Gene Therapy (All) – Page 10

Toward a Cure for Gaucher Disease Type 1: Autologous Transplantation of Genome Edited Hematopoietic Stem Cells

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Post published:April 30, 2025
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Translational Candidate Autologous blood stem cells edited to restore glucocerebrosidase expression Area of Impact Gaucher disease type 1 (non-neuronopathic) Mechanism of Action To treat Gaucher disease, autologous blood stem cells…

Development of AS-241, an UNC13A Targeting Antisense Oligonucleotide (ASO) Treatment for ALS, for IND-enabling Studies

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Post published:April 30, 2025
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Translational Candidate AS-241, an antisense oligonucleotide Area of Impact Amyotrophic Lateral Sclerosis Mechanism of Action AS-241 targets the cryptic exon (CE) of UNC13A and suppresses CE inclusion during RNA splicing,…

Hypoimmunogenic iPSC-derived TCR-NK cells for oncology

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Post published:April 30, 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…

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:April 30, 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…

Optogenetic Therapy for Treatment of Geographic Atrophy

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Post published:April 30, 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 an AAV gene therapy immunotherapy for the treatment of glioblastoma

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Post published:April 30, 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:April 30, 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:April 30, 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…

Hematopoietic Stem Cell Gene Editing for X-linked Agammaglobulinemia (XLA)

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Post published:April 30, 2025
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Translational Candidate Autologous CD34+ hematopoietic stem and progenitor cells (HSPC) with BTK gene insertion for treatment of X-linked agammaglobulinemia (XLA). Area of Impact The candidate will provide improved outcomes for…

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:April 30, 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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