SPK-3006
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Experimental gene therapy
SPK-3006 is an experimental gene therapy developed for Pompe disease by Spark Therapeutics. It is delivered via adeno-associated virus and is intended to increase alpha-glucosidase production in the liver.[1][2]
- ↑ Costa-Verdera, Helena; Collaud, Fanny; Riling, Christopher R.; Sellier, Pauline; Nordin, Jayme M. L.; Preston, G. Michael; Cagin, Umut; Fabregue, Julien; Barral, Simon; Moya-Nilges, Maryse; Krijnse-Locker, Jacomina; van Wittenberghe, Laetitia; Daniele, Natalie; Gjata, Bernard; Cosette, Jeremie; Abad, Catalina; Simon-Sola, Marcelo; Charles, Severine; Li, Mathew; Crosariol, Marco; Antrilli, Tom; Quinn, William J.; Gross, David A.; Boyer, Olivier; Anguela, Xavier M.; Armour, Sean M.; Colella, Pasqualina; Ronzitti, Giuseppe; Mingozzi, Federico (4 November 2021). "Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates". Nature Communications. 12 (1): 6393. Bibcode:2021NatCo..12.6393C. doi:10.1038/s41467-021-26744-4. ISSN 2041-1723. PMC 8568898. PMID 34737297.
- ↑ Philippidis, Alex (1 March 2023). "Magenta Halts Development, Pursues Strategic Alternatives After Patient Death". Human Gene Therapy. 34 (5–6): 177–179. doi:10.1089/hum.2023.29236.bfs. PMID 36944087. S2CID 257664969.