Data-Gouv-ML/pcri-erc-participations-et-projets-laureats huggingface dataset:
Projet ERC : IMPACT (Horizon Europe, appel 2024, Proof of concept grants)
Entité participante : University Medical Center Utrecht — Pays : Pays-Bas — Rôle : PI
Acronyme de l'entité : UMC
Type d'entité : Enseignement supérieur ou secondaire
Financement ERC de l'entité : 150000.0 €

Rare genetic diseases affect over 30 million people in Europe. Although individually rare and very diverse, they are all caused by genetic mutations. Recent progress in genetic technologies now holds the promise to correct the root cause of these diseases. However, it remains difficult to develop these innovative technologies into affordable therapies for these diseases that only affect few patients, limiting commercial interest.  IMPACT aims to overcome the translational gap between scientific gene editing and in human gene correction therapies that will be affordable and readily available for patients with rare genetic diseases.  To overcome this gap, we will use a multifaceted approach to set up the infrastructure for a gene correction therapy for Methylmalonic Acidemia (MMA) by elucidating and addressing the essential (1) regulatory, (2) manufacturing, and (3) financial requirements. This approach involves preclinical testing of the therapy in a perfused ex vivo human liver, clarifying the intellectual property strategy, and involving experts, stakeholders, and end users to define the regulatory, manufacturing, and financial strategies required for academic development and clinical implementation. As such, IMPACT aims to valorise the innovative gene correction strategy developed with the ERC Starting Grant to deliver prime editing tools as mRNA encapsulated in lipid nanoparticles to correct the disease-causing mutation in the liver of patients with MMA.  With IMPACT, we do not only aim to develop gene correction therapies in our academic hospital for MMA. Through our efforts in generating the infrastructure for a therapeutic platform approach with only slight adaptations to target different mutations, we ultimately aim to develop therapies for the many patients with different genetic diseases.

Mots-clés : gene correction therapies,clinical translation,regulation,affordable therapies
Projet ERC : DeepSight (Horizon Europe, appel 2022, Proof of concept grants)
Entité participante : Université de Tel Aviv — Pays : Israël — Rôle : PI
Acronyme de l'entité : TAU
Type d'entité : Enseignement supérieur ou secondaire
Financement ERC de l'entité : 150000.0 €

The number of road accidents continue to increase dramatically each year. Cyclists and pedestrians, being a part of the daily traffic, keep challenging drivers thus making safety issues increasingly important. Furthermore, miniature electrical vehicles, paving their way through traffic and being in many cases unseen by drivers, put themselves in danger. Improving driving safety, being always a subject to technological efforts, is one of the most important challenges of the modern society. Continuously developing technologies offer a broad range of tools, capable to assist a driver in a decision making. Moreover, autonomous vehicles, nevertheless facing a broad range of challenges nowadays, will certainly sooner or later contribute to the endeavor. Optical camera solutions, LIDARs and high-frequency radars are already demonstrating exceptional assistance in driving safety by providing overlapping and complementary data. However, all existent safety systems require having an unperturbed line of sight to obstacles. This is a severe limitation, which cause those systems to overlook numerous dangerous events, including pedestrians, stepping on a road from behind a parked vehicle and many others. MHz and lower GHz frequencies are known to penetrate material bodies and diffract around obstacles - e.g. we have a Wi-Fi signal in office, nevertheless the router is placed behind a wall in a corridor. However, low frequency waves are never used in automotive applications owing to low range and angular resolutions they can grant, as it is commonly believed. While this statement is almost a ground truth in the field, it can be bypassed if a preliminary information on an object does exist. This includes and not limited to rotating bicycle wheels, human breath, and several others. Here we will develop a low-frequency radar for detecting cyclist and pedestrians with a resolution, sufficient to ensure a road safety and assisting drivers to make right decisions.
Projet ERC : Treat-PRYSM (Horizon Europe, appel 2024, Proof of concept grants)
Entité participante : University Medical Center Utrecht — Pays : Pays-Bas — Rôle : PI
Acronyme de l'entité : UMC
Type d'entité : Enseignement supérieur ou secondaire
Financement ERC de l'entité : 70000.0 €

