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Intellia opts out of hemophilia B gene editing work with Regeneron

2024-03-22

交易

临床3期基因疗法引进/卖出

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.

Intellia TIntelliaics iRegeneronout of a hemophilia B program, leaving the CRISPR/Cas9-based gene editing work on factor IX treatATTR amyloidosisds of partner Regeneron.

Intellia Therapeuticsd already been runnihemophilia Bhe factor IX programs, and so Intellia’s decision will have lfactor IXring on Regeneron’s pipeline or nearRegeneronategy, 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 obligafactor IXhare 35% of the coIntelliathe program but also will not be eligiblRegeneron same amount of profits later on. The gene editing biotech will continue to supRegeneronneron 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.

Intelliaout only pertains to the hemophilia B program, while work in hemophilia A continues on, according to a spokesperson for Intellia.Regeneron factor IX Intellia

The decision to opt out of the fahemophiliaogram does not impact the hemophilia other programs, including the phase 3 transthyreIntelliaR) 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 dattransthyretin (ATTR) amyloidosis transthyretin (ATTR)oidosis program were revealIntelliane 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 Intelliaof thRegeneronvelopment agreement as the factor IX programs, which was signed between Regeneron and Intellia inATTR amyloidosiseneron paid $75 million upfront and oNTLA-2001$50 million investment commitment in Intellia’s next equity financing, with “significant milestone and royalty payments” available down the line.Intellia

The deal was amended in June 2020, with Intellia receiving factor IXt payment of $70 million plus an equRegenerontmentIntelliamillion at the tRegeneronpotential milestone payments were not listed.Intellia

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

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