Key Takeaways
- • Specific Biologics completed an oversubscribed $35 million Series A financing led by Hatteras Venture Partners to advance its precision gene-editing therapy for ALS.
- • Existing investors Lumira Ventures and adMare BioInnovations participated alongside new investors Genesys Capital, BDC's Life Sciences Venture Fund, DeepWork Capital, and Seido Capital.
- • The lead candidate, built on the proprietary Dualase platform, targets ALS associated with C9ORF72 gene mutations and is slated to begin an adaptive Phase I/II trial.
- • Dualase is designed to remove abnormal DNA repeat expansions while preserving normal sequences, a mechanism linked to more than 40 genetic disorders.
- • The program remains investigational, and its safety and effectiveness have not yet been established in patients.
Summary
Specific Biologics, a biotechnology company developing precision gene-editing therapies, has closed an oversubscribed $35 million Series A round led by Hatteras Venture Partners, according to the company's announcement as reported by Pulse 2.0. Existing backers Lumira Ventures and adMare BioInnovations joined the round, alongside new investors Genesys Capital, BDC's Life Sciences Venture Fund, DeepWork Capital, and Seido Capital.
The proceeds will fund clinical development of the company's lead therapeutic candidate, which uses its proprietary Dualase gene-editing technology. The company is initially targeting amyotrophic lateral sclerosis (ALS) associated with mutations in the C9ORF72 gene, and plans to advance the candidate through early clinical biomarker proof-of-concept in the first patient cohort of an adaptive Phase I/II trial.
Dualase is designed to excise abnormal DNA repeat expansions while preserving the normal genetic sequences required for biological function. Such repeat expansions are associated with more than 40 genetic disorders, suggesting potential applications beyond the ALS program. Specific Biologics has demonstrated gene-editing activity in preclinical studies involving multiple neuromuscular repeat expansion conditions. The company aims to address certain genetic causes of disease directly rather than only managing symptoms. The clinical program remains investigational, and its safety and effectiveness have not yet been established in patients.
CEO Brent Stead described the financing as "a defining step toward our goal of bringing a potentially transformative medicine to people living with ALS." Ben Scruggs, partner at Hatteras Venture Partners, cited the platform's potential to overcome longstanding challenges in gene editing related to precision, specificity and delivery. Nikhil Thatte, partner at Lumira Ventures, said the firm's additional investment reflects growing conviction in the platform's scientific differentiation and called early clinical proof-of-concept in ALS patients an important inflection point.
The proceeds will fund clinical development of the company's lead therapeutic candidate, which uses its proprietary Dualase gene-editing technology. The company is initially targeting amyotrophic lateral sclerosis (ALS) associated with mutations in the C9ORF72 gene, and plans to advance the candidate through early clinical biomarker proof-of-concept in the first patient cohort of an adaptive Phase I/II trial.
Dualase is designed to excise abnormal DNA repeat expansions while preserving the normal genetic sequences required for biological function. Such repeat expansions are associated with more than 40 genetic disorders, suggesting potential applications beyond the ALS program. Specific Biologics has demonstrated gene-editing activity in preclinical studies involving multiple neuromuscular repeat expansion conditions. The company aims to address certain genetic causes of disease directly rather than only managing symptoms. The clinical program remains investigational, and its safety and effectiveness have not yet been established in patients.
CEO Brent Stead described the financing as "a defining step toward our goal of bringing a potentially transformative medicine to people living with ALS." Ben Scruggs, partner at Hatteras Venture Partners, cited the platform's potential to overcome longstanding challenges in gene editing related to precision, specificity and delivery. Nikhil Thatte, partner at Lumira Ventures, said the firm's additional investment reflects growing conviction in the platform's scientific differentiation and called early clinical proof-of-concept in ALS patients an important inflection point.
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