Editing Technology to Identify and Advance Genetic Dysfunction Therapies

BlueAllele | July 27, 2020

BlueAllele, a biotechnology company focused on enhancing the quality and precision of gene editing systems for patients with genetic diseases, announced the launch of PALIDON™, an innovative and proprietary repair template technology. Many rare genetic diseases are caused by mutations within a single gene. However, the location of the mutation often varies for different individuals with the same disease. Conventional gene editing approaches are primarily limited to correcting small regions or even single locations. Additionally, repair templates have failed to achieve sufficient integration efficiency in vivo needed to sustain therapeutic thresholds and durable efficacy. BlueAllele's PALIDON repair templates were designed to specifically overcome both of these challenges. PALIDON's "bi-directional" templates use a reverse-complementary structure that is designed to leverage the high rate of non-homologous end joining (NHEJ) repair observed in cells while preserving the ability to achieve precise correction of damaged mRNA and protein. PALIDON templates are designed to be compatible with existing gene editing tools (e.g., CRISPR, zinc-finger nucleases) and delivery systems, making them particularly suitable for targeting a variety of genetic diseases.

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