Company: RNAC
Filing Date: 2025-03-13
Form Type: 10-K
Source: 0001453687-25-000060
Chunk: 66

Company: Cartesian Therapeutics, Inc.
Filing Date: 2025-03-13
Form: 10-K
Item: Item 1
Chunk 66
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 pediatric investigation plan, or PIP, that has been approved by the EMA. Normally, the pediatric clinical trials must be completed before the initial MA application for the relevant indication.  However, the EMA may grant a deferral of the studies in order not to delay the approval of the product in adults and the EMA may waive the requirement to conduct studies in certain circumstances, such as if the relevant condition does not occur in 

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children.  If pediatric clinical trials are completed in accordance with an agreed PIP, the product will be entitled to a six-month extension of its supplementary protection certificate.  However, if the product is authorized as an orphan medicinal product, it is entitled to a two-year extension of its 10 years of orphan exclusivity and not to an extension of the supplementary protection certificate.

For other countries outside of the EU, such as countries in Eastern Europe, Latin America or Asia, the requirements governing the conduct of clinical studies, product licensing, pricing and reimbursement vary from country to country. In all cases, again, the clinical studies are conducted in accordance with GCP and the applicable regulatory requirements and the ethical principles that have their origin in the Declaration of Helsinki.

When conducting clinical trials in the EU, we must adhere to the provisions of the EU Clinical Trials Regulation (EU) No 536/2014. If the sponsor of the clinical trial is not established within the EU, it must appoint an entity within the EU to act as its legal representative, who shall be responsible for ensuring compliance with the sponsor’s obligations under the Regulation and be the addressee for all communications. The sponsor must take out a clinical trial insurance policy, and in most EU countries the sponsor is liable to provide ‘no fault’ compensation to any study subject injured in the clinical trial. 

Prior to commencing a clinical trial in the EU, we must obtain a clinical trial authorization, or CTA, in each Member State in which the trial will be conducted.  There is a centralized application procedure where one national authority leads the scientific review of the application, while each concerned member state complete an ethical review of any CTA. The application for a CTA must include, among other things, a copy of the trial protocol and an investigational medicinal product dossier containing information about the manufacture and quality of the medicinal product under investigation. Any substantial changes to the trial protocol or other information submitted with the CTAs must be notified to or approved by the relevant competent authorities.

We are also subject to data privacy and security laws in the jurisdictions outside