Specific Biologics Inc. to Present Dualase® Genome Editing for the Precise Removal of Pathogenic Repeats in Repeat Expansion Disorders at the 28th Annual American Society for Gene and Cell Therapy...
TORONTO, May 6, 2025 /PRNewswire/ - Specific Biologics Inc., a biotechnology company advancing industry leading genome editing therapies, today announced it will present new preclinical data at the 28th Annual Meeting of the American Society for Gene and Cell Therapy (ASGCT), taking place May 13-17, 2025 in New Orleans, LA.
The presentation will highlight the company's proprietary Dualase® genome editing platform and its therapeutic potential in repeat expansion disorders, a class of severe neurological and neuromuscular genetic diseases driven by abnormally expanded DNA sequences. These include disorders such as amyotrophic lateral sclerosis (ALS), Huntington's disease, Fragile X syndrome, and myotonic dystrophy, for which there are limited therapeutic options for patients and currently no curative treatments.
Presentation Details:
Title: Precise Removal of a Large Pathogenic Repeat Expansion In Vitro and In Vivo Using a Dual-guided TevCas9 (Dualase®) Genome Editor Encoded in a Single AAV
Presenter: Dr. Brent Stead, CEO
Date & Time:May 15th, 2025 - 1:45 PM Central Time
Location: NOLA Theater B, Abstract 185
"Our approach is designed to precisely and durably correct the root cause of repeat expansion disorders by excising pathogenic repeat DNA sequences and leave a non-pathogenic number of repeats with no detectable off targets," said Dr. Brent Stead, Chief Executive Officer of Specific Biologics Inc. "The disease-relevant cell and animal model data demonstrating precise repeat removal we will present is a first to our knowledge and marks a significant milestone in our mission to develop transformative, potential one-time treatments for patients who currently face progressive and devastating disease. ASGCT is the ideal venue to share our progress with the broader scientific and medical communities."
Dualase® genome editors use a unique two-site mechanism to seamlessly remove, repair, or insert small or large sequences. Pathogenic repeat expansion lengths vary between patients and even within individual patients. The two-site mechanism allows Dualase® editing domains to be positioned within the repeat on either side of the expansion, enabling removal of the large pathogenic sequence, while preserving a non-pathogenic length of repeats for a highly differentiated therapeutic approach. With a compact coding size of 3.7 kilobases, Dualase® editors can be packaged into clinically and commercially validated delivery systems, including single adeno-associated viral (AAV) vectors, enabling delivery to traditionally hard-to-reach tissues like the central nervous system.
The full abstract is available through the ASGCT website and will be accessible to conference attendees throughout the event.
About Specific Biologics Inc.
Specific Biologics is a venture-backed biotechnology company developing Dualase®, an industry-leading genome editing platform for precise, efficient, and programmable in vivo therapeutics. Dualase® achieves best-in-class accurate editing efficiency with undetectable off-target effects as demonstrated in preclinical cell and animal models at diverse targets and indications. Its unique two-site mechanism enables the seamless removal, repair, or insertion of both small and large DNA sequences, offering broad therapeutic potential. Specific Biologics has a focused pipeline of development candidates where Dualase® has a differentiated mechanism of action, coupled with single AAV or all-RNA delivery, with an initial focus on repeat expansion disorders.
For more information, visit www.specificbiologics.com or follow Specific Biologics Inc. on LinkedIn at https://www.linkedin.com/company/specific-biologics
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