Intellia opts out of hemophilia B gene editing work with Regeneron

Intellia Therapeutics is opting out of a hemophilia B program, leaving the CRISPR/Cas9-based gene editing work on factor IX treatments in the hands of partner Regeneron.

The bigger biotech had already been running lead on the factor IX programs, and so Intellia’s decision will have little bearing on Regeneron’s pipeline or near-term strategy, a spokesperson confirmed to Fierce Biotech. Regeneron remains committed to advancing the program, according to the statement. 

Intellia, on the other hand, will leave behind an obligation to share 35% of the costs for the program but also will not be eligible for the same amount of profits later on. The gene editing biotech will continue to support Regeneron as needed for the factor IX programs, according to a Securities and Exchange Commission document. Intellia will continue to be eligible for milestones up to $320 million and royalties in the high-single digits to low teens.

The opt-out only pertains to the hemophilia B program, while work in hemophilia A continues on, according to a spokesperson for Intellia. 

The decision to opt out of the factor IX program does not impact the companies’ other programs, including the phase 3 transthyretin (ATTR) amyloidosis program where Intellia serves as the lead.

Together, Intellia and Regeneron made history with the first-ever clinical results for a gene editing therapy when data from the ATTR amyloidosis program were revealed in June 2021. NTLA-2001 is an in vivo CRISPR/Cas9 genome editing candidate. The first patient in the phase 3 MAGNITUDE trial was recently dosed, according to a March 18 press release from Intellia.

NTLA-2001 is part of the same development agreement as the factor IX programs, which was signed between Regeneron and Intellia in April 2016. Regeneron paid $75 million upfront and offered a $50 million investment commitment in Intellia’s next equity financing, with “significant milestone and royalty payments” available down the line.

The deal was amended in June 2020, with Intellia receiving an upfront payment of $70 million plus an equity investment of $30 million at the time. The potential milestone payments were not listed.

Editor's note: This story was updated at 1:49 p.m. ET on March 22, 2024, with comments from Intellia and to clarify that the opt-out did not include hemophilia A.