At the presentation of the financial results for the second quarter ended 30 June 2021, Intellia Therapeutics, Inc. President and CEO John Leonard, M.D., says:
“This quarter marked an exciting new chapter for Intellia. We presented the first clinical data in history supporting precision editing of a disease-causing gene within the body following a single, systemic dose of CRISPR/Cas9. These data open a new era of medicine – one that holds the potential of curing genetic disease. In addition, together with Blackstone and Cellex, we launched a new company combining our allogeneic cell engineering platform with a clinically validated switchable, universal CAR-T construct. This new venture confers numerous benefits to Intellia, extending the reach of our technology beyond our core internal focus and giving us a meaningful stake in the company’s success, even as we retain our rights across a diverse ex vivo landscape. This transaction also fortified our leading cell engineering capabilities by securing access to high-quality donor cells and dedicated manufacturing capacity to support the development of our wholly-owned ex vivo programs.
We are well-positioned to build on this quarter’s momentum as we close in on a number of upcoming milestones. We look forward to initiating Phase 1 trials this year for our candidate NTLA-2002 for HAE.”
In June, Intellia announced that it had submitted its first Clinical Trial Application (CTA) to the New Zealand Medicines and Medical Devices Safety Authority for NTLA-2002 to initiate a first-in-human study. The Company expects to enroll the first patient by year-end and is also submitting additional regulatory applications to enable enrollment in other countries. The first-in-human trial is expected to evaluate safety, tolerability and activity in patients with HAE, and will continue to leverage insights gained from the development of NTLA-2001.