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And the moment arrived, the group that wins the ZonMw Early Career Challenge is.... Group 3! Tessa Prins (ExoVectory), Federico Cocozza (Utrecht University) and Despoina Trasanidou (Sanquin)! Congratulations!!! #ZonMw #ExoVectory #Sanquin @utrechtuniversity.bsky.social

LNPs that are potent in vitro but often not in vivo, therefore it is necessary to optimize in order to have a functional platform for drug delivery and clinical translation. Important! tiny changes in the lipid molecules of LNPs can result in a 1000-fold higher delivery! #NVGCT2025

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Our second Keynote is Prof. Raimond Schiffelers from the University of Utrecht (@utrechtuniversity.bsky.social). He will delivered a talk on Optimizing Lipid Nanoparticles for Nucleic Acids Delivery. #NVGCT2025 #LNPs #nanoparticles

Next in line is a selected abstract by Peiyu Li from the #Amsterdamumc ( @amsterdamumc.bsky.social ) who will be presenting on the use of airway organoids to evaluate gene silencing in the patient context. #NVGCT2025

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Next Session is on Cellular platforms and we start with Nicola Fattorelli from VIB-UAntwerpen, and he is talking about human microglia xenografts to model disease and develop cell therapies. #NVGCT2025

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We reach the Diamond Sponsor presentation. NecstGen is a non-profit organization aiming to reduce the costs of gene and cell therapy. In this presentation Diederik Lokhorst from #Viralvector is guiding us on how to do the experimental design for different factors using #lentivirus

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Last pitch comes from Tessa Prins (ExoVectory), Federico Cocozza (Utrecht University) and Despoina Trasanidou (Sanquin) who combine their expertise and passion into one project. The final winner will be announced at the end of the meeting! Congratulations to all the participants!! #NVGCT2025 #ZonMw

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Next in line are Stefan Kint and Grigory Kornienko from #Exovectory and #Amsterdamumc @amsterdamumc.bsky.social, respectively

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The time has come... 3 groups are pitching for the ZonMw Early Career Challenge Award... First in line are Peiyu Li from #VUMC and Yue Chen from #AMC @amsterdamumc.bsky.social

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Every year the NVGCT awards the Greiner award for the best thesis conducted in the Netherlands on gene or cell therapy field. This year the winner is Qiang Wang from #LUMC who did the thesis in the group of Dr. Manuel Gonçalves. We would like to thank #Greiner for their continuous support!

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This work highlights how a therapy was developed in 8 months! This poses chances for a platform for n-of-1 studies, however the conventional drug development is way longer, at least in the EU. Can this be sped up?

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the last presentation of this session is given by Stijn van Breda Vriesman from @umcutrecht.bsky.social who will be talking about the regulatory perspective for gene editing. He will be talking about the recently breakthrough paper on personalized based editing work: www.nejm.org/doi/abs/10.1...

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The next speaker is Karin Pike-Overzet from #LUMC who will be talking about a clinical trial for RAG1-SCID patients using hematopoietic stem-cell therapy combined with lentiviral therapy. #SCID #NVGCT2025 #clinicaltrial #RAG1

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The next speaker is selected from the submitted abstracts and is Deja Porenta who is working on assessing the Cas9-directed immune response in mice at the @utrechtuniversity.bsky.social #NVGCT2025 #LNPs #Cas9 #nanoparticles

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The next session is on Genetic Technologies, and the first speaker is Prof. Sabine Fuchs (from @umcutrecht.bsky.social). She will talk about prime editing for inherited metabolic diseases. #NVGCT2025 #metabolicdiseases #primeediting #liver

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To end, he give the look to the future. Once again, congratulations to Prof. Vincenzo Cerullo for receiving this NVGCT award for his contribution to the field! #NVGCT2025 #award #oncolyticvirus #peptides #cancer

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for that a peptichip was created as otherwise many cells were needed. Still after finding the peptides they need to be tested for immunogenicity

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while generating a batch of virus is very expensive. So in that sense having a regular virus decorated with peptides decreases the price. But how to find these peptides?

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Other examples are PeptiENV (using again oncolytic viruses) or PeptiBAC (using Bacteria) modified with the peptides. By combining peptides with the virus or bacteria you can make the product cheaper because generating the peptide to decorate the virus is very cheap and controlled

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So basically an oncolytic virus has become an anti-tummor vaccine, steering the Ferrari in the desired direction. The delivery also was improved to deliver subcutaneously and intratumoral, developing a stronger response. The results of the clinical trial will be presented at the #ESGCT 2025

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Oncolytic viruses are good vehicles but need improvements. Prof. Cerullo has been guiding us on how to built these viruses by comparing them to a Ferrari. But how he did that? For example by anchoring a tumor-specific peptide, which led to PeptiCRAd (currently in clinical trials).

