Agreements

Date: 2017-06-07

Type of information: Licensing agreement

Compound: gene therapy treatments using RegenXbio’s NAV AAV9 vector

Company: RegenXBio (USA - MD) Avexis (USA - IL)

Therapeutic area: Rare diseases - Genetic diseases - Neurological diseases

Type agreement:

• licensing

Action mechanism:

• gene therapy.

Disease: Rett syndrome, genetic form of amyotrophic lateral sclerosis caused by mutations in the superoxide dismutase 1 (SOD1) gene

Details:

• • On June 7, 2017, RegenXbio and AveXis announced an exclusive worldwide license agreement for AveXis to develop and commercialize gene therapy treatments using RegenXbio’s NAV AAV9 vector to treat two rare neurological monogenic disorders: Rett syndrome (RTT) and a genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene. Preclinical data demonstrating promising efficacy and safety of gene therapy treatments for RTT and ALS using NAV AAV9, generated by AveXis’ Chief Scientific Officer Dr. Brian Kaspar at Nationwide Children's Hospital, has been licensed by AveXis. AveXis intends to move forward with initiating IND-enabling studies in both RTT and ALS and plans to provide more details on these programs in the second half of 2017.

Financial terms:

• Under the terms of the license agreement, REGENXBIO will receive an upfront payment upon execution, ongoing fees, milestone payments and royalties on net sales of products incorporating the NAV AAV9 vector.

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Is general: Yes