Specific Biologics Inc. Unveils Dualase Genome Editing at ASGCT Meeting

Specific Biologics Inc. Set to Showcase Dualase® Genome Editing Innovations

Specific Biologics Inc., a pioneer in genome editing solutions, has announced its presentation of new preclinical data regarding its proprietary Dualase® platform at the upcoming 28th Annual Meeting of the American Society for Gene and Cell Therapy (ASGCT) in New Orleans, Louisiana, scheduled for May 15, 2025. This significant event in the biotechnology sector will spotlight the latest advancements in genome editing, specifically focusing on the precise removal of pathogenic repeats that characterize several severe neurological and neuromuscular disorders.

What is Dualase®?

Dualase® is a state-of-the-art genome editing technology developed by Specific Biologics that offers a targeted approach to treating repeat expansion disorders. These disorders include debilitating conditions such as amyotrophic lateral sclerosis (ALS), Huntington's disease, Fragile X syndrome, and myotonic dystrophy. Currently, these conditions lack effective therapeutic options, making innovative approaches like Dualase® more crucial than ever.

The platform's distinct mechanism empowers it to excise pathogenic DNA sequences accurately while leaving a functional residue, which is vital for maintaining cellular health. This innovative editing technique is designed to effectively tackle the core issues of genetic conditions caused by abnormal DNA expansions, potentially transforming patient outcomes in the future.

Presentation Highlights

The presentation titled "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", will be delivered by Dr. Brent Stead, CEO of Specific Biologics Inc. on May 15, 2025, at 1:45 PM Central Time, in NOLA Theater B. This session will showcase the company’s groundbreaking findings that reveal the dual capabilities of Dualase® in both working with cellular and animal models.

Dr. Stead emphasizes the critical importance of this research, stating, "Our approach is designed to precisely and durably correct the root cause of repeat expansion disorders by excising pathogenic repeat DNA sequences and leaving a non-pathogenic number of repeats with no detectable off-target effects." This represents an essential milestone in the journey toward developing potential one-time transformative therapies for patients suffering from these progressive conditions.

The unique two-site mechanism employed by Dualase® editors facilitates the removal, repair, or insertion of genetic material, whether large or small. This flexibility is extremely beneficial, as the pathogenic expansions can vary not only across different patients but also within the same individual, necessitating a highly adaptable tool for effective treatment.

Moreover, Dualase® has a compact coding size of 3.7 kilobases, positioning it well for integration within clinically validated delivery systems such as adeno-associated viral (AAV) vectors. This ensures that the treatment can reach traditionally hard-to-access tissues, such as those in the central nervous system, where many of these conditions manifest.

Future Implications

As Dr. Stead notes, sharing this data at ASGCT will allow Specific Biologics to engage with a community committed to advancing scientific understanding and therapeutic innovations. He believes that the insights gained from their research hold great promise for transforming the landscape of genetic disorder treatments, potentially offering hope to countless patients who currently face limited options.

Specific Biologics Inc. is indeed poised to influence the future of genetic therapy with its Dualase® platform, focusing on diseases that have long been underserved. As advancements in biotechnology continue, the industry looks forward to the breakthroughs that companies like Specific Biologics may deliver, ultimately striving towards eradicating these genetic disorders altogether.

For more information, visit Specific Biologics or connect with them on LinkedIn.