Biotech Review of the Year: key updates

Authors: Claire smith, Adam Coughlin & Gabriela Swiderska

China’s regulation of CGTs and impact on licensing deals

When we wrote earlier this year here about the rollercoaster ride of the cell and gene therapy (CGT) sector, we suggested that one reason for optimism was the growing importance of China in the CGT ecosystem. Just six months later, we are already seeing that prediction come true. China has introduced a new regulatory framework for novel biomedical technologies that act at the cellular or molecular level, including CGTs. China’s State Council Order No. 818 (Regulations on the Administration of Clinical Research and Clinical Transformation Application of New Biomedical Technologies) came into effect on 1 May 2026. This creates a parallel regulatory route in China for these cutting-edge treatments, which will exist alongside the traditional drug approval pathway. The new route is designed to speed up patient access to these much-needed treatments and will enable CGTs to be clinically translated and commercialised far earlier than before, in a qualified hospital setting (and generate commercial revenue in the process), once they have demonstrated safety and basic efficacy.

At the same time, China's medicines regulator (the National Medical Products Administration (NMPA)), through its Center for Drug Evaluation (CDE), is proposing a package of reforms (including the “Xianrui (先锐) Plan”), to streamline and accelerate the development and approval of cell and gene therapies (CGTs), including earlier regulator–developer engagement, more flexible review pathways and greater support for innovative and urgently needed therapies.

Together, these developments signal a clear desire to make China an increasingly attractive jurisdiction for CGT development and commercialisation.

Order No. 818 brings a fundamental change in how CGTs may reach the Chinese market in the future which will, in turn (amongst other things), have an impact on licensing deals in the sector. In particular, from a licensor’s perspective, licence payments that are based on regulatory milestones or first commercial sales in China will need to cater for the Order No. 818 pathway (and should not purely be linked to NMPA drug approvals, for instance), and “net sales” definitions (on which royalties are payable) need to be flexible enough so they capture revenue generated through this route.

Advancements in in vivo CGTs

Another notable development since our original article is the continued and growing momentum behind in vivo CGTs.

Earlier this summer, researchers reported here one of the first clinical demonstrations of CAR-T cells being generated directly within patients using a CD8-targeted lipid nanoparticle platform, potentially circumventing some of the manufacturing and logistical challenges associated with traditional ex vivo CAR-T therapies. Although still at an early stage, the study provides a glimpse of how future CGTs could become more scalable and accessible.

In April 2026, Intellia announced here that its lonvoguran ziclumeran (lonvo-z) therapy has delivered what may prove to be a landmark moment for the field, reporting positive Phase 3 data in hereditary angioedema and reportedly becoming the first in vivo CRISPR gene-editing therapy to reach this stage of development. 

Together, these developments suggest that some of the sector's most ambitious goals, namely delivering genetic medicines and even engineered cell therapies directly inside the body, are moving from concept towards clinical reality.

Author: Luke Norton

Since publication of Biotech Review, there have been two major updates on the SPC Waiver regulation, both relating to the ongoing Janssen v Samsung Bioepis disputes concerning ustekinumab. 

Dutch Supreme Court 

The Attorney General at the Supreme Court of the Netherlands published its opinion which rejected Janssen’s appeal and supported the reasoning of the court of appeal. The Attorney General also rejected the referral to the CJEU. The decision of the Supreme Court is still pending. However, if typical timelines are followed, the final decision should be issued before the end of the year.

CJEU Referral

In parallel, a SPC manufacturing waiver CJEU referral has emerged from Denmark in a case between the same parties relating to the same actions/products. The referral focuses on the divergences we noted in our article and on the issue of storage prior to export: 

• Storage and stockpiling: Does the export waiver cover manufacture for indefinite EU storage pending potential future export, rather than a specific planned export?
• "Strictly necessary" acts: Can prolonged storage before export qualify as an act "strictly necessary" for the actual export of the product?
• Marketing authorisation at notification: Must a marketing authorisation in the intended export country already exist when the waiver notification is submitted?
• Third-country IP rights: Can the waiver be relied upon where the product could not yet lawfully be marketed in the export country because relevant IP rights remain in force there?

Given the divergent national approaches seen to date, particularly regarding the waiver's notification requirements, the referral could provide much-needed guidance on the balance between facilitating early biosimilar market entry and preserving the protection afforded by SPCs. It is not clear whether the CJEU decision or the outcome of the European Commission’s public consultation (the call for evidence has still not opened) will come first. 

Dutch Supreme Court 

The Attorney General at the Supreme Court of the Netherlands published its opinion which rejected Janssen’s appeal and supported the reasoning of the court of appeal. The Attorney General also rejected the referral to the CJEU in the Dutch case. The decision of the Supreme Court is still pending. However, if typical timelines are followed, the final decision should be issued before the end of the year. The Supreme Court, considering the Danish referral to the CJEU, may decide to stay its proceedings. 

Authors: Marc Dautlich & Kiran Sidhu

There is some way to go in the European legislative process for the proposed Biotech Act. At the time of writing, work in the various parliamentary committees tasked with scrutinising the proposals is ongoing. The Act is in the first reading stage, but neither the European Parliament nor the Council of the European Union has adopted its first-reading position. The first reading stage is unique as it has no time limit, though generally most EU legislation is adopted during the first reading. In short, the committees are not likely to finalise their work before Q1 2027. A European Parliamentary plenary vote is therefore unlikely before the summer of 2027.