GenEdit & Editas deliver nanoparticle boost for gene therapy

nanoparticle delivery of cas12a

Date: 12th December 2019

CRISPR-based therapeutics have the potential to transform the lives of patients with serious diseases.  This year has seen the first patients treated with CRISPR-Cas9 gene-edited hematopoietic stem cell therapy, and initial data appears promising.

However, delivering gene editing cargo safely and efficiently into patients is one of the next crucial breakthroughs into seeing this technology prevail in a clinical setting.

Yesterday saw an announcement that will accelerate the development of more comprehensive delivery systems for CRISPR-based therapeutics, as GenEdit and Editas Medicine enter a worldwide, exclusive license and collaboration agreement.

GenEdit, based in California, US, is transforming the delivery of gene therapies, driving the next generation of non-viral, gene editing-based therapeutics.  Their approach includes both proprietary engineering and novel polymer nanoparticles, which offer safer, targeted delivery options and improved efficiencies.   Their objective to enable gene knockout therapies but also gene repairs.

Editas Medicine is a leading genome editing company, based in Cambridge, US.  They are dedicated to treating patients with genetically defined diseases with use of CRISPRs.  In an earlier announcement at the beginning of this week Editas announced in vivo proof-of-concept data for EDIT-301, in development for the treatment of sickle cell disease and β-Thalassemia.

However, whilst both of these diseases have already been targeted for CRISPR-Cas9 based therapy by CRISPR Therapeutics and Vertex Pharmaceuticals, Editas are the first to develop such therapies using Cas12a.

The partnership should see the evaluation of delivery for Cas12a-based technologies with GenEdit’s nanoparticle platform with the option to further extend the collaboration later down the line.

The hope is that the partnership will expand the potential of gene therapies to treat more diverse sets of diseases and certainly, in the short term, it will be interesting to compare the outcomes of Cas9 v Cas12a for the treatment of sickle cell disease and β-Thalassemia. 


For more information please see the press release from GenEdit