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…
Translational Candidate The therapeutic candidate is a central nervous system tissue-derived GMP line developed under DISC2-10753 with an established GMP qualified seed bank. Area of Impact The target is chronic…
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…
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…
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,…
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…
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…
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…
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,…
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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