Therapeutic Approach – Page 25

Optogenetic therapy for treating retinitis pigmentosa and other inherited retinal diseases

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Post published:May 23, 2025
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Translational Candidate Ray-001 is an AAV gene therapy delivering a light sensitive gene to treat patients with advanced Retinitis Pigmentosa. Area of Impact Retinitis pigmentosa (RP) is a genetic disease…

CD72 nanoCARs for the treatment of refractory pediatric B-cell acute lymphoblastic leukemia

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Post published:May 23, 2025
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Translational Candidate CD72-targeting chimeric antigen receptor (CAR) T cells incorporating fully synthetic nanobodies Area of Impact Pediatric B-cell acute lymphoblastic leukemia refractory to currently available treatments without other potentially curative…

Development of MyoDys45-55, a gene editing therapy for Duchenne muscular dystrophy

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Post published:May 23, 2025
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Translational Candidate A gene editing therapy for Duchenne muscular dystrophy that permanently removes a hotspot region of patient mutations to restore dystrophin. Area of Impact Duchenne muscular dystrophy (DMD), a…

IND-enabling Studies of Wearable Evolve-FSTL1 for Cardiac Regeneration after MI

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Post published:May 23, 2025
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Translational Candidate The therapeutic candidate is the Regencor’s proprietary Cardio-Regenerative Factor (FSTL1.37) formulated in the Wearable Injector Evolve-FSTL1. Area of Impact The targeted area of impact is to restore cardiac…

MPS II: Plasma cell delivery of iduronate sulfatase

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Post published:May 23, 2025
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Translational Candidate The patient’s own B cells will be engineered to express the therapeutic enzyme needed for care in Mucopolysaccharidosis type II (MPS II) patients Area of Impact MPS II,…

Development of novel synNotch CART cell therapy in patients with recurrent EGFRvIII+ glioblastoma

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Post published:May 23, 2025
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Translational Candidate Human T cells transduced with a lentiviral vector encoding anti-EGFRvIII synNotch-primed anti-EphA2/IL-13Rα2 chimeric antigen receptor. Area of Impact Glioblastoma is the most common malignant brain tumor, affecting approximately…

Hematopoietic Stem Cell Gene Therapy for IPEX Syndrome

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Post published:May 23, 2025
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Translational Candidate Human hematopoietic stem cells that have been modified to express a functional FOXP3 gene to treat patients with IPEX Syndrome Area of Impact These studies will bring stem…

Targeting stromal progenitors to prevent the development of heart failure

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Post published:May 23, 2025
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Translational Candidate Monoclonal antibody targeting Ectonucleotide phosphodiesterase/pyrophosphatase 1 (ENPP1) Area of Impact Heart disease: To prevent the development of heart failure after heart attacks Mechanism of Action After myocardial infarction,…

Clinical Translation of Allogenic Regenerative Cell Therapy for White Matter Stroke and Vascular Dementia

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Post published:May 23, 2025
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Translational Candidate Human induced pluripotent stem cell-derived glial enriched progenitors Area of Impact Vascular dementia and white matter stroke, addressing a current bottleneck of poor scale up for existing cell…

Clinical Translation of Allogenic Regenerative Cell Therapy for White Matter Stroke and Vascular Dementia

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Post published:May 23, 2025
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Translational Candidate Human induced pluripotent stem cell-derived glial enriched progenitors Area of Impact Vascular dementia and white matter stroke, addressing a current bottleneck of poor scale up for existing cell…

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