The History of Casgevy
Surveying great inventors and businesses
Axial: https://linktr.ee/axialxyz
Axial partners with great founders and inventors. We invest in early-stage life sciences companies such as Appia Bio, Seranova Bio, Delix Therapeutics, Simcha Therapeutics, among others often when they are no more than an idea. We are fanatical about helping the rare inventor who is compelled to build their own enduring business. If you or someone you know has a great idea or company in life sciences, Axial would be excited to get to know you and possibly invest in your vision and company. We are excited to be in business with you — email us at info@axialvc.com
The story of Casgevy began in 2012 with the revolutionary discovery of the CRISPR-Cas9 gene editing system, which allows precise editing of DNA. Researchers quickly realized CRISPR could be harnessed to correct disease-causing mutations.
In 2013, CRISPR Therapeutics was founded to translate CRISPR into medicines. The company decided early to tackle blood disorders like sickle cell disease. They chose sickle cell for its well understood genetics and because editing blood stem cells ex vivo (outside the body) could reduce risk.
Over 2014-2016, CRISPR Therapeutics optimized its approach to editing, guided by prior academic insights into manipulating the BCL11A gene to spur production of fetal hemoglobin, which protects against sickle cell symptoms. They also built extensive manufacturing capabilities.
In 2016, CRISPR Therapeutics announced a collaboration with Vertex Pharmaceuticals to co-develop CRISPR medicines. Vertex added its extensive drug development experience to position the Casgevy program for success.
The first sickle cell disease patient received Casgevy in mid-2019. Early results showed remarkably high levels of fetal hemoglobin production, reducing sickle cell crises. Positive multi-year, multi-patient data built confidence it could be a transformational medicine.
After accelerated development, regulators in the U.K. and U.S. approved Casgevy in late 2022 for certain sickle cell disease patients. It was the first ever regulatory OK for a CRISPR therapy. Ten years after its founding, CRISPR Therapeutics achieved a landmark gene editing advance thanks to years of scientific discovery and tireless efforts translating CRISPR’s promise. The full story contains many twists and turns by a cast of scientists, executives and patients who never lost faith this revolution was possible. Their vision has ushered in a new genre of CRISPR medicines, with more hopefully soon to come.
