WALTHAM, Mass.– SeQure Dx, a cutting-edge gene editing diagnostics company, has announced its emergence from stealth mode with an aim to partner with biopharma companies developing gene editing therapeutics. The company is armed with technology discovered and developed by the Joung Lab at Massachusetts General Hospital and Harvard Medical School and funding from a $17.5M Series A raised in 2021. SeQure Dx’s platform comprises the only scalable, population-based and editing technology-agnostic in vitro off-target evaluation method complemented by orthogonal methods required for off-target risk validation.
“I am very excited for SeQure Dx to bring the ONE-seq technology to market to support the emerging and dynamic gene editing therapeutics space,” said Dr. Joung, a co-founder of SeQure Dx and Chair of its Scientific Advisory Board as well as the Robert B. Colvin, M.D. Endowed Chair in Pathology at Mass General Hospital. “Evaluating potential off-target edits created by these therapies plays an enormous role in the advancement of safe and effective gene editing. It’s crucial to define any potential off-target edits from program discovery to eventual patient treatment, and SeQure Dx’s comprehensive assay and data solutions does this using state-of-the-art methods.”
The last decade since the characterization of CRISPR has seen immense optimism surround the clinical potential of gene editing therapy as well as the emergence of dozens of companies focused on bringing a variety of gene editing approaches into the clinic. Quickly, the optimism was tempered by the discovery that off target editing is a companion effect for all the gene editing methods conceived so far. The ever-looming potential off target effects drove a critical need to identify and characterize these impacts to advance.
The FDA has confirmed its off-target concern with the March 2022 release of draft guidance highlighting the need for comprehensive off-target evaluation, placing an emphasis on frequency, as well as potential off-target types and site location. As part of this guidance, the FDA recommends using off-target risk assessment methods that provide sufficient sensitivity along with multiple cell donor analyses to ensure a higher confidence level. SeQure Dx’s proprietary technologies, under license from MGH, position the company to be the gold standard in demonstrating therapeutic safety throughout research, development, and commercialization, meeting the FDA’s standard proclaimed in the draft guidance.
The company’s core mission is to leverage its cutting-edge platform to partner with biopharma in a way that allows the gene editing developers to solely focus on the advancement of their programs and outsource the entirety of the off-target evaluation workstream to SeQure Dx. SeQure Dx’s offerings span the drug development lifecycle, providing value for its partners preclinically, during clinical development and throughout future commercialization via rigorous IND-enabling data package(s), ensuring patient safety from a trial enrollment and follow-up perspective, and clinical decision support and long-term safety monitoring, respectively.
