Ardat

The Coordinator of ARDAT is recognised by a BSGCT Award

peter.kiskovacs — Fri, 29 May 2026 06:27:27 +0000

Professor Mimoun Azzouz has been awarded the BSGCT Outstanding Achievement Award, in recognition of his major contributions to gene therapy research, translation and training.

We are proud to share that our Director, Professor Mimoun Azzouz has been recognised by the British Society for Gene and Cell Therapy for his contribution to gene therapy research, translation and training.

Over the past 20 years, Mimoun has generated £34.1 million in research income and supervised the research training of 81 individuals, including 20 PhD students. He is the scientific founder of BlackfinBio and co-founder of Crucible Therapeutics and has also led the €25.5 million Accelerating Research & Development for Advanced Therapies consortium involving 34 EU partners. His pioneering studies include demonstrating the success of SMN gene therapy in mouse models of spinal muscular atrophy, driving clinical translation in SOD1-ALS research and clinical studies of a gene therapy for patients with HSP47. Mimoun played the lead role in the vision and efforts to attract an array of funds from various sources to establish GTIMC and continues to provide leadership to the team.

In response to the award Mimoun says “I’m humbled and incredibly honoured to accept this award. I would like to thank BSGCT for selecting me for this award. This recognition belongs to my amazing teams at SITraN and GTIMC, whose hard work and dedication made this possible. Huge thanks to all my collaborators, partners and colleagues for their support. I would also like to thank our sponsors and funding bodies for supporting our projects. Big thank you to my family for their unwavering support. This recognition means so much. Thank you!”

With thanks to BSGCT for allowing us to use the image. Credit to Danny Loo Photography

Final ARDAT General Assembly Meeting

peter.kiskovacs — Tue, 28 Apr 2026 13:54:54 +0000

The ARDAT (Accelerating Research and Development for ATMPs) project held its Final General Assembly Meeting (GAM) on April 20–21, 2026, hosted by Bayer in Berlin, Germany. The meeting brought together partners both in person and online to review progress across work packages, with a strong focus on delivering key outputs in the final months of the project.

Significant advances have been made toward ARDAT’s core objectives, particularly in developing data and tools to address knowledge gaps in areas such as AAV intracellular metabolism and cellular immunogenicity—efforts that are expected to accelerate research and development of Advanced Therapy Medicinal Products (ATMPs).

The assembly was marked by engaging discussions, a wealth of ideas, and overwhelmingly positive feedback, underscoring the project’s lasting impact and promising future despite its conclusion.

Thanks to all those that contributed to making this final meeting a great success!

Novel assays to assess the prevalence and neutralizing potential of anti-IdeS antibodies in healthy humans

peter.kiskovacs — Thu, 26 Feb 2026 08:35:51 +0000

Our ARDAT colleague Sebastien Lacroix-Desmazes (INSERM) and colleagues have published their work on the development of novel assays to assess the prevalence and neutralizing potential of anti-IdeS antibodies in healthy humans. Further details about the Frontiers in Immunology publication can be accessed via the link.

Regulatory requirements for assessment of immunogenicity for gene therapy medicinal products

Ardat — Wed, 05 Nov 2025 08:56:04 +0000

We are proud to announce that ARDAT colleagues from work package 5 have published an important open access paper in Cell Reports Medicine on the regulatory requirements for assessment of immunogenicity and the implementation of immunomodulation protocols for development of advanced therapy medicinal products (ATMPs), in particular gene therapy. This paper completes one of the key regulatory deliverables of the ARDAT project and represents an important nexus of discussion and experience sharing between industry and academic partners. In brief, the paper summarises all the key global regulatory guidance documents that are directly and indirectly related to assessment of immunogenicity and immunomodulation. These are broken down into key areas to identify where such issues are covered and where future guidance may address specific issues. This is considered important given the growing diversity or therapeutic technologies and mechanisms of action. The paper can be accessed via the link.

Technology Transfer

Ardat — Thu, 16 Oct 2025 09:37:42 +0000

Zih-Liang Yang from The University of Sheffield visited the ViraVector facilities at GeneT, The University of Coimbra, as part of the Accelerating Research & Development for Advanced Therapies (ARDAT) project. ARDAT aims to accelerate the research and development of advanced therapy medicinal products (ATMPs), with a strong focus on rare genetic diseases.

The visit involved a technology transfer and training program covering several standardized operating procedures (SOPs) related to key analytical techniques — including ELISA, qPCR, ddPCR, and Next-Generation Sequencing (NGS) — used for the quantification of AAV viral titers.

