Advancing Next-Gen Gene Editing to Combat Genetic Diseases

February 22, 2024 / New Investments

Gene editing holds incredible potential to cure a wide range of genetic diseases by directly altering DNA in the genome inside cells. Yet, today’s gene editors remain facing many limitations. “Current gene editing technologies are constrained by low on-target editing efficiency and high unintended off-target effects,” noted Dr. Daniel Hetu, Managing Director at Lumira Ventures. The team at Specific Biologics is shifting this paradigm. Through the development of their novel gene editing therapies, the company is breaking down barriers to better realize the significant opportunity of this therapeutic approach and optimize its application across new therapeutic areas. “Data suggest that Dualase™ has the potential to overcome these well-known challenges in relevant therapeutic indications,” mentioned Dr. Hetu.

Specific Biologics, an early-stage biotechnology company has developed a unique next-generation Dualase™ gene editing platform that can more accurately alter DNA sequences by cutting DNA differently than current editors. The platform gene editing technology cuts DNA twice and leaves non-compatible DNA ends. Other gene editors leave compatible DNA ends when they cut DNA. When a cell repairs compatible ends, it leads to random insertions or deletions or a low frequency of repair, reducing efficiency and target effects. Dualase™ by creating non-compatible ends influences cellular repair and depending on the presence or absence of template DNA, the cell may introduce defined length deletions or increase the frequency of repair. This step is critical to the company’s mission as defined length deletions & increased repair allow targeting of genetic diseases that could not be targeted before.

“Based on our early work with Dualase™, we believe it will have broad utility across a number of diseases with high unmet need,” said Dr. Brent Stead, CEO of Specific Biologics Inc. “This investment by Lumira and adMare, two recognized leaders in life sciences, will help accelerate the advancement of our promising platform, and a pipeline of differentiated Dualase™-based gene editing therapeutics in a diverse range of indications.”

Initial indications for Dualase™ were set to target a set of cystic fibrosis-causing nonsense mutations where patients currently have fewer therapeutic options available. By delivering the gene editing technology using an inhalable lipid nanoparticle carrier, the treatment will aid in delivering the gene editing machinery inside the cells of the lung. Following, if the CFTR nonsense mutations are corrected in enough cells, the gene editing therapeutic will provide a more effective treatment option with long-lasting benefits for CF patients.

The company is on a mission and it does not stop at cystic fibrosis. The promising breadth of the platform and widespread indications allow the company to expand its research and build a pipeline of therapeutics based on Dualase, offering hope to treat many other genetic diseases with no current therapies.

“Built on the work of Dr. David Edgell at Western University’s Schulich School of Medicine & Dentistry, Specific Biologics represents another example of how Canadian health science innovators are at the leading edge of treatment modalities,” added Nikhil Thatte, Principal at Lumira Ventures. “We are excited to work alongside adMare and the Specific team to grow the company and bring transformative therapies to patients.”

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