Specific Biologics Inc. an early-stage biotechnology company developing novel gene editing technologies, announced that it will receive more than US$527,000 to support the preclinical development of its Dualase™ gene editing platform to target a set of cystic fibrosis -causing nonsense mutations where patients currently have fewer therapeutic options available.
"Incredible advancements in the treatment of cystic fibrosis have been made since the identification of genetic mutations in the CFTR gene as the cause of CF Recent developments in gene editing technologies are creating the potential to correct these causative mutations at their source. We are truly grateful for the support of this Cystic Fibrosis Foundation award which will allow us to apply our Dualase™ gene editing technology to several frequent CFTR nonsense mutations."
Brent Stead, CEO of Specific Biologics Inc
Dualase™ cuts DNA differently than existing gene editors. This feature enables the high frequency insertion of new sequences into precise locations in the human genome. One can think of gene editing like a word processor that can find a specific spelling mistake in billions of letters and replace it with a correction. Similarly, inside a cell, Dualase™ can find a specific spelling mistake in the genome and replace it with the corrected sequence. Specific will deliver its gene editing technology using an inhalable lipid nanoparticle carrier, which will aid in delivering the gene editing machinery inside the cells of the lung. It is believed that if the CFTR nonsense mutations are corrected in enough cells, a gene editing therapeutic could provide a long-lasting benefit for CF patients.
The award will help fund preclinical testing in disease relevant models.
About Specific Biologics Inc.
Specific Biologics Inc. is a venture-backed early-stage biotechnology company on a mission to develop novel gene editing technologies to treat diseases through precision gene editing. Our two-site Dualase™ platform gene editors cut DNA in a way that optimally exploits the cell's naturally occurring DNA repair pathways. This enables two gene editing outcomes, precise DNA deletions to disrupt genes or increased repair to correct genes. Specific also develops lipid nanoparticles to deliver the gene editor to target cells and is developing a pipeline of Dualase™-based therapeutics in areas of high unmet medical need.