This collaboration represented an important step towards:

Harmonizing analytical workflows across institutions;
Strengthening our expertise in viral vector characterization;
Ensuring reproducibility and consistency in AAV quantification methods;
Building stronger connections within the ARDAT consortium

For more information follow the link.

Felix Bubeck successfully defended his PhD

peter.kiskovacs — Wed, 01 Oct 2025 08:19:47 +0000

Felix Bubeck successfully defended his PhD entitled: “Searching the hairpin in the haystack: Engineering the replication origin of AAV vectors” researching the use of synthetic AAV ITRs for enhanced vector persistence. Felix completed his PhD at the Heidelberg University, Germany and is pictured with his PhD supervisor and fellow ARDAT colleague Professor Dirk Grimm wearing his “PhD hat” depicting two ITR’s protruding from the hat. Congratulations Felix!

BIC International conference

peter.kiskovacs — Wed, 01 Oct 2025 08:09:42 +0000

Amelia Trecco, INSERM PhD student supervised by Dr Sebastien Lacroix-Desmazes and Dr Claire Deligne presented her data obtained in the ARDAT project at the 13^th BIC International conference in Padua, Italy 12^th-14^th September 2025. Her talk was entitled; Immunogenicity of IdeS: the challenge of repeated use for clinical applications. As one of the leading events for experts in hemophilia, von Willebrand disease, gene therapy, and thrombotic microangiopathies, the BIC Conference offers a unique opportunity to engage in cutting-edge scientific discussions and connect with leading professionals in the field. For more information, including conference abstracts, follow the link.

Attending the International Congress of Immunology

peter.kiskovacs — Mon, 01 Sep 2025 14:52:38 +0000

Principle Scientist Stephan Holtkamp from Bayer recently attended the 19th International Congress of Immunology in Vienna and presented his published ARDAT work in collaboration with colleagues at The University of Liverpool. The work provides insights into adaptive immunity with rAAV vector serotypes, which will be important in advancing gene therapy safety. For more information follow the link Stephan Holtkamp and the published paper can be accessed via the link.

ARDAT student Diana Lobo has successfully defended her PhD thesis

peter.kiskovacs — Mon, 01 Sep 2025 14:48:26 +0000

ARDAT student Diana Lobo has successfully defended her PhD thesis, titled “Overcoming the Major Roadblocks for Successful AAV-Based Gene Therapy in the CNS”. Diana completed her PhD under the supervision of Luis Pereira de Almeida and Rui Jorge Nobre at the Center for Neuroscience and Cell Biology (CNC) at The University of Coimbra, Portugal. For more information, follow the link Diana Lobo.

Congratulations Dr Lobo!

5th ARDAT General Assembly Meeting

peter.kiskovacs — Tue, 13 May 2025 06:22:12 +0000

42 ARDAT members gathered in Barcelona, Spain, for the 5^th ARDAT General Assembly Meeting, 28-29^th April 2025, hosted by colleagues Asphalion. The two-day meeting began during an unprecedent nationwide power outage, resulting in colleagues using their problem-solving skills to present their work in a somewhat innovative and traditional manner. Despite the challenges, the first day kicked off with a management and ethics update by Dr Rachel Waller and lots of conversation and discussion was had regarding the work being completed across work packages 5, 4 and 3.

Still with no power, WiFi or phone signal the first day concluded with a site-seeing walk to Barcelona Cathedral and luckily enough those that joined the walk, were rewarded with the evening meeting meal as the power started to return to Spain!

The second day begun with virtual attendees now being able to join the meeting with updates provided from work package 2 and Professor Mimoun Azzouz’s recent IND approval from the FDA for SPG47 gene therapy. Dr Sol Ruiz Head of Biologics and ATMP, AEMPS joined remotely to provide an ATMP regulatory update before our panel discussion followed. ARDAT’s PPIE group coordinator Dr Amy Hunter from Genetic Alliance UK, Dr’s Sandra Silva and Christian Brander from GRIN and Dr Francina Munell from Vall d’Hebron University Hospital involved in DMD clinical trials sat on the panel discussing the personal perspectives and the unmet needs of treatment and care for patients and families with rare diseases. The proposed no-cost extension was presented and voted on by consortium members, with final work package breakout sessions providing the opportunity for members to discuss plans for fulfilling remaining tasks and deliverables in the final part of the project.

Overall, despite the challenges the meeting was a great success with lots of discussion and plans for going forward. Thank-you to all involved!