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RAFT: A CRISPR-Free RNA Trans-Splicing Platform for MYH7 Correction in Hypertrophic Cardiomyopathy
Unmet Need Hypertrophic cardiomyopathy (HCM) is a common inherited heart disease affecting 1 in 500 individuals, frequently caused by mutations in the MYH7 gene. Current gene therapy approaches, including AAV-based gene replacement, face significant challenges…
Methods and compositions for enhancing viral and mRNA gene therapy with engineered RNA elements
Unmet Need An outstanding challenge in creating effective gene therapies, particularly those based on adeno-associated virus (AAV) and mRNA delivery platforms, is overcoming sub-optimal transgene expression. Inefficiencies in translation and stability of therapeutic gene products…
CRISPR-based antiviral treatment of coronavirus infections
Unmet Need The global market for antiviral drugs is a large one, topping $60B, and is getting larger due to the ongoing COVID-19 pandemic. Spurred on by a $3B investment from the U.S. government into…