by jgao | Jul 23, | HiTech Health Latest News
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:
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:
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
References
www.alliancerm.org
www.citeline.com
by jgao | Jul 1, | HiTech Health Latest News
The global clinical trials market is expected to nearly double to over £80 billion by 2032 and the United Kingdom remains one of the world’s leading destinations for conducting clinical research. Notably, the UK accounts for approximately 9.5% of all global cell and gene therapy clinical trials, highlighting its strength in the Advanced Therapy Medicinal Product (ATMP including cell and gene therapies) sector as well as broader pharmaceutical and biotechnology clinical research and development.
The consistent growth in the number of clinical trials conducted in the UK reflects its attractive research ecosystem, supported by strong infrastructure, a skilled workforce, and a progressive regulatory framework led by the Medicines and Healthcare products Regulatory Agency (MHRA). If you’re developing an Investigational Medicinal Product (IMP) and considering where to run your clinical trial, here are four reasons why you should consider the UK:
- Access to a Single, Publicly Funded Healthcare System – the NHS
The UK’s National Health Service (NHS) is the world’s largest publicly funded healthcare provider, delivering free care to approximately 69 million people and treating around 1.4 million patients every 24 hours. Furthermore, the NHS is deeply committed to engaging in clinical research and trials. The NHS Constitution pledges that all patients have the right to be informed about research studies they may be eligible for. In the past five years, 100% of NHS organisations have been research-active, and 75% have contributed to commercially sponsored clinical trials, making it a uniquely integrated and research-friendly healthcare system.
- Streamlined Regulatory Processes
The UK’s Combined Review allows for a single application to both the MHRA and Research Ethics Committee (REC), enabling faster approvals and reducing administrative burden. Recently introduced reforms to clinical trial regulation aim to make the process even more efficient and adaptable. In addition to this, the National Institute for Health and Care Research (NIHR) provides national infrastructure and support for high-quality clinical research, including feasibility assessments, site identification, and patient recruitment.
- Large and Diverse Population
With a population of approximately 69 million, the UK offers access to a large and ethnically diverse patient population, particularly in major urban centres like London. This diversity supports inclusive recruitment strategies and enables trial outcomes that are more generalisable, aligning with the increasing regulatory emphasis on population diversity from agencies including the FDA and EMA. HiTech Health have established relationships with clinical trial sites across the UK and our team have in-depth knowledge on what is required to initiate your clinical trial in the UK. The UK offers close collaboration between IMP developers, CDMOs, CROs, research institutes, and clinical trial sites, enabling efficient and coordinated study delivery.
- Access to a Skilled Workforce and Qualified Persons (QPs)
The UK is home to a highly skilled life sciences workforce, supported by world-class universities and a strong track record in innovative research. Clinical trial sponsors benefit from access to experienced scientists, clinical professionals, and Qualified Persons (QPs) who play a critical role in IMP batch certification and release.
While Brexit introduced separate regulatory pathways and licence requirements for the UK and EU, such as independent Manufacturer’s/Importer’s Authorisations (MIAs) and QP release, the industry has adapted well. At HiTech Health, we hold MIAs in both the UK and EU, and our QP teams operate across both regions, enabling seamless support for sponsors running trials in either or both jurisdictions. We are delighted to support a diverse portfolio of developers as they navigate the clinical trial landscape across Europe.
About HiTech Health (HTH)
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:
Interested in Learning More?
Contact us today to arrange a call and discover how HiTech Health can support your clinical trial journey in the UK.
IE: +353-1-9631489 | UK: +44-20-30267419 | US: +1-857-3265835
Email: info@hitech-health.com
Author: Paul Crozier
Date: 1st July 2025
by jgao | Apr 10, | HiTech Health Latest News
Dublin, Ireland, 10th April 2025 – HiTech Health, a Contract Development and Manufacturing Organisation (CDMO) specialising in advanced medicines, is delighted to communicate that the CanVas consortium led by HAON life sciences (a leading cell therapy biotech company) has received a €10.7 grant from the Disruptive Technology Innovation Fund (DTIF). The grant will help develop one of the first ever therapeutics for early brain injury by advancing Haon Life Science’s CanVas-001 therapy to the clinic.
