Genome editing pioneer Intellia Therapeutics has acquired Rewrite Therapeutics, a little-known biotech developing new tools for genome editing, including DNA writing via CRISPR/Cas9-guided polymerases.
As part of this transaction, Intellia will pay the Rewrite shareholders $45 million in an upfront payment and an additional $155 million in pre-specified research and regulatory approval milestones through a mix of Intellia common stock and cash. Additional financial details were not disclosed.
“At Intellia, we have built the industry’s broadest and deepest genome editing platform by staying at the forefront of new techniques, while also extending the capabilities of CRISPR/Cas9 editing to make precisely targeted changes to DNA,” said Intellia president and chief executive officer John Leonard, M.D.
He added that, “We could not be more excited to add Rewrite’s additional capabilities to our growing platform, offering us new possibilities and the potential to target diseases beyond those currently being explored in our pipeline.”
Rewrite has developed an approach that could improve the efficiency of genome editing in non-dividing cell types, a key challenge for some existing editing platforms. Rewrite’s technology could potentially be delivered using Intellia’s lipid nanoparticle (LNP) technology and adeno-associated virus (AAV) vectors.
Intellia made headlines in November 2020 when, as reported by Genetic Engineering & Biotechnology News, it and partner Regeneron launched the first clinical trial for an in vivo CRISPR therapy— CRISPR/Cas9 genome editing candidate, NTLA-2001 for patients with transthyretin (ATTR) amyloidosis. They published results of this first small (six patient) study in The New England Journal of Medicine in August 2021. The treatment bested standard of care, and this was the first-time gene editing was proven to work in humans.
Today there are almost a dozen clinical trials of in vivo CRISPR therapeutics ongoing. But there are also new technologies in the field, such as base and prime editing, which has gene editing companies looking to expand their tool boxes.
Rewrite has been focused on such opportunities. Founded by pioneering scientists Shakked Halperin and professor David Schaffer of the University of California, Berkeley and backed by Civilization Ventures and Prefix Capital, Rewrite’s DNA writing technology may enable a range of precise editing strategies. These strategies include targeted corrections, insertions, deletions, and the full range of single-nucleotide changes, which could provide new ways to edit disease-causing genes and broaden the therapeutic potential for genomic medicines.
Intellia, on its website, says it is “employing a modular genome editing platform to create diverse in vivo and ex vivo therapeutics, spanning a range of disease indications.” Currently, it has pipeline projects underway in hereditary angioedema, acute myeloid leukemia, solid tumors and others. One of the company’s co-founders is Jennifer Douda who, with Emmanuelle Charpentier, was awarded the 2020 Nobel Prize in Chemistry for pioneering work in CRISPR.
Halperin, Rewrite’s president and chief executive officer and the inventor of Rewrite’s gene editing platform, said, “Since my initial discovery that CRISPR-guided polymerases could help advance genome editing capabilities, I have focused my efforts on developing a suite of genome editing tools that could one day be used to create innovative and potentially curative treatments for patients with life-threatening diseases. I am thrilled that these inventions will now be leveraged by the industry leader, Intellia, so that the full power and potential of genome editing can be harnessed to benefit patients.”