Minnesotta Patent of the Month – September 2024
BlueAllele’s groundbreaking gene therapy approach introduces a novel solution to overcome limitations in treating monogenic disorders. Their innovative vector technology enables precise gene editing by delivering a transgene that mimics the coding sequences of the body’s endogenous Factor VIII gene. This vector is designed to correct mutations at the 3′ end of genes, allowing for the effective treatment of disorders like spinocerebellar ataxia, caused by gain-of-function mutations.
One of the major challenges in gene therapy is the delivery of large functional genes, which often exceed the size limits of traditional viral vectors. BlueAllele’s invention overcomes this by splitting the gene into two coding sequences, both encoding the same essential protein, Factor VIII, but through slightly different nucleotide sequences. These sequences are strategically packed within a viral vector, allowing efficient delivery and integration without compromising therapeutic efficacy.
The versatility of this technology is further enhanced by its compatibility with multiple integration pathways, including homologous recombination (HR) and non-homologous end joining (NHEJ), making it adaptable to various gene repair mechanisms. This approach not only ensures high precision but also reduces the risk of off-target effects or incomplete gene correction.
This breakthrough offers hope for tackling genetic diseases that were previously difficult to address due to their complex mutations and large gene size. With its capacity to precisely target and edit defective genes, BlueAllele’s invention represents a significant leap forward in gene therapy, opening new doors for treating conditions like hemophilia, spinocerebellar ataxia, and other monogenic disorders, with the potential to transform patient outcomes.
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