CanVas-001 is an allogeneic investigational product containing two proprietary sourced cell types; ECFC (Endothelial Colony Forming Cells) and MSC (Mesenchymal Stromal Cells). CanVas-001 has demonstrated significant therapeutic benefit in large animal preclinical models of early brain injury. The CanVas platform is also developing drug candidates for adult neurodegenerative diseases.
The lead indication is Hypoxic Ischemic Encephalopathy (HIE), a devastating rare disease occurring when a baby’s brain does not receive enough oxygen or blood flow just before or shortly after birth. It is the principal cause of infant mortality and long-term neurologic disability, including accounting for a fifth of all cerebral palsy cases.
The CanVas consortium is a multidisciplinary team of experts, made up of the National Institute for Bioprocessing Research and Training (NIBRT) who will carry out analytical development, HiTech Health who will facilitate development and manufacture of CanVas-001 for clinic and University College Cork Infant Maternal and Child Health Research Centre, who will provide clinical development expertise in early brain injury and in particular, neonatal HIE.
About HiTech Health
HiTech Health (HTH) is a CDMO specialising in advanced therapies and small volume aseptic products. HiTech Health has process development laboratories and GMP manufacturing capabilities to support the development and manufacture of a diverse range of advanced medicines. HTH provides an end-to-end service including Qualified Person (QP), Responsible Person (RP), project and supply chain management services to ensure that products are manufactured, approved and distributed to clinical sites on time.
About DTIF
The Disruptive Technologies Innovation Fund (DTIF) is a €500 million challenge-based fund established under Project Ireland 2040. The objective of DTIF is invest in the development and deployment of disruptive technologies and applications on a commercial basis, driving collaboration between Ireland’s research base and industry to foster economic growth and innovation.
It is managed by the Department of Enterprise, Trade and Employment and administered by Enterprise Ireland.
About HIE
Hypoxic Ischemic Encephalopathy (HIE), a devastating rare disease occurring when a baby’s brain does not receive enough oxygen or blood flow just before or shortly after birth. It is a leading cause of infant mortality and long-term neurologic disability including accounting for a fifth of all cerebral palsy cases.
It is estimated one to three in every 1,000 births in Europe and the United States will be impacted by HIE resulting in 30,000 babies affected in these regions, each year. The incidence is far higher in low incomes countries with an estimate 1 million babies affected annually.
Contact us
by jgao | Apr 7, | HiTech Health Latest News
If you are planning on conducting clinical trials in the EU and are importing Investigational Medicinal Products (IMPs) then there are many factors to consider prior to clinical trial initiation. An IMP refers to a medicinal product that is being tested or used in a clinical trial to evaluate its efficacy, safety, or pharmacokinetics. The term is defined under the European Union’s Clinical Trials Regulation (EU) No. 536/2014 and applies to any medicine (including vaccines, biologics, cell or gene therapies) that is used in the context of clinical research. Commencing the journey to conduct clinical trials in the EU involves navigating both the specific country and general EU requirements, which is an intricate task.
Here are a few key considerations when importing IMPs into the EU:
1. Regulatory Authorisation
Prior to importing IMPs into the EU for use in clinical trials, you must submit a Clinical Trial Application (CTA) to the National Competent Authority (NCA) in the EU member state where the trial will take place. The CTA is a critical component of conducting clinical trials in the EU. If the proposed clinical trial involves multiple EU countries, the European Medicines Agency (EMA) and the Clinical Trials Regulation (CTR), which came into force in January 2022, provide a centralised process for multi-country trials. However, individual country regulations may still apply and it is recommended to discuss your clinical trial strategy with a regulatory expert.
