Vertex and CRISPR Therapeutics to collaborate on genetic disease research

Vertex Pharmaceuticals, best known for its breakthrough cystic fibrosis (CF) treatments, is to become one of the first partners of CRISPR Therapeutics, a leader in new gene-editing technology.

The CRISPR/Cas9 technology is among the most exciting developments in biotechnology in recent years, a revolutionary technique which will allow faulty genes linked to any inherited disease to be removed or replaced.

Basel-headquartered CRISPR Therapeutics is just one of several rival groups pioneering the technology, and is in a race to prove it can translate its use into a safe and effective therapy for patients.

The companies say their collaboration will focus on the discovery and development of potential new treatments aimed at the underlying genetic causes of human disease, looking at how CRISPR/Cas9 could work against multiple diseases where targets have been validated through human genetics.

The initial collaboration will focus on mutations and genes linked to CF and sickle cell disease, and Vertex will have exclusive rights to license up to six new CRISPR/Cas9-based treatments that emerge from the collaboration.

Vertex has had success with its CF drugs Kalydeco and the follow-up and combination drug Orkambi, which was approved by the US FDA in July. However, the company is still not making a profit, and is looking to the new alliance to help it develop drugs which can directly target the faulty genes found in CF patients.

The initial focus in CF will be the cystic fibrosis transmembrane conductance regulator (CFTR) gene known to result in the defective protein that causes CF and to edit other genes that contribute to the disease.

Vertex made an up-front commitment of $105 million to CRISPR, including $75 million in cash and a $30 million equity investment. CRISPR is also eligible to receive future milestone and royalty payments.

"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.

"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."

"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 Dr Rodger Novak, 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 programmes."

Vertex will fund 100 per cent of the development expenses of licensed treatments. For each, it will pay future development, regulatory and sales milestones of up to $420 million as well as royalty payments on future sales.

Vertex and CRISPR will also collaborate on the research, development and commercialisation of treatments for haemoglobinopathies. Specifically for haemoglobinopathies, including treatments for sickle cell disease, Vertex and CRISPR will share equally all research and development costs and sales, with CRISPR Therapeutics leading commercialisation efforts in the US. For all other diseases, Vertex will lead all development and global commercialisation activities.

The investment will provide Vertex with an ownership stake in CRISPR. The collaboration also provides Vertex with an observer seat on the CRISPR Board of Directors, which will be filled by Dr Altshuler.

CRISPR rivalry

Dr Emmanuelle Charpentier is the founder of CRISPR Therapeutics, and one of the scientists credited with its discovery. However Charpentier is competing with rivals and former colleagues who have started their own commercial ventures, with a dispute over patents still hanging over the field.

While CRISPR Therapeutics already has financial backing from Celgene, Novartis has a stake in rivals Intellia Therapeutics, and is also funding another firm in the field, Caribou Biosciences.

The most hotly-tipped of the competing biotech firms is Editas, which secured $120 million in private funding. It has financial backing from Google and Bill Gates, but has also signed a development deal with Juno Therapeutics, leaders in another 'next big thing' in biotech, CAR-Ts.

AstraZeneca is the only big pharma company to have announced in-house CRISPR/Cas9 research, and earlier this year claimed its programmes were the most advanced in the sector.