继我们最近发布的新兴细胞和基因治疗公司潜在合规风险的文章之后，Stephen Abreu和Chris Fanelli提出了一些问题，供考虑在该领域进行投资或收购的相关人士进行探讨。

Following our recent post highlighting potential compliance risks at emerging cellular and gene therapy companies, Stephen Abreu and Chris Fanelli provide some questions and topics to explore when considering an investment or acquisition in this space.

1、许多新兴细胞和基因治疗公司都是从孵化器或学术机构中分拆出来的，并没有按照监管机构期望的方式成立。因此，您需要了解目标公司是否只符合大学实验室的标准，而不符合监管机构的标准。例如，是否有独立的质量监督部门？负责日常检测或生产操作的员工是否核查自己的工作？

1.Many emerging cellular and gene therapy companies spin out from incubators or academic institutions and are not set up in the way in which the regulator might expect from an oversight perspective. Consider whether your target complies with the standard of a university laboratory rather than with the expectations of the regulator. For example, is there independent quality oversight? Or are the employees responsible for the day-to-day testing or manufacturing operations checking their own work?

2、许多新兴公司都在努力满足监管机构对提供的生产工艺和检验方法的数据（作为研究申请的一部分）日益严格的要求。例如，在美国，由于化学、生产和控制方面的缺陷，许多处于早期阶段的公司已收到FDA的临床暂停函。您的目标企业是否有从监管机构获得调查申请的经验？是否对工艺和检验方法有足够的了解，使其能够获得调查申请的批准并继续推进？

2.Many emerging companies struggle with meeting regulators’ increasingly stringent expectations of the provision of data about the manufacturing process and testing methods as part of an investigational application. For example, in the U.S. a number of early stage companies have received clinical hold letters from the FDA due to deficiencies in chemistry, manufacturing, and controls. Does your target have experience in obtaining investigational applications from regulators? Does it have a sufficient understanding of the process and testing methods to enable it to obtain approval of an investigational application and move forward?

3、查看目标公司的生产活动实际上是在哪里进行的，是在学术实验室，还是在符合药品生产质量管理规范（GMP）标准的专用场地（无论是否位于学术机构）？

3.Look at where the target’s manufacturing activity is actually being conducted. Is it at an academic laboratory, or is it in a dedicated space—whether or not located in an academic institution—that meets Good Manufacturing Practice (GMP) standards?

4、该领域的许多公司专注于未解决疾病领域的产品，这些产品可以快速地完成开发，并可以从加速审批中受益，这意味着可以在极其压缩的时间内开发产品。然而，公司想要扩大生产规模是不容易的，这会增加测试水平、所需的物料量以及随之而来的监管要求，所以了解一下您的目标公司是否会陷入无法满足其产品需求的境地。询问其是否有扩大规模的计划？它是否聘请了CMO？如果是，聘请条件是什么？

4.Many companies in this space are focused on products in unaddressed disease areas. These products can move through development extremely quickly, and can benefit from accelerated approvals. This means that products can be developed over an extremely compressed timeline. However, production may not easily adapt to scaling up, which increases the level of testing, the amount of material needed and the attendant regulatory requirements. Consider whether your target could end up in a situation where it is unable to meet demand for its product. Ask about its plans for scaling up. Has it engaged a CMO and, if so, on what terms?

5、仔细查看目标公司迄今为止从监管机构收到的信函的性质，如果会议纪要显示监管机构一再提出问题，但您发现目标公司没有去解决这些问题，请将此视为危险信号。阅读公司的源文件，并与负责处理监管机构提出问题的工作人员交谈，考察此人是否具备足够的经验来解决监管机构的问题？

5.Look carefully at the nature of the correspondence that the target has so far received from the regulator. If meeting minutes show that the regulator has repeatedly raised concerns, but you find that they have not been addressed, consider this to be a red flag. Read the source documents and speak to whomever was responsible for dealing with the issues that the regulator raised from a quality and regulatory perspective. Does this person appears to have the necessary experience to resolve the regulator’s concern?

6、查看目标公司在生产方面已有的现有协议。目标公司是否拥有其产品和相关IP的生产工艺？于一家新兴的细胞和基因治疗公司而言，生产工艺与产品一样有价值。如果生产商拥有该工艺，那么未经第三方生产商许可，您作为收购方将不能使用该工艺。如果该生产商遇到监管问题，那么您很可能会遇到无法转移的麻烦。

6.Look at the existing agreements that your target has in place for manufacturing. Does the target own the manufacturing process for its products and related IP? For an emerging cellular and gene therapy company, the manufacturing process is as valuable as the product. If the manufacturer owns the process, then you as the acquirer will not be able to use that process without permission from the third-party manufacturer. And if that manufacturer experiences regulatory issues, then you are likely to find that you are unable to go elsewhere.

7、考虑目标公司是否具备将其生产工艺转移给另一家生产商的实际技术能力，如果其扩大规模且当前生产场地不再能够满足需求，目标公司是否计划扩大生产设施规模？更为重要的是，其是否有能力将其生产技术转移到那里？

7.Consider whether the target has the actual technical ability to transfer its manufacturing process to another manufacturer, if it scales up and the place where it is currently manufacturing is no longer able to meet the demand. Has your target planned for a scaled-up manufacturing facility? Crucially, does it have the capability to transfer its manufacturing technology there?