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

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• Post published:July 20, 2026
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Gene Therapy for Alpha-1 Anti-Trypsin Deficiency

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• Post published:July 20, 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:July 20, 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:July 20, 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:July 20, 2026
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TY1 and Semaglutide to Treat Cardiometabolic HFpEF

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Noncoding RNA drug TY2 for arrhythmogenic cardiomyopathy

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Development of an AAV Epigenetic Gene Therapy for Intractable Chronic Pain Disorders

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Transplantation of human forebrain assembloids as a platform for therapeutic screening in neurodevelopmental disorders

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Translational epigenomics: dissecting cell type-specific function of neuropsychiatric risk genes in vivo

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• Post published:July 20, 2026
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Research Objective Our objective is to enable scalable genetic screening to study how different cell types and epigenetic networks are impacted by risk genes implicated in human psychiatric disorders. Impact…

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