The HiTech Health team has reviewed recent developments in the Advanced Therapeutic Medicinal Product (ATMP) sector during the first half of 2025. Below are some key updates from the industry so far:
Sector Summary:
- In Europe, the ATMP sector is on track to achieve 5-6 regulatory approvals in 2025, positioning the region to exceed the three approvals it saw in 2023 and 2024 combined.
- The US ATMP sector is presently expecting 5-6 approvals in 2025.
- There are at least nine ATMP developers aiming to submit regulatory applications for ATMPs in 2025. Further developers are targeting to file regulatory submissions in early 2026.
- The U.S could still potentially see four or more approvals for gene therapies to treat rare diseases in 2025, adding to the momentum of eight total approvals in 2023 and 2024.
- CAR-T cell therapies remained the most common technology used in the pipeline of genetically modified cell therapies (preclinical through to pre-registration).
- The first half of the year has seen significant clinical progress for therapies treating neurological disorders such as Parkinson’s Disease.
The global ATMP sector has seen significant progress in the first half of 2025, fuelled by breakthroughs in cell and gene research, encouraging clinical developments, and streamlined regulatory frameworks. The table below provides a snapshot of the estimated number of ongoing clinical trials, active ATMP developers, and investment activity within the sector.
| North America | Europe | Asia-Pacific | H1 2025 Total | |
| Number of Clinical Trials | 844 | 304 | 838 | 1,905 |
| Number of Developers | 770 | 453 | 750 | 2,070 |
| Investment | $4.4B | $0.8B | $0.5B | $5.0B |
Clinical Developments:
Below are some clinical developments across the ATMP sector:
- Epicrispr Biotechnologies announced in April that the FDA cleared its experimental epigenetic therapy for clinical trials in the United States, making it the first in-human trial to use epigenetic editing for this indication.
- Ensoma announced in May that its first-in-class in-vivo hematopoietic stem cell (HSC)-directed therapy to treat chronic granulomatous disease was approved for phase 1 clinical trials.
- Capsida Biotherapeutics announced in May that the FDA cleared its gene therapy for syntaxin-binding protein 1 developmental and epileptic encephalopathy, marking the first human clinical trial using an IV-administered blood-brain barrier crossing capsid.
- BrainStorm Cell Therapeutic’s Nurown received FDA clearance for phase 3b trials.
- XellSmart’s allogenic IPSC-derived cell therapy for Parkinson’s disease and ALS has been cleared for phase 1 clinical trials.
Regulatory and Policy Updates:
FDA Eliminates Risk Evaluation and Mitigation Strategy (REMS) Requirements From Approved CAR-T Cell Therapies
In June of this year, the FDA eliminated REMS requirements from approved CAR-T cell therapies and made other labelling updates, including reducing restricted driving time after the treatment from eight to two weeks and shortening the required stay near a healthcare facility post-treatment from four weeks to two. This decision was made in an effort to remove complex barriers affecting patient access.
European Commission Seeks to Drive ATMP Competitiveness
The European Commission announced in Q2 2025 the timely release of its European Life Sciences strategy, which aims to make Europe a global hub for the life sciences industry. The strategy outlines several positive steps to reduce barriers to the development of advanced therapies, such as European Centres of Excellence for ATMPs and funding for multi-country clinical trials.
Commercial Market Trends:
There are currently two cell and gene therapy blockbuster products ($1B+ in annual global sales). The table below gives an overview of the Q1 2025 revenue of the top grossing ATMPs:
| Therapy | Q1 2025 Revenue | Increase from Q1 2024 | Blockbuster Status |
| Yescarta | $383 million | 2% | Achieved in 2022 |
| Elevidys | $375 million | 179% | Possible in 2025 |
| Carvykti | $369 million | 135% | On track for 2025 |
| Zolgensma | $327 million | 10% | Achieved in 2021 |
| Breyanzi | $262 million | 148% | On track for 2025 |
| Vyjuvek | $88 million | 95% | Possible by 2029 |
About HiTech Health:
We are a GMP-certified Contract Development and Manufacturing Organisation (CDMO) specialising in advanced therapies, including cell and gene therapies. Our team provide professional consulting services to support with product development, launch and supply across the advanced therapy, pharmaceutical, biotechnology, and medical device sectors.
Key Services or areas where HTH can help include:
- Qualified Persons (QPs) for both the EU and the UK.
- Contract Development and Manufacturing (CDMO) for advanced medicines and small volume aseptic products.
- Packaging, labelling, storage (incl Cryo) and distribution to clinical trial sites in the UK as well as the EU.
- HTH is an Importer of Record (IOR) for both the EU and the UK. We have trained Responsible Personnel (RPs) who can support logistics and supply of medicines.
- Clinical site readiness for patient treatment (CMC activities) for example using advanced therapies where handling, storage and thawing of cells is critical.
Interested in Learning More?
Contact us today to arrange a call and discover how HiTech Health can support your organisation.
IE: +353-1-9631489 | UK: +44-20-30267419 | US: +1-857-3265835
Email: info@hitech-health.com
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