2. Manufacturer’s /Importation Authorisation (MIA)
A Manufacturer’s/Importation Authorisation (MIA) is required if a company is involved in manufacturing and/or importation activities relating to human, veterinary or investigational medicinal product, within the EU as per GMP Annex 21. The term manufacturing and/or importation includes manufacturing, primary packaging, secondary packaging, physical importation of medicinal products or batch certification of medicinal products. To conduct clinical trials in the EU, it is not required that the Marketing Authorisation Holder (MAH)/Sponsor of the IMP hold their own MIA or hire an internal Qualified Person (QP) but they may partner with a company like HiTech Health who already hold MIAs in both the EU and UK and have a deeply experienced QP team in place. This can save you substantial time and money, thus enabling the MAH/Sponsor to commence clinical trials sooner.
3. Storage and Distribution Controls
Organisations are required to have appropriate controls in place for managing the storage and distribution of IMPs. Compliance with Good Distribution Practice (GDP) will help ensure safety, security and high product quality throughout the EU clinical supply chain. In some cases, IMPs need to be stored in a licenced depot or distribution centre in the EU, which must be approved for storing IMPs. Companies should consider how product temperature requirements will be maintained throughout the importation, storage and distribution activities.
HiTech Health holds both EU and UK MIAs as well as the ability to act as IOR for both the EU and the UK, allowing us to support global clients in overcoming complex challenges of importing IMPs and conducting clinical trials Europe as well as the UK. Our team have extensive hands-on experience with all aspects of importing IMPs into the EU/UK for clinical use including complex products such as cell or gene therapies, where direct to site importation is often required. By partnering with HiTech Health, our team guide you through the EU requirements and allow you to focus on other core business areas such as product development, clinical studies, and market access planning.
Schedule a call with our team today to learn more about our services and how we can support your organisation.
IE: +353-1-9631489 | UK: +44-20-30267419 | US: +1-857-3265835
Email: info@hitech-health.com
by jgao | Feb 5, | HiTech Health Latest News
Ireland has a strong track record of attracting major investment from the largest pharma companies in the world. The foundation of Ireland’s life sciences industry has been the small-molecule sector and has since developed expertise in biologics manufacturing. Ireland is home to nine of the world’s top 10 pharmaceutical companies and is the world’s third-largest pharmaceuticals exporter. The country has proven to be a successful gateway to the EU, UK, US, and other RoW markets, with companies availing of the operational, financial, and geographic benefits of setting up here or partnering with Irish-based companies. World-class regulation is an important element of that. Ireland has an exemplary compliance record with agencies like the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), both of which work closely with Ireland’s Health Products Regulatory Authority (HPRA).
The Growing Demand for Cell & Gene Therapies in Europe
There is rapidly growing diversity in therapeutic pipelines, including advancements in Cell & Gene Therapies Manufacturing. The global Advanced Therapeutic Medicinal Product (ATMP) market size was valued at USD 27.04 billion in 2022 and is expected to grow at a compound annual growth rate (CAGR) of 16.8% from 2023 to 2030. The emergence of cell and gene therapies has transformed the pharmaceutical industry and disease treatment landscape. It has opened new routes for the treatment of incurable diseases and several cancer types. The success of products such as Kymriah, Zolgensma, and others is the key factor driving the market.
Why Ireland for ATMP and Cell and Gene Therapy Manufacturing?
Ireland is actively supporting the growing field of cell and gene therapy, and companies are capitalising on the list of benefits that Ireland affords them. The Irish government is committed to making the country a global leader for the development, manufacture, supply, and adoption of advanced therapies, including cell and gene therapies and novel vaccines, through strong investment in world-class training and academic development, an innovative research base, and excellent clinical trials infrastructure. This synergy between industry and academia, bolstered by strong government support, has contributed to Ireland’s competitive edge in attracting and retaining talent in advanced therapies. For developers of cell and gene therapies, Ireland offers research and development incentives, access to an extensive network of clinical trial sites, and the availability of providers who have exemplary track records with regulatory authorities.
