IND enabling – Page 2

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

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Post published:June 21, 2026
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Microglia replacement therapy for CSF1R-related Leukoencephalopathy

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Post published:June 21, 2026
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TY1 and Semaglutide to Treat Cardiometabolic HFpEF

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Post published:June 21, 2026
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CRISPR/Cas9-mediated gene editing of Hematopoietic stem and progenitor cells for Friedreich’s ataxia

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Post published:June 21, 2026
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Noncoding RNA drug TY2 for arrhythmogenic cardiomyopathy

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Post published:June 21, 2026
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A Translating Center designed to expedite the development and delivery of high quality stem cell therapies to patients

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Post published:June 21, 2026
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A successful partnership, resulting in the creation of a Translating Center recognized as the leader in providing end-to-end preclinical development services (preclinical research, CMC, regulatory) to accelerate the availability of…

Advancing a novel antisense oligonucleotide for the treatment of SCA3, a devastating neurodegenerative disease

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Post published:June 21, 2026
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Expression of Ube3a by the hematopoietic system for the treatment of Angelman syndrome

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Post published:June 21, 2026
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Development of a novel intravitreal AAV gene therapy for the rare disease blue cone monochromacy.

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Post published:June 21, 2026
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Novel Gene Therapy Targeting Multiple Pathological Drivers of Desmoplakin Associated Arrhythmogenic Cardiomyopathy

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Post published:June 21, 2026
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Therapeutic Candidate or Device RJB-0402 is a liver-targeting adeno-associated virus vector-based gene therapy that drives over expression of FGF21. Indication Desmoplakin-related arrhythmogenic cardiomyopathy (DSP ACM) at high risk of life-threatening…