Schimmer Joshua, Breazzano Steven
Piper Jaffray & Co. , New York, New York.
Piper Jaffray is a leading full-service investment bank and asset management firm. Joshua and Steven are research analysts covering the biotechnology industry.
Hum Gene Ther Clin Dev. 2017 Jun;28(2):57-61. doi: 10.1089/humc.2017.29025.ind. Epub 2017 May 24.
The year 2016 was an exciting one for the field, with several notable successes outweighing a few setbacks. As the number of patients treated successfully (and safely) with gene therapy grows, the totality of evidence points to a robust platform with utility in orphan/ultra-orphan diseases as well as broader indications, and with hopefully increasing predictability of results. This year promises to feature more patients treated, more clinical data, and more gene therapy products in registration-enabling studies. For the field to continue to advance and mature into the next great drug delivery platform, a few unsolved and remaining questions need to be addressed, including the business model for cures, a broader safety/efficacy profile once more patients are treated, optimization of delivery (including next-generation approaches), and greater understanding of the impact of competitive dynamics. In this report, we detail the success and setbacks of 2016 and highlight the unanswered questions-and how the answers may shape the field in the years ahead.
2016年对该领域来说是令人兴奋的一年，取得了几项显著的成功，胜过一些挫折。随着接受基因治疗并获得成功（且安全）的患者数量不断增加，所有证据都指向一个强大的平台，该平台在罕见/超罕见疾病以及更广泛的适应症中都有用武之地，并且有望提高结果的可预测性。今年有望有更多患者接受治疗，有更多临床数据，以及更多基因治疗产品进入支持注册的研究阶段。为了使该领域继续发展并成熟为下一个伟大的药物递送平台，需要解决一些尚未解决的遗留问题，包括治愈方案的商业模式、随着更多患者接受治疗而展现出的更广泛的安全性/有效性概况、递送方式的优化（包括下一代方法），以及对竞争动态影响的更深入理解。在本报告中，我们详细阐述了2016年的成功与挫折，并突出了未解答的问题，以及这些答案如何在未来几年塑造该领域。
