BlueAllele Corporation, a biotechnology company focused on advancing transformational gene correction technologies, announced that the United States Patent and Trademark Office (USPTO) has issued U.S. Patent No. 11,365,407 (the ‘407 Patent) related to its proprietary in vivo gene correction methodologies.
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BlueAllele was founded with the ambitious objective to develop methodologies for single-dose treatments to cure the most debilitating and devastating genetic diseases. Over 5,000 genetic diseases, which are caused by mutations within a gene, have been identified and diagnosed in humans. The mutations can inactivate gene function, referred to as loss-of-function mutations, or create an undesirable function, referred to as gain-of-function mutations. BlueAllele has developed transformational gene correction technologies utilizing its proprietary PALIDON® repair template platforms to achieve gene correction that is precise, durable, and capable of treating a broad range of both loss-of-function and gain-of-function mutations.
Dr. Nicholas Baltes, BlueAllele’s chief scientific officer, said, “We saw that there was a large gap in the capabilities of the genetic tools for gene correction and the development of effective, safe and lifelong therapies. We set out to close the gap.”
Unlike traditional gene therapy where the corrective sequences remain extrachromosomal, PALIDON is integrated into the cell’s DNA thereby providing potential lifelong therapeutic effects. Further, unlike gene knockout approaches where gene function is destroyed, PALIDON provides replacement sequence for restoration of gene function.
The distinctive palindromic design of BlueAllele’s PALIDON repair templates has unlocked the full potential of the cell’s highly active NHEJ pathway. In doing so, PALIDON can increase the efficacy of gene correction events, improve safety and be used for the therapeutic treatment of a wide range of diseases. BlueAllele has expanded upon this foundational feature with the design of additional repair template platforms for treating specific classes of genetic diseases. PALIDON+™ was designed for autosomal dominant diseases and functions to remove unwanted gene products while simultaneously restoring normal protein function. PALIDON DT™ was designed to address repeat expansion diseases and functions to prevent transcription through the expansion.