Specific Biologics Secures $35 Million in Funding to Advance ALS Gene-Editing Therapy
Specific Biologics, a biotech firm at the forefront of precision gene-editing, has recently announced the completion of an oversubscribed Series A financing round, securing an impressive $35 million. This significant investment was primarily led by Hatteras Venture Partners, along with foundational supporters like Lumira Ventures and adMare BioInnovations. New investors such as Genesys Capital, the Business Development Bank of Canada's Life Sciences Venture Fund, DeepWork Capital, and Seido Capital also participated in this funding round.
The capital raised will be instrumental in progressing Specific Biologics' flagship therapeutic candidate, Dualase®, through its initial clinical biomarker proof-of-concept phase, which will be conducted within an adaptive Phase I/II clinical trial targeting patients suffering from C9ORF72-associated amyotrophic lateral sclerosis (ALS). ALS is a neurodegenerative disorder that currently lacks any approved treatments, underscoring the urgency behind this initiative.
Brent Stead, CEO of Specific Biologics, articulated the importance of this funding, stating, ‘This financing represents a defining step toward our goal of bringing a potentially transformative medicine to people living with ALS.’ He emphasized the necessity of addressing this debilitating disease at its genetic source, providing hope to countless individuals who have faced the challenges of ALS.
The innovative gene-editing approach proposed by Specific Biologics has the potential to reshape the treatment landscape for genetic disorders. Ben Scruggs, a partner at Hatteras Venture Partners, pointed out the significant challenges that have affected the development of gene editing technologies, such as precision and delivery methods. The proprietary Dualase® platform aims to distinctly tackle these issues by removing deleterious DNA repeat expansions while maintaining the integrity of necessary biological sequences.
Moreover, Nikhil Thatte from Lumira Ventures voiced his strong belief in the scientific innovation and clinical prospects of the Dualase® platform, noting that early clinical proof-of-concept in ALS patients marks a critical turning point for the company, setting the stage for realizing the full potential of its technology.
The Dualase® platform, which utilizes an efficient two-site gene editing mechanism, is specifically designed to address repeat DNA expansions linked to more than 40 genetic disorders. This technology has yielded promising preclinical results, achieving substantial edits in neuromuscular disorder models.
ALS, known for its progressive and fatal nature, results from the degeneration of motor neurons, leading to critical loss of voluntary muscle control. The most commonly inherited genetic cause of ALS involves repeat expansions in the C9ORF72 gene, which are also connected to frontotemporal dementia. Specific Biologics’ targeted approach through Dualase® aims to shorten these pathogenic expansions directly within the cell, potentially delivering lasting therapeutic benefits by addressing the genetic cause rather than merely alleviating symptoms.
In preclinical models, Dualase® demonstrated remarkable effectiveness, collapsing nearly all pathogenic repeat expansions in ALS patient-derived cell lines and significantly impacting relevant animal models. The approach yielded no detectable off-target effects, making it a highly promising candidate for future therapies against ALS.
As Specific Biologics embarks on this ambitious journey, the backing of notable investors and a focus on groundbreaking science signifies their commitment to redefining the future of gene therapy – offering hope to patients facing challenges posed by inherited genetic disorders, particularly ALS. For more information about Specific Biologics and their innovations, visit Specific Biologics Inc. or follow them on LinkedIn.
The capital raised will be instrumental in progressing Specific Biologics' flagship therapeutic candidate, Dualase®, through its initial clinical biomarker proof-of-concept phase, which will be conducted within an adaptive Phase I/II clinical trial targeting patients suffering from C9ORF72-associated amyotrophic lateral sclerosis (ALS). ALS is a neurodegenerative disorder that currently lacks any approved treatments, underscoring the urgency behind this initiative.
Brent Stead, CEO of Specific Biologics, articulated the importance of this funding, stating, ‘This financing represents a defining step toward our goal of bringing a potentially transformative medicine to people living with ALS.’ He emphasized the necessity of addressing this debilitating disease at its genetic source, providing hope to countless individuals who have faced the challenges of ALS.
The innovative gene-editing approach proposed by Specific Biologics has the potential to reshape the treatment landscape for genetic disorders. Ben Scruggs, a partner at Hatteras Venture Partners, pointed out the significant challenges that have affected the development of gene editing technologies, such as precision and delivery methods. The proprietary Dualase® platform aims to distinctly tackle these issues by removing deleterious DNA repeat expansions while maintaining the integrity of necessary biological sequences.
Moreover, Nikhil Thatte from Lumira Ventures voiced his strong belief in the scientific innovation and clinical prospects of the Dualase® platform, noting that early clinical proof-of-concept in ALS patients marks a critical turning point for the company, setting the stage for realizing the full potential of its technology.
The Dualase® platform, which utilizes an efficient two-site gene editing mechanism, is specifically designed to address repeat DNA expansions linked to more than 40 genetic disorders. This technology has yielded promising preclinical results, achieving substantial edits in neuromuscular disorder models.
ALS, known for its progressive and fatal nature, results from the degeneration of motor neurons, leading to critical loss of voluntary muscle control. The most commonly inherited genetic cause of ALS involves repeat expansions in the C9ORF72 gene, which are also connected to frontotemporal dementia. Specific Biologics’ targeted approach through Dualase® aims to shorten these pathogenic expansions directly within the cell, potentially delivering lasting therapeutic benefits by addressing the genetic cause rather than merely alleviating symptoms.
In preclinical models, Dualase® demonstrated remarkable effectiveness, collapsing nearly all pathogenic repeat expansions in ALS patient-derived cell lines and significantly impacting relevant animal models. The approach yielded no detectable off-target effects, making it a highly promising candidate for future therapies against ALS.
As Specific Biologics embarks on this ambitious journey, the backing of notable investors and a focus on groundbreaking science signifies their commitment to redefining the future of gene therapy – offering hope to patients facing challenges posed by inherited genetic disorders, particularly ALS. For more information about Specific Biologics and their innovations, visit Specific Biologics Inc. or follow them on LinkedIn.
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