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Regulatory cell therapy raises tremendous hope to get rid of long-term immunosuppression, and ensuing infectious and neoplastic complications, especially in solid organ transplant recipients. However, the first clinical studies have yielded mixed results. The challenge of isolating and expanding don

Premature stop codons (PTC), UAA, UAG and UGA, are responsible for 12% of genetic diseases. PTCs readthrough strategies aim to restore the synthesis of a complete and functional protein. Cystic fibrosis, which may be due to CFTR mutations linked to PTCs, is a good model to evaluate these strategies.

Proteins serve as primary targets for most pharmaceutical interventions, yet the therapeutic focus on disease-related proteins, which represent a mere fraction of the human genome, limits the scope of medicinal chemistry. Expanding the target landscape to include coding and non-coding RNAs offers a

The project seeks to show the importance of neutralizing the extracellular action of the AGR2 protein (eAGR2) in the pathophysiology of inflammatory intestinal diseases (IBD) through the use of a specific antibody developed by French Biotech Thabor Therapeutics. AGR2 is an endoplasmic reticulum-resi

Gaucher disease results from mutations that alter the enzyme beta-glucocerebrosidase, involved in the metabolism of glycosphingolipids. This defect in lipid homeostasis affects macrophage function. The phenotypic expression of the disease is variable but predominantly affects the liver, spleen and b

The incidence of allergies has dramatically increased over the last three decades in industrialized countries, with an estimate of 15% to 30% allergic patients in Europe and the US. Moreover, the World Health Organization now estimates that half of the people will develop allergies by 2050. Despite

Diseases of the central nervous system (CNS) could be eligible for biodrugs but the blood-brain barrier (BBB) remains the main obstacle for their delivery in the CNS. Metastatic breast cancer is a leading cause of mortality in women and particularly the brain metastases. Although trastuzumab (TZB) h

Type 1 diabetes (T1D) is an autoimmune disease caused by the progressive loss of tolerance towards antigens of insulin-producing pancreatic beta cells leading to their destruction and chronic hyperglycemia. The steeply rising disease incidence suggests an urgent need to develop new treatment strateg

Glomerulonephritis (GN) is a group of immune-mediated diseases and a major cause of end-stage renal disease. Antineutrophil cytoplasmic antibody (ANCA)–associated vasculitis (AAV) and lupus nephritis (LN) are autoimmune GN. Glucocorticoids and immunosuppressive drugs are used to control autoimmune G

Evidence accumulated over the past decade shows that long non-coding RNAs (lncRNAs) are widely expressed and exert key roles in gene regulation. Recent studies have begun to unravel how the biogenesis of lncRNAs is distinct from that of mRNAs and is linked with their specific subcellular localizatio

Opioid analgesics remain the most effective drugs for moderate to severe pain, but while their efficacy is undisputed in acute and cancer pain, their efficacy for chronic pain on the long term is insufficient, being partly impaired by the development of analgesic tolerance and paradoxical hyperalges

Mucosal vaccination, especially nasal administration, constitutes a strategic approach because i) induced immune responses can be both mucosal and systemic, ii) active ingredients are not exposed to extreme gastrointestinal pH or digestive enzymes, iii) numerous microvilli in the nasal epithelium pr

At present, while there are innovative non-invasive biomarkers developed to diagnose acute rejection, there are no sufficiently reliable non-invasive biomarkers to assess the lesional state of the graft. Serum creatinine is not specific for renal allograft injury and does not clearly distinguish los

Nuclear imaging is now a key component of patient management. Monoclonal antibodies are the carriers of choice for molecular imaging, due to their exceptional affinity and selectivity for their target antigen. However, due to their large size, they diffuse poorly into tissues and are slowly eliminat

MEROU aims to develop new biocoatings covering medical devices (MD) to 1) prevent bacterial and fungal infections and 2) promote the healing process. These biocoatings will be constructed from a combination of multilayers of antibacterial polypeptide polymers incorporating innovative nanoclusters (N

