SPK-3006 explained

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]}

Notes and References

1. 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 . Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates . Nature Communications . 4 November 2021 . 12 . 1 . 6393 . 10.1038/s41467-021-26744-4 . 34737297 . 8568898 . 2021NatCo..12.6393C . en . 2041-1723. free .
2. Philippidis . Alex . Magenta Halts Development, Pursues Strategic Alternatives After Patient Death . Human Gene Therapy . 1 March 2023 . 34 . 5–6 . 177–179 . 10.1089/hum.2023.29236.bfs. 36944087 . 257664969 .