Rupture of intracranial aneurysms (IA) results in aneurysmal subarachnoid hemorrhage (aSAH), an often fatal type of stroke. Invasive IA treatment to prevent aSAH is a high-risk procedure, limiting its use and leaving most IA patients untreated. Thus, there is an urgent need for non-invasive drugs preventing aSAH, but these are lacking as our understanding of the causal disease pathways is poor. The considerable female predominance in IA and aSAH suggests the existence of sex-specific regulation of these pathways. In the ERC PRYSM StG we identified candidate drugs lisinopril, amlodipine, tamsulosin, simvastatin and metformin associated with a reduced risk of aSAH. These targets have an inhibitory effect on the Transforming Growth Factor-β (TGF-β) pathway, a potent inducer of Endothelial-to-Mesenchymal transition (EndMT), a driver of vascular disease. The Treat-PRYSM team’s main aim is to transition the research from the ERC PRYSM StG into innovation by performing the following activities: 1) Confirm and validate our 5 candidate drugs—lisinopril, amlodipine, tamsulosin, simvastatin and metformin—as effective drugs in reducing aSAH incidence, by employing the principle of drug repurposing and triangulating evidence from epidemiological and genetic data. 2) Find genetic evidence that the EndMT and TGF-β pathways are involved in IA pathogenesis, thereby identifying these pathways as potential therapeutic targets for the candidate drugs, using a large-scale genomics approach. 3) Determine whether the mode of action of the candidate drugs and EndMT/TGF-β pathways is influenced by sex. 4) Evaluate the opportunities for a clinical trial of the most promising candidate drug to assess its ability to inhibit growth of IAs, serving as a surrogate marker for rupture. This approach will ultimately provide a non-invasive, cost-effective drug to prevent aSAH in patients with unruptured IAs and will significantly enhance our understanding of the disease's causal pathways.
Projet ERC : BiomeDxRx (Horizon Europe, appel 2023, Proof of concept grants)
Entité participante : Institut Weizmann — Pays : Israël — Rôle : PI
Acronyme de l'entité : WIS
Type d'entité : Enseignement supérieur ou secondaire
Financement ERC de l'entité : 150000.0 €

The problem: Much effort has been directed to identifying disease risk factors and indeed, numerous genetic, lifestyle, anthropometric and clinical risk factors are routinely used for this task. However, the clinical utility of existing risk factors is limited and most of them appear when physiological derangements have already occurred. The gut microbiome plays a role in human physiology and health and as such, combining it with existing risk factors can lead to earlier and more robust disease detection. However, very few microbiome-based markers predictive of disease onset and progression were found to date and none are currently used by healthcare systems.  The solution: In the ERC-funded project, we used our unique multi-omics and longitudinally profiled 10,000-person cohort and biobank to develop methods for metagenome-wide association studies. We found microbial genetic variation at the single nucleotide level and single bacterial genes level whose baseline levels were predictive of future disease onset. In this ERC-PoC, we propose to prioritize the bacteria found in the ERC-funded project (WP1) and use them within machine learning disease diagnostic models (WP2) that we will then license to diagnostic companies. We further propose to build a microbial biobank of the ERC-identified strains by isolating them from cohort participants, functionally characterizing them with metabolomics and combining them into bacterial consortiums (WP3). As these strains have therapeutic potential, we will license them to probiotic or pharmaceutical companies (WP4). Notably, we already validated this approach in two licensing agreements, one with a Japanese-based company who licensed bacteria that we found for treating Atopic Dermatitis, and another with a U.S. based probiotics company who licensed bacteria that we found for weight loss. If successful, our ERC-PoC may thus result in novel microbiome-based diagnostic and therapeutic products for various disease indications.
Projet ERC : KITZYME (Horizon Europe, appel 2023, Proof of concept grants)
Entité participante : Université de Gérone — Pays : Espagne — Rôle : PI
Type d'entité : Enseignement supérieur ou secondaire
Financement ERC de l'entité : 150000.0 €

