Vertex and CRISPR Therapeutics Partner to Advance Gene Therapies for Cystic Fibrosis, Sickle Cell

Vertex and CRISPR Therapeutics Partner to Advance Gene Therapies for Cystic Fibrosis, Sickle Cell

Vertex Pharmaceuticals, Inc., and CRISPR Therapeutics announced a new strategic research partnership focused on utilizing the CRISPR-Cas9 gene editing platform in the potential discovery and development of new gene therapies. The companies plan to focus initial research efforts on the mutations and genes known to cause cystic fibrosis, such as the abnormal cystic fibrosis transmembrane conductance regulator (CFTR) gene, and those behind sickle cell disease. Later goals are to evaluate the platform across multiple diseases known to have genetic targets.

“CRISPR-Cas9 is an important scientific and technological breakthrough that holds significant promise for the future discovery of potentially transformative treatments for many genetic diseases,” said David Altshuler, MD, PhD, Vertex’s Executive Vice President, Global Research and Chief Scientific Officer, in a press release. “As a company founded on innovative science, we’re excited to begin this collaboration with CRISPR, as it puts us at the forefront of what we believe may be a fundamental change in the future treatment of disease — using gene editing technologies to address the underlying genetic causes of many diseases.”

Under the partnership, Vertex has exclusive rights to license up to six novel treatments using the CRISPR-Cas9 that emerge  from the collaborative efforts, and it made an upfront commitment to CRISPR of $105 million, inclusive of a $75 million cash payment and a $30 million equity investment. CRISPR also remains eligible for future development, regulatory and sales milestones, and royalty payments.

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Vertex’s move to partner with CRISPR positions the company to expand upon its proven leadership in the development of effective CF therapies, which already include the FDA-approved Kalydeco (ivacaftor) and the approved landmark combination therapy Orkambi (lumacaftor/ivacaftor).

“Vertex has a track record of developing innovative medicines for cystic fibrosis and other serious diseases, making them a great partner to accelerate the therapeutic promise of gene editing,” said Rodger Novak, MD, Chief Executive Officer of CRISPR Therapeutics. “For CRISPR, this collaboration validates the potential for gene editing in human therapeutics and provides important financial support for continued investment in our platform and proprietary pipeline of programs.”

Vertex will fund 100 percent of the development expenses of its six potential licensed treatments. For hemoglobinopathies, such as sickle cell disease, Vertex and CRISPR will equally share all research and development costs and sales. CRISPR will lead commercialization efforts in the U.S regarding this disease; Vertex will lead development and global commercialization activities for all other diseases that arise from their collaboration.

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