Therapeutic Approach – Page 22

Hypoimmunogenic iPSC-derived TCR-NK cells for oncology

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Post published:May 23, 2025
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Translational Candidate iPSC-derived NY-ESO-1 TCR/IL-15 NK cells Area of Impact NK drug product homogeneity and engraftment/response durability will be improved by this hypoimmunogenic iPSC-TCR-NK therapy in cancer patients Mechanism of…

Optogenetic Therapy for Treatment of Geographic Atrophy

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Post published:May 23, 2025
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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…

Development of a Gene Therapy for Treatment of Guanidinoacetate Methyltransferase Deficiency-Translating In Vivo Proof of Concept to Support a Pre-IND

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Post published:May 23, 2025
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Translational Candidate Adeno-associated viral vector serotyped for tropism to express guanidinoacetate methyltransferase in hepatocytes and brain cells. Area of Impact Developing a new therapy for Guanidinoacetate Methyltransferase Deficiency, where present…

A high quality, accessible cell therapy for Parkinson’s Disease produced in a scalable bioreactor system for 3D cell expansion and differentiation

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Post published:May 23, 2025
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Translational Candidate Human pluripotent stem cell expanded and differentiated dopaminergic precursor cells to treat Parkinson’s Disease at high quantity and high quality Area of Impact This candidate uses a 3D…

Neurogenic hydrogel stimulation of stem cells to regenerate radiation-damaged salivary glands

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Post published:May 23, 2025
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Translational Candidate Ceviginate is a neuromimetic encapsulated in a hydrogel Area of Impact Dry mouth as a result of injury to the salivary glands by radiation therapy for head and…

Development of an AAV gene therapy immunotherapy for the treatment of glioblastoma

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Post published:May 23, 2025
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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…

Noncoding RNA drug TY1 as a therapeutic candidate for scleroderma and systemic sclerosis

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Post published:May 23, 2025
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Translational Candidate Modified synthetic noncoding RNA molecule Area of Impact Systemic Sclerosis Mechanism of Action The mechanism of action of TY1 is alleviating cell stress and damage through enhancing genes…

Adenine Base Editing for Autologous Hematopoietic Stem Cell Gene Therapy of CD3δ SCID

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Post published:May 23, 2025
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Translational Candidate The translational candidate is Autologous Hematopoietic Stem and Progenitor Cells from CD3δ SCID Patients Corrected by Adenine Base Editing Area of Impact The candidate will provide treatment for…

Pro-regenerative infusible ECM biomaterial for treating acute myocardial infarction

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Post published:May 23, 2025
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Translational Candidate Injectable biomaterial derived from the natural scaffolding of pig hearts Area of Impact Improving the quality of life of patients with heart attacks Mechanism of Action The proposed…

Development of a Gene Therapy for the Treatment of Arginase Deficiency – Translating from Proof of Concept to Support Pre-IND Meeting

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Post published:May 23, 2025
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Translational Candidate Adeno-associated viral vector serotyped for hepatic tropism to express Arginase 1 in hepatocytes. Area of Impact Developing a new therapy for Arginase Deficiency, where present day this is…

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