Key benefits of establishing ATMP manufacturing in Ireland include:
- World-class regulatory environment ensuring compliance with EMA, FDA, and HPRA standards.
- Strategic location offering seamless access to the EU, UK, and global markets.
- Tax incentives and R&D grants for biopharmaceutical companies.
- Highly skilled workforce with specialised expertise in advanced therapy manufacturing.
- Strong collaboration between industry and academia, fostering innovation in advanced therapies.
Comprehensive ATMP Manufacturer and Cell & Gene Therapies CDMO in Europe
HiTech Health is an ATMP Manufacturer in Europe, positioned at the forefront of Ireland’s Cell & Gene Therapies CDMO sector. Our EU GMP-certified manufacturing facility supports the development and manufacturing of advanced therapies from a strategic European base. HiTech Health was the first Irish company to secure a licence to contract manufacture cell and gene therapies on behalf of international clients. As a leading ATMP Manufacturer in Europe, our team of experts facilitates the transformation of a promising new therapy idea from initial development to the GMP manufacture of a drug to treat patients in a clinical environment, helping to accelerate the journey of advanced therapies towards commercialisation. Our GMP facility is equipped with cutting-edge technology designed to advance the development, production, and testing of sterile formulations.
HiTech Health also provides consulting services to biopharma companies to develop, launch, and support products. We hold Manufacturer’s / Importer’s Authorisations (MIAs) in both the EU and UK to seamlessly support companies with batch importation and release for clinical and commercial use. HiTech Health is working with early-stage developers to provide end-to-end cell & gene therapy CDMO services as well as batch approval and supply to clinical sites. For companies located outside Europe, we can support navigating the landscape for advanced medicines across all EU countries. Unlike the traditional pharmaceutical CDMO model, HiTech Health also has a logistics team to support the shipping and storage of time and temperature-sensitive therapies. Our expertise as a Cell & Gene Therapies CDMO allows us to provide comprehensive solutions tailored to the needs of biotech innovators and pharmaceutical companies.
Our full range of services to accelerate cell and gene therapy commercialisation, covering but not limited to:
- GMP manufacturing of ATMPs for clinical and commercial use.
- Regulatory consulting to support EMA, FDA, and HPRA approvals.
- Batch importation and release for the EU and UK markets under our Manufacturer’s / Importer’s Authorisations (MIAs).
- End-to-end CDMO services for early-stage developers.
- Logistics solutions for time and temperature-sensitive therapies.
- Clinical trial support, ensuring seamless supply chain management.
If you’d like to connect with a member of our team to learn more about our cell and gene therapy manufacturing services, please email info@hitech-health.com.
References
Enterprise Ireland – Pharma
IDA Ireland – Biopharma
Grand View Research – ATMP Market
HiTech Health – Cell and Gene Therapy Facility Launch
HiTech Health – Key Considerations for Selecting a Cell and Gene Therapy CDMO
by jgao | Jan 21, | HiTech Health Latest News
HiTech Health are delighted to have an article entitled ‘Cell and Gene Therapy Manufacturing Considerations for Early Stage Companies’ published in distinguished publications, cellandgene.com and outsourcedpharma.com. The article should be of benefit to companies who are looking for a partner to develop and manufacture their clinical products.
Read the full article by clicking here: https://www.cellandgene.com/doc/cell-gene-therapy-manufacturing-considerations-for-early-stage-companies-0001
HiTech Health is a leading European CDMO and service provider for ATMPs. Learn more about our cell and gene therapy services by clicking here
Schedule a call with our team today to learn more about our services and how we can support your organisation.
IE: +353-1-9631489 | UK: +44-20-30267419 | US: +1-857-3265835
Email: info@hitech-health.com
