Vertex Joins with Affinia Therapeutics to Develop Gene Therapies for CF

Vertex Pharmaceuticals announced a multiyear partnership with Affinia Therapeutics to develop gene therapies for cystic fibrosis (CF) and other genetic disorders.

Gene therapy seeks to replace a malfunctioning gene with a working version. In people with CF, in particular, a gene therapy would deliver a healthy copy of the CFTR gene to replace the faulty copy that causes the disease.

These therapies commonly use engineered viruses, referred to as vectors, to serve essentially as transport agents. Viruses are useful for this purpose because they have evolved to be able to inject genetic material (DNA or RNA) into cells — the goal of a gene therapy.

Affinia specializes in the development of viral vectors using engineered adeno-associated virus (AAV). This virus has a relatively simple structure, making it well-suited to adapt for gene therapy purposes. AAV also is not known to cause disease in people, which is important from a safety perspective.

Under the terms of the agreement, Affinia will apply its AAV technology toward improving Vertex’s gene therapy efforts, with a particular focus on cystic fibrosis, Duchenne muscular dystrophy, and myotonic dystrophy type 1.

Affinia will be responsible for designing the viral protein shells (capsids) that will be used in these gene therapies. The company will be given $80 million in upfront payments and research milestones, and be eligible to receive more than $1.6 billion based on future development milestones.

“This collaboration with Affinia Therapeutics will enhance our existing capabilities in discovering and developing transformative therapies for people with serious diseases,” Bastiano Sanna, executive vice president and chief of cell and genetic therapies at Vertex, said in a press release.

“Affinia Therapeutics’ innovative approach to the discovery and design of AAV capsids brings yet another tool to our Vertex Cell and Genetic Therapies toolkit, and we’re excited to partner with them to bring together their technology platform with our research and development expertise,” Sanna said.

Vertex will fund the development of these gene therapies, including preclinical and clinical development efforts, and be responsible for the commercialization of any approved products that may result.

“Vertex is an established leader in developing transformative medicines for genetic diseases and renowned for its scientific rigor,” said Rick Modi, Affinia’s CEO. “We are thankful for the scientific validation this partnership brings, and look forward to working closely with them to advance life-changing, differentiated genetic therapies and make a meaningful difference to those affected by these diseases,” Modi said.

Vertex also is collaborating with CRISPR Therapeutics on gene-editing treatments for blood disorders such as sickle cell disease.