Translational Candidate Optogenetic gene therapy for patients with geographic atrophy age related macular degeneration. Area of Impact Blindness from geographic atrophy age related macular degeneration Mechanism of Action Gene therapy…
Translational Candidate SRN-101, an experimental AAV gene therapy for treating glioblastoma Area of Impact Cancer - solid tumors Mechanism of Action Following delivery with an AAV, engineered cytokines are expressed…
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 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 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 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…
Therapeutic Candidate or Device SENTI-202 is an allogeneic off-the-shelf chimeric antigen receptor (CAR) natural killer (NK) cellular therapy targeting CD33 and/or FLT3 malignancies. Indication CD33 and/or FLT3 expressing hematologic malignancies,…
Therapeutic Candidate or Device NXC-201 Indication AL Amyloidosis Therapeutic Mechanism Genetically modified T-cell targeting B-cell maturation antigen (BCMA) Unmet Medical Need Amyloid light-chain (AL) amyloidosis is a devastating, rare plasma…
