Gene Therapy for Alpha-1 Anti-Trypsin Deficiency

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• Post published:May 11, 2026
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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:May 11, 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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• Post published:May 11, 2026
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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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• Post published:May 11, 2026
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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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Noncoding RNA drug TY2 for arrhythmogenic cardiomyopathy

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Patient Support Program to Improve Accessibility to Eligible CIRM-Trial Patients

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• Post published:May 11, 2026
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To help improve accessibility to eligible CIRM patients This project will allow patients in underserved communities in California equal access to clinical trials without bias due to financial circumstances.

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