MOHAMED BENDERDOUR

Topic nanovectors for targeted gene therapy of cutaneous psoriasis

Challenge: Psoriasis is a chronic inflammatory skin disease that manifests itself as scaly patches, especially on the elbows, knees and scalp. The skin undergoes a profound modification of the epidermis, with an infiltration of inflammatory cells and increased proliferation of skin cells. Inflammatory modulators called Interleukin (IL) -17 and -23 have been identified as key factors in this disease. These … Read More

MASSIMILIANO PAGANELLI

The Encapsulated Liver Tissue as a treatment for acute liver failure: proof-of-concept efficacy and safety in large animals using liver organoids composed of primary cells

Challenge: Acute Liver Failure (ALF) is an extremely severe, progressive syndrome resulting from a sudden insult on the liver that exceeds the organ’s innate regenerative capacity. No effective treatment exists for ALF except for liver transplant, which is limited by organ shortage, heavy morbidity and need of lifelong immunosuppression treatments. Temporary replacement of liver functions while the patient’s own liver … Read More

TULLY MICHAEL UNDERHILL

Platform to target small-molecule therapeutics to bone – Novel prodrugs to selectively deliver therapeutics to bone

Challenge: Musculoskeletal diseases represent the second largest contributor to disability worldwide (WHO). Despite efforts made in the past few years, drugs development in bone therapy is challenging. Indeed, bone is a difficult tissue to drug due to its material properties and limited vascularity, requiring high drug doses and frequent administration to achieve efficacy. Most FDA approved bone therapeutics are focused … Read More

*NATHAN YOGANATHAN

Developing New Alzheimer’s Therapeutics Using a Novel Carrier That Can Cross the Blood-Brain Barrier

Challenge: Of the top 10 most fatal illnesses in the world, Alzheimer’s disease is the only one for which there is currently no disease-modifying therapy available. Abnormal clusters of toxic amyloid protein fragments build up between nerve cells in the brain leading to impaired memory. The current therapies in development are biologics, antibodies and peptides that neutralize toxic amyloid. However, … Read More

*DERRICK GIBBINGS

Engineering Endogenous Vesicles as a Drug Delivery Platform to Inhibit Gene Expression Using microRNAs and Silencing RNAs

Challenge: Silencing RNAs (siRNAs) are small nucleic acids that can inhibit virtually any gene. Because most diseases could benefit from shutting-down the action of a specific protein encoded by its gene, it is suggested that siRNAs could be used to treat virtually any diseases. However, naked siRNA is unstable in the bloodstream and cannot efficiently cross cell membranes. So far, … Read More

*FRÉDÉRIC CALON

Intracellular delivery of antibodies and peptides using a peptide-based technology

Challenge: The popularity of therapeutic proteins has recently increased mostly due to their broad applicability, but the druggable space for biologics is still restricted to extracellular targets. Indeed, the number of potential targets either secreted or with extracellular domains represents only one-third of the proteome, thus significantly restricting attainable pathways. In parallel, intracellular delivery methods are still plagued by inefficiency … Read More

*MICHEL MEUNIER

Novel Non-Invasive Laser-Assisted Intraocular Drug Delivery System for an Efficient and Selective Gene Transfer Therapy

Challenge: Retinal degenerative diseases, such as age-related macular degeneration and glaucoma, are the leading causes of vision loss and affect tens of millions of individuals in the world. Emerging solutions with nucleic acids and therapeutic genes show promise. However, there remains a void in effective and non-invasive drug delivery systems for the back of the eye, which has hindered the … Read More

*SYLVAIN MARTEL

Magnetically Guided Bacteria to Specifically Deliver Anti-Cancer Agents to Colorectal Cancer Sites

Challenge: Many cancers are treated with cocktails of chemotherapeutic drugs designed to prevent growth and spread of cancerous cells. Chemotherapy damages healthy cells, causing undesired side effects such as myelosuppression and cardiotoxicity. Localized drug therapy, if appropriately delivered could have a major impact on quality of life and treatment efficacy. Solution: The lead investigator has pioneered a revolutionary drug delivery … Read More