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However, before starting his presentations he had a clear message to the quizzmasters of the famous NVGCT pubquiz "Ricotta is a fantastic cheese but is not included in Lasagna or Pasta Alfredo" Statement from an Italian-dominated team #NVGCT2025

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This year the the NVGCT Outstanding Award is awarded to Prof. Vincenzo Cerullo, who has been working on precision cancer oncolytic immunotherapy and will provide a presentation "Dressing up viruses to fool cancer: fast pipeline for personalized cancer vaccines" #NVGCT2025 #award #oncolyticvirus

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We are back for the second day of our Annual Meeting. This year 22 early career researchers participated in the ZonMw challenge. 10 of them found a match and 3 groups managed to submit a proposal. Later today they will participate in the final round. Good luck to all of them!

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The last presentation of this session and today is given by Olivier de Jong (@utrechtuniversity.bsky.social) on delivery and release strategies for CRISPR/Cas9 system using extracellular vesicles #EVs #NVGCT2025 #CRISPR

It is great to see so many AAV-based approaches with market approval! #NVGCT2025

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We have the honor of having today Prof. Ahad Rahid from @ucl.ac.uk who is talking about Gene Therapy for Lysosomal Disorders. Special thanks for the effort to join today given the current situation with trains in the Netherlands. In particular he will talk about Niemann-Pick type C gene therapy.

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The next session on delivery started with the invited lecture of Roy van der Meel (@tue.nl) to talk on nanotechnology for delivery of RNA, small molecules, protein to immune cells by systemic delivery using #LNPs #NVGCT2025

the last speaker of this session is Martina Paladino from @utrechtuniversity.bsky.social and she will explain her postdoc project on the lipid nanoparticles delivery to induce dopaminergic neurons both in vitro and in vivo #NVGCT2025 #LNPs #nanoparticles #neurons

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Thus, personalized approaches like n-of-1 approaches at academic level might be the future. #NVGCT2025 #DCRT @dutchantisense.bsky.social @oligogirl.bsky.social

Our next speaker is Anne-Lot van Esbroeck from the @erasmusmc.bsky.social who is going to present on the development of #antisense therapies for brain diseases. In particular Rare Diseases, which the rarity of the disorders complicates significantly the chances of a treatment.

This approach could be also used in adults, but probably the neurogenesis part will be less effective in adults #NVGCT2025 Good news is that a clinical trial phase II has been granted!

After the fantastic keynote lecture of Prof. Heine, we move forward to the next session on therapy development for neurological disorders. The first speaker is Cora Nijboer from @umcutrecht.bsky.social who will present on intranasal stem cell therapy for brain injury for newborns.

is very crucial as well as the readout and the aspect of the disease you want to use. Overall, iPSC are great models in neuroscience. It is always important to use a model that recapitulates as much as possible the in vivo phenotype, therefore complexity is important.

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But how these models can help therapeutic development? For example for ALD there is a therapy, so using disease-specific tissues novel biomarkers can be identified, allowing diagnostic, prognosis and therapeutic efficacy evaluation. But always very important, the choice of model ...

Therefore, it is always very important to decide which model to use and which readout to use. As the more complex the model is, the more complex the phenotyping will be. In her group they focus on monogenic neurodevelopmental disorders.

In her research they are using several models, organoids allow to study the brain development, the spheroids in contrast not. Co-cultures are very useful 2D models that allow many functional readouts and have homogeneous cultures, however, the more cells are added, those readouts change

After the networking lunch, we are welcoming our first keynote speker: Prof. Vivi Heine from @amsterdamumc.bsky.social who will be talking about the use of iPSC models for modeling disease and assess therapeutics

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This year the #ZonMW challenge has a new set up and we hope that we will increase the excitement! Saty tuned to hear the winning team tomorrow!

Every year #ZonMw sponsors the early career challenge, where early career researches match their expertise to create a feasible project. We heard how Athina Mavoproulo and Alba Montoro Canelo use the prize in research, attend the #ESGCT2024 and how their careers are at the moment.

We had the honor to have Cees Smit who presented his book "Gene and Cell Therapies in Europe". This book describes the breakthroughs in ATMPs, offering real prospects of cures. #NVGCT2025

@revvity.bsky.social @miltenyibiotec.bsky.social Once again, thank you very much for your support and hope you enjoy the experience at #NVGCT2025 #sponsor #sponsorpitch

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We could not have our annual symposium without the support of our sponsors. We are hearing pitches of how their products can help our research. A big big thanks to all of them for their continuous support. @biomerieux.com @cytiva.bsky.social

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To finish the #DARE-NL session, Trudy Straetemans is back on stage to present how DARE-NL can help us preparing and accelerating a first in-human clinical study. #NVGCT2025