Advancing Next-Gen Gene Editing to Combat Genetic Diseases

February 22, 2024 / New Investments

Gene editing technology has the potential to revolutionize the treatment of genetic diseases by directly altering DNA within cells. However, current methods face significant limitations, including low on-target editing efficiency and unintended off-target effects. These challenges underscore the urgent need for novel approaches that can overcome these barriers and more precisely target the underlying causes of genetic diseases.

“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. “Data suggest that Dualase™ has the potential to overcome these well-known challenges in relevant therapeutic indications.” 


About Specific Biologics 

Specific Biologics is transforming gene editing with its next-generation Dualase™ platform, offering a more precise method for DNA modification. Unlike conventional gene editors that cut DNA with compatible ends, Dualase™ utilizes a dual-site cutting mechanism that generates non-compatible DNA ends. This innovation influences the cellular repair process, enabling defined-length deletions and increased repair frequency, which improves editing accuracy and efficiency.

The key advantages of Dualase™ include:

  1. Dual-site DNA cutting mechanism
  2. Creation of non-compatible DNA ends
  3. Enhanced control over cellular repair processes
  4. Ability to introduce defined-length deletions or increase repair frequency

The platform’s first therapeutic application focuses on cystic fibrosis-causing nonsense mutations, offering a more effective and lasting treatment for CF patients. Delivered via an inhalable lipid nanoparticle carrier, Dualase™ aims to correct CFTR mutations in lung cells, providing new hope for patients with limited therapeutic options [1]. “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.


Why Lumira Invested

For Lumira, investing in Specific Biologics represents an opportunity to support next-generation gene editing technology with the potential to deliver transformative treatments. This investment also supports Canadian health science innovators at the forefront of medical advancements. By addressing the limitations of existing gene editing methods, Dualase™ offers a promising solution to genetic diseases once thought to be untreatable.

“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,” said Nikhil Thatte, Principal at Lumira Ventures. “We are excited to work alongside adMare and the Specific team to accelerate the development of their groundbreaking platform and bring transformative therapies to patients.”

“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.” said Dr. Brent Stead, CEO of Specific Biologics.


Looking Ahead: A New Era for Gene Editing

As Specific Biologics continues to advance its Dualase™ platform, it is set to redefine the future of genetic disease treatment. The platform’s ability to introduce precise DNA modifications offers the promise of long-lasting therapies that go beyond symptom management, targeting the root causes of genetic disorders. Lumira Ventures is proud to support Specific Biologics in this groundbreaking work. Together, we are paving the way for next-generation gene editing therapies that could redefine patient care and revolutionize the treatment of genetic diseases.

“By advancing Dualase™, we are looking at a future where genetic diseases no longer have to be managed symptomatically. Instead, we can target the root causes with precision and efficacy,” said Dr. Brent Stead, CEO of Specific Biologics. “Our team is excited to continue our work and expand the range of conditions we can address with this platform, ultimately transforming the lives of millions of patients.”

Learn more about Specific Biologics 


 

Sources:

[1] Source: Specific Biologics, www.specificbiologics.com, 2024

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