Intellia puts pedal to the metal as 3 new candidates prepped for clinic

Intellia has three candidates from its gene editing and cell therapy platforms headed to a clinic near you in the next year or so.

The company previewed its next steps in a first-quarter earnings call Thursday morning, detailing recent clinical activities and the upcoming data readouts that will define the CRISPR biotech for years to come. Intellia made waves last year with the release of first-in-human gene editing data, and, now, analysts and investors are eager to hear how the team will follow that up.

Work is underway to move three candidates into the clinic, with the nearest one being NTLA-3001, a CRISPR-mediated in vivo gene insertion candidate for Alpha-1 antitrypsin (AATD)-associated lung disease. Intellia plans to submit an application for human testing with the FDA in 2023.

NTLA-3001 is designed to insert a copy of the SERPINA1 gene with the aim of restoring the expression of a protein called A1AT, which is missing or deficient in patients with AATD-associated lung disease. Without the ability to create any or enough of the protein, lung tissue is damaged or destroyed.

Another candidate, called NTLA-2003, is being developed for the type of AATD that results in liver damage. Intellia is currently initiating activities that could lead to an FDA filing for human study. The company’s hope is that the in vivo knockout development candidate can halt the progression of liver disease and prevent the need for a liver transplant in patients with AATD-associated liver disease.

Finally, NTLA-6001 is getting prepped for the clinic to treat CD30-expressing hematologic cancers such as relapsed or refractory classical Hodgkin lymphoma. The allogeneic CAR-T therapy was the first to use Intellia’s proprietary allogeneic cell engineering platform. NTLA-6001 combines a variety of sequential gene edits in an effort to protect T cells from immune rejection by the host T cells and natural killer cells.

Chief Financial Officer Glenn Goddard also promised that at least one more in vivo development candidate will be named before the end of the year.

'Full speed ahead'

As for the finances, Intellia, which does not have any approved products, reported cash on hand of $994.7 million as of March 31, which is down from $1.1 billion at the end of 2021. This decrease is due to operation expenses and the $200 million acquisition of Rewrite Therapeutics.

R&D expenses for the quarter increased by $93.8 million to $133.1 million, which is up substantially from the same period a year ago when Intellia spent $39.3 million. The escalation in expenses seems to reflect Intellia’s pedal-to-the-metal approach that has been in place since the first-in-human results for NTLA-2001 were released in June 2021.

CEO John Leonard, M.D., also spoke to the ongoing patent dispute regarding CRISPR gene editing technology, which was appealed to the U.S. Court of Appeals for the Federal Circuit. A recent decision of the U.S. Patent and Trademark Office determined that a group including the Broad Institute of MIT and Harvard were the first to invent CRISPR/Cas9 for editing a certain type of human cell that can be used to make medicines.

The decision could have wide implications for companies like Intellia as well as peers CRISPR Therapeutics, ERS Genomics and Caribou Biosciences. But Leonard and his crew have insisted that there will be no major impact on the biotech’s intellectual property.

Keeping with the company’s previous assertions on the case, the CEO said the findings so far “have no bearing on how we decide what to do, or what we need to do.”

“Full speed ahead with all the programs we do,” Leonard added.

Regardless of what happens with the legal process, Leonard said he believes if the matter is not resolved, “there’s other ways to get to an agreement” because it is in everyone’s interest to continue exploring gene editing with CRISPR.