AveXis and REGENXBIO Sign Licensing Agreement to Develop Therapies for ALS and Rett syndrome

AveXis and REGENXBIO have signed a new licensing agreement to develop therapies for a particular form of ALS and for Rett syndrome.

They already have an agreement that covers spinal muscular atrophy therapies.

The form of amyotrophic lateral sclerosis that the new agreement covers is the one caused by mutations of the superoxide dismutase 1 (SOD1) gene. That form accounts for about 20 percent of ALS cases.

Rett syndrome is an inherited neurological and developmental disorder that affects brain development. A disease that almost exclusively affects girls, it leads to a progressive inability to use muscles for speech and eye and body movements.

AveXis will use REGENXBIO’s gene-delivery platform, NAV AAV9 vector, to help it develop therapies for rare, inherited and life-threatening neurological diseases. NAV AAV9 vector delivers a healthy gene, placed inside the shell of an adeno-associated virus (AAV), directly to a patient cell’s nucleus.

Under the agreement, REGENXBIO will receive an up-front payment, ongoing fees, payments when milestones are achieved, and royalties for any ALS and Rett syndrome therapies that AveXis commercializes.

“This license agreement for our NAV AAV9 vector highlights the strength of our relationship with our existing NAV Technology Licensee, AveXis, and our commitment to bringing important new NAV-based gene therapies to patients with severe diseases with significant unmet medical need,” Kenneth T. Mills, president and chief executive officer of REGENXBIO, said in a press release. “As a leader in AAV-based gene therapy, REGENXBIO continues to selectively and strategically license our NAV Technology Platform for specific vector and indication combinations in a way that allows us to maintain our focus on internal product development while at the same time advancing the overall field by expanding the pipeline of NAV-based gene therapies.”

“Building on our experience and the success we have seen to date with the use of REGENXBIO’s NAV AAV9 vector in our spinal muscular atrophy clinical trials, this new license agreement reflects progress on executing our corporate strategy and our vision of becoming the leader in the treatment of rare and life-threatening neurological genetic diseases,” said Sean Nolan, AveXis’ president and chief executive officer. “While we remain intensely focused on the development and commercialization of AVXS-101 for the treatment of spinal muscular atrophy, we are excited by the potential for gene therapy to address the needs of patients with RTT and ALS – two devastating diseases for which there are no cures and insufficient existing treatments.”

The agreement follows preclinical-trial studies showing that gene therapy that uses the NAV AAV9 delivery platform is effective against ALS and Rett syndrome. AveXis plans new studies that it hopes will lead to regulators approving an Investigational New Drug application for the therapy as a treatment for the diseases.