The clinical importance of antibiotics remains beyond question. However, their over- and mis-uses have created a strong selective pressure on bacteria, which induced the emergence of (multi)resistant strains. Antibioresistance is becoming so pregnant that since 2017, the World Health Organization (W

Deep eutectic solvents (DESs) are currently experiencing a rapid expansion in pharmaceutical development, particularly due to their ability to increase the solubility of poorly water-soluble molecules. Among the various potential routes of administration, the topical cutaneous and the transdermal ro

Autism spectrum disorders (ASD) are severe neurodevelopmental conditions with an increasing prevalence and currently no available cure. Innovative approaches for ASD treatment are thus critically needed. Gastrointestinal dysfunction, microbiota dysbiosis, and abnormally elevated levels of microbiota

2in1-ADRC project aims at the development of a new class of therapeutic agents combining advantages of Antibody Drug Conjugates (ADC) and those of Targeted Radiopharmaceuticals (TRP) and compensating for their respective weaknesses, in order to ensure selective delivery of the killing payload at the

Flaviviridae such as Dengue virus (DenV), West Nile fever virus (WNV) and Yellow fever virus (YFV) are known to infect liver and brain to various degree leading to either hepatitis and/or encephalitis. Early steps and organs responses to the infection are very poorly documented due to lack of effici

The specific correction of RNA splicing using small molecule is an emerging field of drug discovery. The specific correction of gene expression by modulating RNA splicing has begun to provide therapeutic solutions against inherited conditions. In this context, we have deciphered the mode of action o

The aim of the project is to implement a new technology for the controlled delivery of molecules in the brain. Several brain disorders are not efficiently treated, due to the very low accessibility of the drugs to the diseased brain area. To encounter these limitations, intracranial pharmacotherapy

The COVID-19 pandemic caused by SARS-CoV-2 has highlighted the urgent need to identify antiviral compounds against human coronaviruses (HCoV). The multidisciplinary CoroNat2 project involves 4 complementary teams, each with pre-existing technical expertise in virology, phytochemistry, chemistry and

Tertiary lymphoid structures (TLS) are ectopic lymphoid tissues that drive immune responses at sites of chronic inflammation. They are of widespread interest in biology and medicine since their presence dramatically influences disease course in autoimmune disorders, infection, and cancer. However, d

Lysosomal acid lipase (LAL) deficiency is a rare, autosomal recessive condition caused by mutations in the gene encoding for LAL. Wolman disease (WD) is the most severe form with an incidence of 1 in 300.000 livebirth, resulting in failure to thrive, hepatomegaly, and hepatic failure, and a life exp

mtDNA depletion syndrome (MDS) is a group of mitochondrial disorders characterized by a reduced mtDNA copy number which occurr in early childhood and for which no curative therapy is yet available. Depletion of mtDNA result from mutations in nuclear genes especially involved in the regulation of mit

The objective of this project is to rationally design a preclinical candidate optimized for the treatment of autism spectrum disorder (ASD). ASD is a complex neurodevelopmental disorder diagnosed in presence of primary symptoms, namely impaired social communication and interaction together with a re

Pathogenic bacteria are a permanent threat for Humanity even the discovery of antibiotics led to the fade-out of mass epidemies. Nevertheless, human being faces more and more antibiotic-resistant bacterial strains. Thus, the constant development of innovative antibiotics is crucial, especially again

XIAP (X-chromosome-linked inhibitor of apoptosis protein) is involved in various cellular processes, such as apoptosis and the immune response. It also coordinates a series of events leading to the production of pro-inflammatory cytokines. Recently, the key role of XIAP in the NOD signalling pathway

Resistance to antibiotics is a main threat for human health. New drugs and treatment strategies are urgently needed. Ideal next generation antibiotics will interfere with bacterial virulence or with the resistance mechanisms restoring the bacteria sensitivity towards a given antibiotic. We have rece

The DAMUNO project aims to transform four animal venom-derived peptides into therapeutic candidates by minimizing their intrinsic immunogenicity, a significant hurdle for their clinical development. We have selected four molecules with distinct canonical folds—Kunitz, three-finger, and knottin—which

