Onasemnogene abeparvovec
AVXS-101 (INN name: onasemnogene abeparvovec[1]) is an experimental gene therapy product being developed by Avexis (a subsidiary of Novartis[2]) as a treatment for spinal muscular atrophy, a severe neuromuscular disorder caused by a mutation in the SMN1 gene resulting in deficiency of survival motor neuron (SMN) protein.
AVXS-101 is a biologics consisting of self-complimentary AAV9 virus capsids that contain a SMN1 transgene. It is administered intravenously or intrathecally. Upon administration, the self-complimentary AAV9 viral vector delivers the SMN1 transgene to cell nuclei where the transgene begins to encode SMN protein, thus addressing the root cause of the disease.
The drug has shown encouraging results in early clinical trials.[3][4][5] As of October 2018, AVXS-101 is in late-phase clinical trials in Australia, Europe, and the United States.[6]
See also
References
- ^ "Onasemnogene abeparvovec - AveXis - AdisInsight". adisinsight.springer.com. Retrieved 2018-10-06.
- ^ "Novartis successfully completes acquisition of AveXis, Inc. | Novartis". Novartis. Retrieved 2018-10-06.
- ^ Alfano, Lindsay; Lowes, Linda; Al-Zaidy, Samiah; Shell, Richard; Arnold, W. Dave; Rodino-Klapac, Louise; Prior, Thomas; Berry, Katherine; Church, Kathleen (2018-04-10). "AVXS-101 Phase 1 Gene Replacement Therapy Clinical Trial in SMA Type 1: Patients Treated Early with the Proposed Therapeutic Dose Were Able to Sit Unassisted at a Younger Age (S29.002)". Neurology. 90 (15 Supplement): S29.002. ISSN 0028-3878.
- ^ Inc., AveXis,. "AveXis Presents Initial Data from Pivotal U.S. Trial for SMA Type 1 and 24-Month Follow-Up Data from Phase 1 Trial of AVXS-101 in SMA Type 1 at the Annual Meeting of the American Academy of Neurology". GlobeNewswire News Room. Retrieved 2018-10-06.
{{cite news}}:|last=has generic name (help)CS1 maint: extra punctuation (link) CS1 maint: multiple names: authors list (link) - ^ "AVXS 101 results from STRIVE trial for spinal muscular atrophy .-AveXis Inc., 01-May-18". epgonline.org. Retrieved 2018-10-06.
- ^ "Search of: AVXS-101 - List Results - ClinicalTrials.gov". Retrieved 2018-10-06.
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