[1] FDA. Approved Cellular and Gene Therapy Products. Volume 2021. U.S. Food & Drug Administration: U.S. Food & Drug Administration. [2] Scharner J, Aznarez I. Clinical applications of single-stranded oligonucleotides: current landscape of approved and in-development therapeutics. Mol Ther,2021,29:540-554. [3] Scott LJ. Givosiran: First Approval. Drugs,2020,80:335-339. [4] Scott LJ, Keam SJ. Lumasiran: First Approval. Drugs,2021,81(2):277-282. [5] Mickle K, Lasser KE, Hoch JS, et al. The Effectiveness and Value of Patisiran and Inotersen for Hereditary Transthyretin Amyloidosis. J Manag Care Spec Pharm,2019,25(1):10-15. [6] Kim J, Hu C, El Achkar CM, et al. Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease. N Engl J Med,2019,381:1644-1652. [7] FDA CfBEaR. Long term follow-up after administration of human gene therapy products; Guidance for industry. In: FDA, ed, 2020. [8] LTD JWaS. Gene Therapy Clinical Trials Worldwide. In: Medicine TJoG, ed. Volume 2021. [9] Chan YK, Wang SK, Chu CJ, et al. Engineering adeno-associated viral vectors to evade innate immune and inflammatory responses. Sci Transl Med, 2021,13:eabd3438. |