Hunterian Medicine LLC, a gene-editing company working to cure genetic diseases, has developed groundbreaking CRISPR delivery technology for which the United States Patent and Trademark Office (USPTO) has granted a patent (No. 9,907,863). The issued patent protects Hunterian’s core platform technology and covers the discovery of a novel “2-for-1” genetic element, a bidirectional promoter, that allows many CRISPR systems to be delivered into human cells using a single adeno-associated virus (AAV).

Hunterian’s mission is to cure genetic diseases by correcting DNA mutations using its innovative CRISPR delivery technology. Hunterian’s bidirectional promoter shrinks the cellular “instructions” necessary for the in vivo expression of CRISPR, thereby enabling CRISPR delivery well within the limited AAV packaging capacity. This novel approach could lead to the development of therapeutics for a far greater number of diseases than allowed by current technology.

“Hunterian’s patented technology overcomes the CRISPR delivery problem, which has been the most significant barrier to the widespread development of therapeutics from this revolutionary technology,” said Dr. Vinny Jaskula-Ranga, president and CEO of Hunterian Medicine. “Our innovation frees up critical space in AAV that can be used to develop the safest and most effective CRISPR-based therapeutics for literally thousands of diseasecausing mutations.”

The key to unlocking CRISPR’s therapeutic potential is delivery via 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 only therapeutic options of questionable safety and efficacy, or those that are applicable to a limited number of diseases.

“Our focus is on advancing a number of key targets within our internal pipeline,” added Dr. Jaskula-Ranga. “Given the multitude of genetic disorders and the rapid development that Hunterian technology enables, we look forward to engaging in broad partnerships to accelerate the development of these much-needed therapeutics.”

Hunterian has initiated its own research programs and is currently developing a portfolio of therapeutics to address several diseases.

Headquartered in Cambridge, Massachusetts, Hunterian Medicine LLC is a leading geneediting company working to cure genetic diseases by correcting DNA mutations using its innovative CRISPR delivery technology. The company’s patented technology solves the problem of CRISPR delivery with a “2-for-1” genetic element that enables delivery via a single adeno-associated virus (AAV).