Our definitions glossary
The process by which a licensed pharmacist or qualified healthcare professional combines, mixes, or modifies ingredients to create a customised medication for an individual patient. Compounding provides tailored dosage forms and formulations that are not commercially available, addressing specific patient needs such as allergies or unique dosage requirements. This is key to Nanospresso's mission of producing personalised nanomedicines at the bedside, enabling patient-specific treatments for rare diseases that are not feasible through industrial manufacturing.
A rare genetic disorder caused by a deficiency or complete lack of the enzyme uridine diphosphate glucuronosyltransferase (UGT1A1), which is essential for processing and eliminating bilirubin. Without this enzyme, bilirubin accumulates, causing severe jaundice that can lead to irreversible neurological damage if left untreated. Early interventions such as phototherapy or liver transplantation are crucial for managing this condition.
Cystic Fibrosis An inherited genetic disease resulting from mutations in the CFTR gene, which regulates the movement of salt and water in and out of cells. These mutations lead to the production of thick, sticky mucus, particularly affecting the lungs and digestive system, causing chronic respiratory infections and digestive problems. Modern treatments, including CFTR modulators, have significantly improved outcomes for people with cystic fibrosis.
A severe X-linked genetic disorder characterised by mutations in the dystrophin gene, resulting in the absence of the dystrophin protein essential for muscle function. This condition primarily affects boys and causes progressive muscle degeneration and weakness, often leading to loss of mobility by adolescence and a shortened lifespan. Early physiotherapy and novel gene therapies are critical in managing symptoms and improving quality of life.
Lipid-based nanoscale vesicles that encapsulate therapeutic agents such as mRNA or siRNA, facilitating their delivery into cells. LNPs protect these nucleic acids from degradation and enhance their cellular uptake, which is crucial for therapies like mRNA vaccines (e.g., COVID-19 vaccines) and gene silencing technologies. NANOSPRESSO utilises LNPs to deliver personalised nucleic acid-based therapeutics effectively.
A technology involving the manipulation of fluids within microchannels typically less than 1 millimetre in size. This precise control allows for high-throughput and automated processing of tiny fluid volumes, making it useful in diagnostics, chemical synthesis, and drug formulation. Microfluidic devices are critical in point-of-care diagnostic tools due to their speed and accuracy. Nanospresso employs microfluidic technology to produce personalised nanomedicines efficiently at the bedside.
A single-stranded nucleic acid that carries genetic information from DNA to the ribosome, where proteins are synthesised. mRNA serves as a template for assembling amino acids in the correct order to produce specific proteins. It plays a crucial role in modern therapeutics, such as mRNA vaccines and gene therapies, which are forms of nucleic acid-based therapeutics. Nanospresso focuses on mRNA-based treatments for personalised medicine.
The application of nanotechnology to innovate healthcare by utilising materials at the nanoscale (1–100 nanometres). These unique properties enable new diagnostic tools, targeted treatments, and regenerative solutions. Nanoparticles can cross biological barriers, interact with biomolecules, and precisely target diseased tissues, impacting fields like cancer, cardiovascular, and neurodegenerative diseases. With over 80 products on the market and hundreds in clinical trials, nanomedicine enhances personalised and cost-effective healthcare. NANOSPRESSO leverages nanomedicine to produce personalised treatments for rare diseases.
Nucleic acid-based therapeutics are treatments that use DNA or RNA to either modify the activity of certain genes or correct genetic mutations. Examples include gene therapy, antisense oligonucleotides, small interfering RNA (siRNA), and mRNA vaccines. These therapies are promising for treating conditions caused by genetic defects, such as muscular dystrophies or some cancers. NANOSPRESSO specialises in developing these therapeutics to meet the unique needs of each patient.
A healthcare approach that customises treatment based on an individual's genetic profile, lifestyle, and environmental factors. By considering patient-specific characteristics, personalised medicine aims to maximise treatment efficacy and minimise adverse reactions. For instance, targeted cancer therapies are tailored to the genetic mutations present in a patient's tumour. NANOSPRESSO embodies personalised medicine by producing customised treatments at the point of care.
The on-site manufacturing of medical products, such as pharmaceuticals or diagnostics, at the location of patient care (e.g., hospitals, clinics). This approach allows for immediate availability and customisation of treatments, particularly beneficial in emergency situations or for rare diseases that require specific formulations. NANOSPRESSO utilises point-of-care production to manufacture personalised nanomedicines directly within hospital settings.
A microfluidics technique involving the controlled and accurate combination of fluids at microscopic scales. Precision mixing ensures uniformity and consistency in producing complex formulations, such as nanoparticles, by enabling precise control over reaction conditions. This is particularly useful in the development of drug delivery systems and diagnostic tools. NANOSPRESSO employs precision mixing to ensure consistent and high-quality production of nanomedicines at the bedside.
Diseases that affect a small percentage of the population. In the European Union, a disease is considered rare if it affects fewer than 1 in 2,000 people; in the United States, fewer than 200,000 individuals. These conditions are often chronic, progressive, and life-threatening, with limited treatment options available. Examples include cystic fibrosis, Duchenne muscular dystrophy, and Gaucher disease. NANOSPRESSO focuses on developing treatments for rare diseases through personalised nanomedicine.
A genetic disorder characterised by the degeneration of motor neurons in the spinal cord and brainstem, leading to progressive muscle weakness and atrophy. It is commonly caused by mutations in the SMN1 (Survival Motor Neuron 1) gene, affecting the production of a protein essential for motor neuron survival. Early intervention with gene therapy or SMN-enhancing drugs can improve motor function and prolong life.
Drugs that are designed to specifically interact with molecular targets involved in disease progression, such as proteins or receptors. By focusing on these targets, such therapies offer higher efficacy and reduced side effects compared to conventional drugs. Targeted therapies are particularly common in oncology, where they aim to attack cancer cells without damaging healthy tissue. NANOSPRESSO aims to produce targeted medicines through personalised nanomedicine approaches.
A gap in current medical capabilities where existing treatments or diagnostics are inadequate for effective management of a health condition. Addressing unmet clinical needs often drives innovation in drug development and medical devices, as it highlights the necessity for improved patient outcomes in areas like rare diseases or chronic pain management. NANOSPRESSO aims to fill these gaps by developing personalised treatments for rare diseases like Crigler-Najjar Syndrome and methylmalonic acidemia.
