Gene Therapy Briefs

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References

Novartis. 2018. Novartis announces landmark EU approval for one-time gene therapy Luxturna^® to restore vision in people with rare inherited retinal disease. Available at: www.novartis.com/news/media-releases/novartis-announces-landmark-eu-approval-one-time-gene-therapy-luxturna-restore-vision-people-rare-inherited-retinal-disease (accessed November 30, 2018).

Spark Therapeutics. 2018. European commission approves spark yherapeutics' LUXTURNA^® (voretigene neparvovec), a one-time gene therapy for inherited retinal disease caused by confirmed biallelic RPE65 mutations. Available at: http://ir.sparktx.com/news-releases/news-release-details/european-commission-approves-spark-therapeutics-luxturnar (accessed November 30, 2018).

Orchard Therapeutics. 2018. Registration No. 333-227698, registration statement filed pursuant to Rule 424(b)4. Available at: https://ir.orchard-tx.com/static-files/399ca0db-9ccb-4ffc-925f-bbab3f2c0223 (accessed December 1, 2018).

Genetic Engineering & Biotechnology News. 2018. GSK hands off rare disease gene therapy portfolio to orchard therapeutics. Available at: https://www.genengnews.com/topics/genome-editing/gsk-hands-off-rare-disease-gene-therapy-portfolio-to-orchard-therapeutics/ (accessed December 1, 2018).

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Abeona Therapeutics. 2018. Abeona Therapeutics and REGENXBIO announce worldwide exclusive licenses for the treatment of four rare Lysosomal Storage Disorders using NAV AAV9 vector. Available at: https://investors.abeonatherapeutics.com/press-releases/detail/137/abeona-therapeutics-and-regenxbio-announce-worldwide (accessed December 2, 2018).