Regenxbio is an early beneficiary of Novartis’ buyout of AveXis. The gene therapy tech provider just picked up $100 million of accelerated payments following the $8.7 billion acquisition.
The company $RGNX says the payout includes $60 million in annual fees and $40 million for a commercial milestone. Another $80 million marker is up for grabs along with royalties.
The pact with Regenxbio now gives the pharma giant an exclusive right to use the NAV Technology Platform for developing new treatments for SMA, including AVXS-101, which uses Regenxbio’s NAV AAV9 vector, the delivery vehicle.
Last November AveXis riveted the biotech world with data from a small study on SMA. Of 15 patients in Phase I, all were able to reach 20 months without the need for constant respiratory support, or dying. And many achieved improvements in motor symptoms, speaking to the potential — though still unproven — of this as a once-and-done therapy for a rare but dire ailment.
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