The first human is treated with a CRISPR gene-edited therapy

Date: 25th February 2019

The translation of CRISPR-based therapies into FDA-approved, market-ready products is at the start of its journey however the first wave of these revolutionary treatments are starting to break into clinical trials.

One such product, CTX001, an investigational, autologous, CRISPR-Cas9 gene edited hematopoietic stem cell therapy aimed at patients suffering from severe hemoglobinopathies (in this case transfusion-dependent beta thalassemia) has entered into phase1/2 trials.  Massachusetts based proprietors CRISPR Therapeutics and Vertex Pharmaceuticals have announced the treatment of the first patient, with a second to follow shortly.  The aim of the trial is to assess the safety and efficacy of a single dose at sites in Canada and Europe.  It is hoped that the USA will be added as a trial location later in the year. The companies are also assessing CTX001 for the use in patients with severe sickle cell disease and has recruited the first patient for a phase 1/2 clinical trial, with infusion likely to commence later this year.

This is an exciting but apprehensive time for this embryonic market.  We will be watching in hope that this offers efficient, safe therapies for patients worldwide.  However, side effects whether it is specificity or immunological will be an important determinant on tipping the balance of these products becoming more readily available.  Patents and costs will also be a big consideration before these techniques become mainstream.  For a more in depth article on CTX001 read the press release by following the link.