CRISPR-Cas9 gene editing tech could eliminate orphan diseases, entrepreneur

CRISPR-Cas9​ is a tool for gene editing that can be faster and more precise than traditional knock-out options.

There has been a surge in interest over the past two years with Big Biopharma – such as AstraZeneca​ and Novartis​ – investing in it, along with services companies like Horizon Discovery​, and tech companies like Thermo Fisher​ to develop everything from new preclinical disease models to cell lines used for biopharmaceutical production

But the future of the technology lies in developing therapies to treat orphan diseases, serial entrepreneur and life science investor Jim Mellon told delegates at Biotrinity in London this week.

“At the moment CRISPR-Cas9 has greater application outside of the body, through the testing of new therapies, but in due course it will be used in living human beings, in particularly in the elimination of orphan diseases,”​ Mellon, the founder and co-chairman of Mann Bioinvest, told delegates.

“There are 7000 orphan diseases and some huge companies have been built on these – Alexion and Shire for example - and if we could actually eliminate these then the industry will be doing a great service for mankind.”​

He added orphan diseases place an enormous cost burden on both the patients and payers. Alexion’s mAb for the rare disease aHUS, Soliris (eculizimab), costs roughly $570,000 per patient per year​, for example.

But as technology advances and the cost of genome sequencing drops further, “CRIPSR-Cas9 has tremendour potential,” ​Mellon said.

He named the Massachusetts company Editas as leading the way in using the tech to edit patient DNA and help cure diseases. The firm received a boost last August through a $120m investment​ by a group of public and private investors led by Google and the top science advisor to Bill Gates.

Legal issues​

CRISPR itself cannot be patented and Cas9 is a naturally occurring protein and part of a naturally-occurring bacterial process, but there are ongoing legal challenges over who owns the IP on engineered components and compositions of the technology.

Multiple organisations have filed international patents related to uses of CRISPR-Cas. A team at the Broad Institute and MIT was issued 23 patents with claims to CRISPR and/or Cas9 in December last year​, but was challenged by the Regents of the University of California, the University of Vienna, and Emmanuelle Charpentier in January.

But Mellon said while these patent issues have tripped up some of the companies which originally invested in the technology, he remained positive these problems will be resolved in due course.

Intellia going public​

In related news, the gene editing company Intellia Therapeutics, which uses CRISPR-Cas9 technology licensed from Editas has launched an IPO and is looking to raise around $140m to support its therapy pipeline.

“​We intend to use the net proceeds from this offering to advance the research and development of our sentinel indications, progress additional in vivo and ex vivo pipeline product candidates, further develop our delivery technologies and CRISPR/Cas9 gene editing platform and for working capital and other general corporate purposes,”​ the firm wrote in its prospectus​.

The firm entered into a collaboration agreement with Novartis regarding the discovery of new CRISPR-Cas9-based therapies using chimeric antigen receptor T cells in December 2014, and earlier this month entered into a collaboration agreement with Regeneron focused on gene editing in the liver.