Enzymes exhibit high efficiency, specificity, selectivity, biodegradability, non-toxicity, and the ability to function effectively under gentle biological conditions. These qualities render enzymes a sustainable and eco-friendly substitute for traditional catalysts within industrial settings. However, harnessing enzymes for industrial applications often necessitates extensive and costly experimental engineering efforts. Computational methods hold promise as potential solutions, but these have not yet demonstrated the ability to rapidly design highly efficient enzymes that mimic those found in Nature. As opposed to other methods, our computational approach developed in the previous ERC-STG can introduce active site and distal mutations that modulate the enzyme conformational dynamics, achieving increases in catalytic efficiency of up to 1000-fold. This project aims to exploit the proprietary technology developed in ERC-STG and ERC-POC for generating a set of patentable rationally designed enzyme kits focused on stereoselective carbon-carbon bond formation. KITZYME aims to create a spin-off for the exploitation of the new set of rationally designed enzyme kits as well as the proprietary technology developed for enzyme optimization. Both the enzyme kits and our technology are the cornerstone of the project, and will provide industries with a cost-effective, scalable, and environmentally sustainable solution.
Projet ERC : TOP-EVICARE (Horizon Europe, appel 2023, Proof of concept grants)
Entité participante : University Medical Center Utrecht — Pays : Pays-Bas — Rôle : PI
Acronyme de l'entité : UMC
Type d'entité : Enseignement supérieur ou secondaire
Financement ERC de l'entité : 150000.0 €

More than 3.5 million people are newly diagnosed with heart failure every year in Europe with a long-term prognosis of 50% mortality within 4 years. There is a major need for more innovative, regenerative therapies that have the potential to change the course of disease. In the ERC-Extracellular Vesicle-Inspired CArdiac REpair (EVICARE), I hypothesized that we can recondition heart failure by stimulating cardiac repair with extracellular vesicles that are derived from progenitor cells. From the EVICARE application results, however, we realized that enhancing the therapeutic load of EVs will enhance their functional effect size to treat chronic diseases. In TOP-EVICARE (Technology Of Protein delivery in Extracellular Vesicle-induced Cardiac Repair), I aim to explore active loading and release systems that will allow protein therapeutic loading in extracellular vesicles to stimulate myocardial repair. Within this project, I aim to: I. Validate our innovative EV-loading strategy by assessing biodistribution and functional delivery in vivo, and exploring the potential for upscaling GMP production to meet industry standards and requirements; II. Conduct a comprehensive intellectual property (IP) landscape analysis to establish freedom-to-operate (FTO) and ensure that our technology can be commercialized without infringing on existing patents; III. Execute an in-depth market, competitor, and stakeholder analysis to identify strategic opportunities and potential challenges in the EV-based therapeutic landscape; and IV. Develop a robust and detailed business plan to effectively guide the commercialization of our loading technology, thus bringing a transformative solution to the treatment of various diseases.
Projet ERC : CACure (Horizon Europe, appel 2022, Proof of concept grants)
Entité participante : Institut Weizmann — Pays : Israël — Rôle : PI
Acronyme de l'entité : WIS
Type d'entité : Enseignement supérieur ou secondaire
Financement ERC de l'entité : 150000.0 €

Cancer-associated cachexia (CAC) is a multifactorial syndrome causing significant morbidity and mortality in cancer patients and indicates a poor prognosis. Nearly 80% of cancer patients experience CAC, which directly accounts for almost 20% of all cancer deaths, mainly in patients with pancreatic adenocarcinoma (PDAC). Clinically, CAC manifests as weight loss and wasting of skeletal muscle mass. Thus, CAC patients experience reduced physical, emotional, and social well-being, leading to increased use of healthcare resources. The massive phenotypic manifestations point to a significant imbalance in the systemic metabolism of CAC patients. Unlike starvation, CAC cannot be reversed by standard nutritional interventions, and currently, there are no biomarkers for identifying patients at risk for CAC and no effective therapeutic modalities. With our ERC (#818943), we identified changes in liver metabolism during extra hepatic carcinogenesis. We now demonstrate that these metabolic changes occur early, precede CAC, and can be diagnosed by routine liver biochemical profile. We generated a biochemical liver score that can identify PDAC patients at risk for CAC, irrespective of disease stage at diagnosis. Furthermore, we find that these metabolic changes in the liver are mediated by early infiltrating immune cells that lead to HNF4a depletion in hepatocytes independent of liver metastasis. Importantly, we find that re-expressing HNF4a with Adeno Associated Virus (AAV8) preserves liver metabolism in PDAC mouse models and alleviates CAC. We hence hypothesize that generating liver-specific mRNA of HNF4a will be highly therapeutically beneficial for PDAC patients identified by our liver score to be at risk for CAC. In this PoC we will generate and test the efficiency of novel HNF4a-mRNA-LNPs in preventing or alleviating CAC in PDAC mouse models. When successful, our optimized construct will undoubtedly serve identified PDAC and other cancer patients at risk for CAC.
Projet ERC : P3D Endoscopy (Horizon Europe, appel 2022, Proof of concept grants)
Entité participante : Université de Tel Aviv — Pays : Israël — Rôle : PI
Acronyme de l'entité : TAU
Type d'entité : Enseignement supérieur ou secondaire
Financement ERC de l'entité : 150000.0 €

