BlueAllele Launches PALIDON™ Gene Editing Technology to Discover and Develop Therapies for Genetic Diseases
OAKDALE, Minn., July 24, 2020 /PRNewswire/ -- 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.
BlueAllele is advancing several research programs with its proprietary PALIDON template design as the cornerstone of its approach for in vivo gene correction.
"PALIDON is a disruptive technology that will enhance the overall efficacy and utility of treatment approaches for patients with genetic diseases," said Joseph B. Saluri, CEO of BlueAllele. "Its compatibility with existing gene editing tools and delivery systems will facilitate collaboration efforts and accelerate development."
Dr. Nicholas Baltes, chief science officer of BlueAllele, added, "Editing efficiency is one of the biggest challenges for in vivo therapies. PALIDON is designed to address this problem by allowing for correction of defective mRNA and protein sequences, regardless of whether the NHEJ pathway chooses to integrate PALIDON in the forward or reverse direction. Additionally, the unique design provides coverage for multiple mutation locations, thus addressing a broader patient population than other approaches."
BlueAllele is a biotechnology company committed to advancing the quality and precision of gene editing systems for patients with genetic diseases. BlueAllele is developing novel and efficient therapeutics for patients with genetic diseases caused by loss-of-function or gain-of-function mutations. Learn more: www.blueallele.com.
SOURCE BlueAllele
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