In what can be regarded as a persistent effort in bringing gene editing to masses, several biotech companies in the healthcare & medical devices industry have recently announced their decision to use the new Crispr gene editing techniques soon on humans to treat blindness & blood diseases. If sources are to be believed, two biotech companies, namely the Crispr Therapeutics and Editas Medicine have unveiled their plans to start testing the technology in humans in 2018 itself.
For those unfamiliar, CRISPR is a family of DNA sequences in bacteria and stands for “Clustered Regularly Interspaced Short Palindromic Repeats.” This technology has been in works for quite some time, but not until recently in 2013, did the technique finally penetrate the market and gain prominence in gene editing industry.
Reports state that Crispr Therapeutics, a Switzerland-based company has already submitted its findings and is waiting for the European regulators’ green nod to test its product. Code-named CTX001, this product will be tested in patients undergoing beta-thalassemia – a blood disease where the body lacks the ability to produce healthy red blood cells. The company is also planning to seek a go-ahead nod from the US FDA by the end of this year to test the CTX001 on people suffering from sickle cell disease – another inherited blood disorder.
Crispr’s US-based rival, Editas Medicine, is another prominent firm in the healthcare & medical devices industry that plans to apply to US regulators and the FDA by the middle of 2018. The company aims to test its own Crispr gene-editing products on patients suffering from congenital blindness – a disorder causing sever vision loss at birth. Reports reveal that on receiving the green-light from the FDA, the company should be able to commence its commercial trials within 30 days of the application.
It has also been reported that if the company trials succeed, healthcare & medical devices industry giants Editas, Crispr, and a third firm Intellia Therapeutics, plan to study gene editing techniques in range of diseases comprising Duchenne muscular dystrophy, cystic fibrosis, hemophilia, and cancer.