The proliferation of artificial intelligence (AI) has led to a major advancement in many fields. It has enabled achieving non-precedent achievements such as human-level image recognition, intelligible language translation, and accurate medical diagnosis. The main research goal of the PI’s ERC-StG SPADE project is to use tools for data modelling to improve our understanding and usage of deep learning (DL) and thus enhance its performance in medical problems and computational imaging [1-4]. One of the fields that could greatly benefit from such progress is sensitive medical treatment, using AI-based medical devices. Such devices can initially assist the physicians with a more accurate diagnosis, and potentially save lives. As such technology matures, the information provided to the physicians will be richer, thus, leading to more accurate diagnoses to the cases at hand. Such advancement will allow high quality and efficient treatment to a much wider public. The scope of this POC is to apply intelligent optical imaging methods developed in the SPADE project, specifically, for introducing reliable 3D and polarization information to medical imaging devices.
Projet ERC : YourFutureU (Horizon Europe, appel 2024, Proof of concept grants)
Entité participante : Société Max-Planck pour le développement des sciences — Pays : Allemagne — Rôle : PI
Acronyme de l'entité : Société Max Planck
Type d'entité : Recherche
Financement ERC de l'entité : 150000.0 €

This PoC action harnesses the potential of artificial intelligence (AI) to enhance future orientation and mental well-being, and to mitigate self-defeating behaviors. Building on recent evidence that future-oriented thinking —a key factor in academic performance, health, wealth, and goal achievement— can be stimulated, we aim to address the scaling and impact-related challenges of existing interventions. Leveraging the power of AI-powered large language models (LLMs), our proposed solution is to develop an intelligent conversational agent that allows for meaningful interaction with one's future self. This agent will utilize user-specific inputs —such as personality traits and other individual characteristics— to create a backstory of their personal history, i.e., a synthetic memory, and enable a highly personalized and relatable conversation between users and their future, i.e., 10-year older, self.  Frequent dialogues with the future self instill a more vivid image of the future self and increase the psychological connection to it. This in turn increases the motivation and ability to consider the longer-term consequences of decisions and reduces the likelihood that people opt for immediate gratification. This effort is facilitated by features known to enhance decision-making: perspective taking, a vivid mental image of the future, and the ability to distance oneself from situations.  Originating from our ERC CoG action, in which we used virtual reality technology and smartphone apps to generate future-self avatars, we have begun to see promising results, such as higher levels of engagement and a demonstrated capacity to stimulate future orientation as well as symptom reduction in depression and suicidal ideation. Expanding on this prior work, we aim to develop an innovative solution that integrates the behavioral sciences and computer science, making mental health care more affordable and accessible while addressing self-defeating behaviors in diverse populations.
Projet ERC : NIBIOX (Horizon Europe, appel 2022, Proof of concept grants)
Entité participante : Donostia International Physics Center — Pays : Espagne — Rôle : Partner
Acronyme de l'entité : DIPC
Type d'entité : Recherche
Financement ERC de l'entité : 20000.0 €

