A monoclonal antibody that depletes blood stem cells and enables chemotherapy free transplants

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• Post published:April 14, 2026
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This trial proposes to replace SCID patients' dysfunctional immune cells with healthy ones using a safer form of bone marrow transplant (BMT). Current BMT procedures must use toxic chemotherapy to…

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Retinal progenitor cells for treatment of retinitis pigmentosa

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• Post published:April 14, 2026
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Retinitis pigmentosa (RP) is a severe form of blindness that runs in families with an incidence of 1:4000. A team at UC Irvine, is using cells called retinal progenitor cells…

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A First-in-Class CIRSPR-CasX Gene Editing Therapy for Lowering Lp(a) to Prevent Cardiovascular Events

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• Post published:April 14, 2026
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Gene Therapy for Alpha-1 Anti-Trypsin Deficiency

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Late-stage development of AS-241, an UNC13A Targeting Antisense Oligonucleotide treatment for Amyotrophic Lateral Sclerosis, for IND-enabling studies

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• Post published:April 14, 2026
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Continue ReadingLate-stage development of AS-241, an UNC13A Targeting Antisense Oligonucleotide treatment for Amyotrophic Lateral Sclerosis, for IND-enabling studies

Advancement of a myotropic, liver-detargeting therapy for LGMD2i/R9

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A first-in-class CRISPR-CasX gene editor silencing APOC3 transcription for the treatment of Severe Hypertriglyceridemia

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Microglia replacement therapy for CSF1R-related Leukoencephalopathy

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TY1 and Semaglutide to Treat Cardiometabolic HFpEF

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CRISPR/Cas9-mediated gene editing of Hematopoietic stem and progenitor cells for Friedreich’s ataxia

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Continue ReadingCRISPR/Cas9-mediated gene editing of Hematopoietic stem and progenitor cells for Friedreich’s ataxia