Plasmodium falciparum (Pf) is responsible for the most lethal form of malaria, which is a major health threat in tropical and subtropical countries. In 2021, the World Health Organization estimated the number of malaria deaths at 619,000 and 247 million cases of malaria worldwide, the latter having

The challenge of this project is the development of a multimodal treatment against thrombosis (stroke) using innovative theranostic inorganic nano-objects. Our approach is based on the design of biodegradable nanomaterials of core@shell type (iron oxide@porous silica with large pores) allowing a loc

Recent studies have shown that a single administration of psilocybin, the active compound of magic mushrooms, has a fast-acting and long-lasting efficacy in treatment-resistant depression. Psilocybin is also well-known to induce acute psychedelic effects, characterized by important changes in self a

Antimicrobial resistance to antibiotics is a serious threat to the human health. In this frame, the WHO identified the ESKAPE pathogens (E. faecium, S. aureus, K. pneumoniae, A. baumannii, P. aeruginosa, and Enterobacter spp.) as critical targets for drug discovery. They are the leading cause of nos

The polycystic ovary syndrome (PCOS), the main cause of women infertility, associates reproductive and metabolic disorders. The daughters of women with PCOS mainly suffer from the clinical signs of hyperandrogenism. The treatments of adult and adolescent women with PCOS, are only symptomatic and can

The current therapeutic options for chronic pain are limited and often come with significant side effects, including the potential for addiction. To address this challenge, the MyPainkiller project focuses on harnessing a natural product derived from actinomycetes, known as mycolactone, which has de

Infections caused by Non-Tuberculous Mycobacteria (NTM) are on the rise worldwide, not only in cystic fibrosis and immuno-compromised patients, but also in immune-competent individuals. These infections represent a major healthcare challenge with treatment using current antibiotics remaining less th

The considerable and rising impact of respiratory diseases worldwide, such as the COVID-19 pandemic, highlights the need to improve in vitro models to predict better and treat such diseases. The Print@Lung project aims to develop a physiologically relevant in vitro model of the human lung for studyi

Transposable elements (TEs) are DNA sequences capable of moving within the genome and represent nearly 50% of the human genome compared to only 3 to 4% for protein-coding genes. In cancer, TEs are hypomethylated and it has been shown that some of them can encode peptides that are presented by HLA cl

Alzheimer’s disease (AD) is the most common neurodegenerative disorder associated with dementia, cognitive decline and memory loss. AD is characterized by the pathological formation of intraneuronal aggregates of tau protein and extracellular aggregates of amyloid ß (Aß). The view of the brain as an

Antisense Oligonucleotides (ASO) and gene therapy approaches hold great promise for the treatment of genetic diseases and Duchenne muscular dystrophy (DMD) in particular. Some of these therapeutic strategies have even reached market approval, however none of the currently approved therapies addresse

Chimeric antigen receptor (CAR) cell therapy represents a ground-breaking advancement in cellular immunotherapy, showcasing remarkable clinical efficacy in hematological cancers. However, its broader application is hindered by several challenges, with the primary obstacle being the low specificity o

Precise gene editing based on the homology-directed repair (HDR) repair mechanism holds great promise to manipulate the genome with the goal to correct gene mutations or insert specific DNA sequences. However, several hurdles remain, such as the poor efficiency of HDR-mediated events. Furthermore, i

Despite being preventable, diagnosable and treatable, malaria is still responsible by the highest number of deaths caused by vector-borne diseases. Recently, vaccines targeting sporozoites displayed elevate efficacy against clinical malaria, but more efficient vaccine are needed to block transmissio

Despite the improvements made in treating cancers during the past decades, resistance to classical chemotherapeutic agents continues to be a major problem in cancer therapies. Indeed, drug resistance is responsible for most relapses of cancer, one of the major causes of death of the disease. Therefo

Clostridioides difficile (Cd) and Clostridium perfringens (Cp) are often involved in severe intestinal infections. Both species secrete toxins that severely damage the intestinal epithelium. These infections are generally treated with antibiotics. However, resistant strains are emerging worldwide an