CF Foundation Invests Up to $3.5M in Gensaic for Novel Gene Delivery Method

The Cystic Fibrosis Foundation’s investment will go toward conducting preclinical research on a novel gene delivery vehicle. If successful, this gene delivery method could overcome some of the biggest challenges to delivering a gene therapy into the lung cells of people with cystic fibrosis.

| 2 min read

The Cystic Fibrosis Foundation is investing up to $3.5 million in Gensaic Inc. to conduct preclinical research to develop a novel approach to deliver a gene therapy to the lung cells of people with cystic fibrosis. If successful, this gene delivery vehicle would deliver a healthy cystic fibrosis transmembrane conductance regulator (CFTR) gene to lung cells, which would enable the production of functioning CFTR protein.

Gensaic is exploring how to engineer bacteriophage — specialized viruses that typically target bacteria — to deliver the CFTR gene to people with CF. Researchers believe phage-derived particles might be effective because they are unlikely to trigger an immune response, allowing the gene therapy to be re-dosed when needed. Other advantages of this approach include the ability to target specific organs and carry full-length human genes.

The CF Foundation’s investment in this novel approach represents our ongoing efforts to overcome the challenges of delivering gene therapy to lung cells. Delivering DNA molecules into lung cells is a big hurdle because of the body’s natural defenses to block germs and other foreign invaders from entering cells. Mucus and the cell membrane are the two other remaining barriers that make it difficult to deliver DNA to lung cells.

This investment is part of the Foundation’s $500 million Path to a Cure, an ambitious research initiative to accelerate treatments for everyone with CF and ultimately deliver a cure.

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