Working towards
safe and effective
Advanced Therapy
Medicinal Products
The impact of rare disease
There are over 7,000 known rare and orphan diseases – and potentially many others yet to be discovered. Many are chronic, progressive and life-threatening and can impose a significant impact on a patient, their families and caregivers. Individuals living with these conditions are often young – sometimes very young and diagnosis can be slow, with treatment options limited. This, alongside costly and frequent hospital visits is likely to lead to physical, emotional and financial strain.
The transformative promise of ATMPs
Advanced Therapy Medicinal Products or ATMPs offer the potential to revolutionise medicine by offering transformative treatments that go beyong symptom management and address the root of a disease, particularly rare disease.
By harnessing the power of gene and cell therapy, and tissue engineering, ATMPs can provide personalised and potentially curative solutions for conditions that would otherwise be considered untreatable.
These innovative therapies have already shown remarkable success in treating rare genetic disorders such as Spinal Muscular Atrophy (SMA) and Metachromatic Leukodystrophy (MLD). And with it, they bring renewed hope to patients and their families.
But we are still at the beginning. Developing these therapies has been a long-held ambition for scientists in biomedical research and development – and of course the patients who stand the benefit.
Only through continuous investment in research and technology and by harmonising the global regulatory environment, can we truly expect ATMPs to deliver on this transformative promise and provide those impacted by rare disease with the hope and treatment they deserve.
About ARDAT
In 2019, the Innovative Medicines Initiative (IMI) now called the Innovative Health Initiative (IHI) committed to funding the ARDAT (Accelerating Research & Development for Advanced Therapies) project with the aim of advancing our collective knowledge about ATMPs and accelerating the development of new treatments for rare diseases.
The consortium is led by the University of Sheffield and Roche, and brings together 32 international partners from academia, industry, and small/medium enterprises.
Explore Our Project
Work packages
The project consists of several research groups, each responsible for key parts of the work plan
Partners
The consortium has members from across the established pharmaceutical industry, small/medium sized businesses and academia
News
Read all about our recent advancements and activities as we engage with patient groups and regulators
Outcomes
We are planning to publish our results through several articles in scientific peer reviewed journals