The vast majority of industrial chemical oxidations catalyzed by heterogeneous catalysts (chemical and enzymatic) in aqueous solvents suffer from several technical hindrances related to diffusion restrictions of oxygen This limitation derives from the low transport rate of molecular oxygen from the gas phase to the solvation sheath on the surface of the solid catalyst where catalysis occurs. The restriction of molecular oxygen availability at the reaction point creates a bottleneck in the productivity of industrial processes. To overcome a such major limitation in industrial productivity of oxidative biocatalysis, we propose the use of a new generation of heterogeneous biocatalysts based on oxidoreductases immobilized on polymeric matrices for the oxidation of alcohols, replacing the molecular oxygen with water soluble inorganic salts as ultimate electron acceptor. The results of NIBIOX will be exploited in the fine chemicals industry. Oxidative reactions are one of the pillars of the organic synthesis toolbox. For example, the global market for aldehydes is expected to reach more than $2 billion by 2025. This means a total global market of $232 billion in industrial fine chemistry by 2027. Therefore, the successful outcome of NIBIOX and its implementation in industrial processes will result in a profitable business with a positive impact on the economy of the stakeholders involved in the exploitation of our solution.
Projet ERC : TRANSIONICS (Horizon Europe, appel 2022, Proof of concept grants)
Entité participante : CERCA Institution — Pays : Espagne — Rôle : PI
Acronyme de l'entité : CERCA;I-CERCA
Type d'entité : Recherche
Financement ERC de l'entité : 150000.0 €

Neuromorphic computing will revolutionize artificial intelligence for applications such as autonomous driving, smart diagnosis or natural-language understanding by emulating the operation of efficient biological neural networks. The main challenge in this field is the substitution of conventional transistors for synaptic transistors able to learn in ways similar to a neural synapse, i.e. the development of multistate non-volatile transistors. However, currently existing synaptic transistors have been developed using electrolytes that are by nature unstable and difficult to integrate such as ionic liquids or proton conducting polymers. TRANSIONICS will deliver highly stable (non-volatile), silicon-compatible and scalable solid state synaptic transistors by exploiting the first-ever room temperature oxide-ionic electrolyte developed in the ERC CoG grant (ULTRASOFC) held by the PI. TRANSIONICS transistors are able to modulate its channel properties with external stimulus like real neurons by reducing/oxidizing a mixed ionic-electronic conductor unveiled at ULTRASOFC. Additionally, TRANSIONICS is compatible with mainstream microelectronics fabrication technology, which makes it ideal for developing high density brain-like computer chips.  The goals of the TRANSIONICS project are i) to evaluate the technical feasibility for the fabrication of unique all-solid-oxide synaptic transistors with lateral architecture; ii) to assess the silicon- compatibility and scalability of the TRANSIONICS transistors; iii) to define an IPR strategy for technology transfer; iv) to build a value proposition for a startup company and to identify customer segments with industrial partners. To achieve these goals, the PI has joined around the project team that combines applied research, technology transfer and market uptake expertise.
Projet ERC : BiofilmEradicate (Horizon Europe, appel 2022, Proof of concept grants)
Entité participante : Institut Weizmann — Pays : Israël — Rôle : PI
Acronyme de l'entité : WIS
Type d'entité : Enseignement supérieur ou secondaire
Financement ERC de l'entité : 150000.0 €

Bacterial biofilms are widespread, both as infections in humans and on devices that have come in contact with infected tissue, and pose a major health and economic burden. Such biofilms are difficult to treat as they present strong resistance to drug delivery, due to a dense, hydrated polysaccharide/protein matrix in which the bacterial communities encase themselves, and from the bacterial membrane itself. Liposomes stabilized by poly(ethylene glycol), PEG moieties, the current gold-standard and most commonly used means of stabilization, are versatile drug-delivery vehicles, but such PEGylation is associated with significant shortcomings, greatly reducing the efficiency of PEGylated liposomes for biofilm treatment. Here we explore a novel strategy to improve such drug delivery and treatment of biofilms. In particular, is it effective? is it efficient? Can it be readily applied both in vitro (for which we have some promising preliminary indications) - as for infected biomedical devices - and, crucially, in vivo? What is the IPR position for future exploitation of this new drug delivery strategy? These questions illustrate the high-risk/high-gain nature of this proposal, and are systematically addressed in this proposal through several inter-related work packages. Success of our project in demonstrating that our novel drug-encapsulating vehicles can efficiently treat and eradicate bacterial biofilm infections would not only have benefits affecting large populations, but also tap into a large drug-delivery market.