Many scientists believe the key to unlocking CRISPR’s therapeutic potential is delivery via an adeno-associated virus (AAV), an FDA-approved delivery vehicle with a history of safety, efficacy, and lack of toxicity. Until now, the small size of AAV has been a significant obstacle for the packaging of CRISPR, leaving less safe and less effective therapeutic options.
Hunterian’s technology uses a novel “2-for-1” genetic control element, a bidirectional promoter, to overcome the challenge of delivering CRISPR via AAV. The bidirectional promoter shrinks the “instructions” necessary for in vivo expression of CRISPR, thereby enabling CRISPR delivery well within the limited AAV packaging capacity.
By freeing up critical space in AAV, Hunterian's technology allows for the development of the safest and most effective CRISPR-based therapeutics. Hunterian's approach has the immediate potential to bring multiple CRISPR technologies to the clinic and dramatically accelerate the development of CRISPR-based therapeutics.
Our platform technology enables scientists to target far more regions in the human genome – over a BILLION more than existing technologies – meaning that many more mutations in skeletal or cardiac muscle, lungs, brain, and other tissues can now be addressed using CRISPR technology.