UCSF that will focus on childhood hereditary hematological and immunologic disorders and in anti-inflammatory lung cellular therapies. This alpha clinic operation at UCSF…

Therapeutic targeting of Glioblastoma Stem Cell survival and self-renewal signaling

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Post published:January 25, 2026
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Research Objective This DISC2 tests a strategy to target survival and self-renewal in glioma stem cells (GSC), and will identify a therapeutic candidate for safety, dosing and efficacy testing. Impact…

Chemically engineered photoreceptors for vision restoration in retinal degeneration associated blindness.

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Post published:January 25, 2026
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Research Objective This proposal will develop a cell-based therapy that can restore vision in retinal degeneration associated blindness such as Stargardt disease and age-related macular degeneration. Impact Chemically induced method…

Developing a universal CRISPR gene therapy approach to treat C9orf72 ALS

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Post published:January 25, 2026
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Research Objective Development of a CRISPR genome editing therapy for ALS caused by C9orf72 and the delivery vehicle required to bring this novel therapy to patients. Impact A cure for…

B cell receptor-mediated lentiviral expression of anti-HIV antibody

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Post published:January 25, 2026
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Research Objective With a single administration to an individual, lentiviral vectors that selectively transduce B cells in vivo and express highly potent anti-HIV-1 proteins to suppress HIV-1 replication throughout life…

Drug discovery for Charcot Marie Tooth Disease using hPSC-derived Schwann cells

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Post published:January 25, 2026
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Research Objective The therapeutic candidate we aim to discover under this award is clomipramine, an antidepressant that might help reduce a harmful protein in the nerve cells of patients with…

Development of a Trifunctional Self-Renewing Memory CAR T Cell Therapy to Overcome the Heterogeneity and Suppressive Microenvironment of Glioblastoma

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Post published:January 25, 2026
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Research Objective This personalized chimeric antigen receptor (CAR) T cell therapy will attack a wider range of cancer cells, and block cancer's defense mechanisms to empower the immune system to…

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