Hunterian’s mission is to cure genetic diseases by correcting DNA mutations using its innovative CRISPR delivery technology.

Every year, nearly 8 million children are born with genetic diseases caused by mutations in DNA. There are 6,000-7,000 known genetic disorders – examples include ALS, cystic fibrosis, muscular dystrophy, or Fuchs’ dystrophy. For over 95% of these diseases, no FDA-approved treatments or cures exist. 

CRISPR is a revolutionary gene-editing tool with the ability to edit nearly any DNA sequence within the human genome, and the potential to correct a multitude of disease-causing mutations. While traditional medical research has focused on developing pharmaceuticals to treat the symptoms of genetic disorders, correcting DNA mutations directly could provide permanent, one-time cures.

Hunterian’s patented technology overcomes the delivery problem that has been the most significant barrier to widespread development of CRISPR-based therapeutics.

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 over a billion more regions in the human genome than any existing technology.