Frequently asked questions
Our FAQs provide easy-to-understand definitions of complex concepts, helping you stay up-to-date on the latest developments in the field.
What is a biomolecule?
A biomolecule, or biological molecule, is a molecule that is produced by cells and living organisms. Biomolecules have a wide range of sizes and structures and perform a vast array of functions. The major types of biomolecules are nucleic acids (DNA and RNA), proteins, carbohydrates, and lipids.
What are synthetic biomolecules?
Synthetic biomolecules are artificially manufactured biomolecules that mimic the properties and actions of naturally-occurring biomolecules. They possess great significance in the development of new medicines, as they can be specifically designed for their intended purpose
What is a peptide?
A peptide is a short chain of amino acids (typically 2 to 50) that are linked by peptide bonds.
What is a stapled peptide?
Stapled peptides are short peptides that contain two unnatural amino acids that are coupled together by a so-called Ring Closing. The location, orientation, and number of staples that can be applied to a peptide are very numerous, allowing for great flexibility in design options even for a single peptide sequence. This design strategy was developed for peptide-based products to be able to reach, and interact with, intracellular targets that were previously considered undruggable.
What are Enzyme Replacement Therapies (ERTs)?
ERTs are used to treat lysosomal storage disorders where a deficient lysosomal enzyme results in the toxic build up of a specific substance. Current treatments have limitations due to low ERT uptake by cells. We believe our technology can improve cell entry to maximize the efficacy of the treatment.
Of the known rare genetic diseases, Lysosomal Storage Diseases (LSDs) constitute a group of around 70 diseases that are the main application of ERT. Each LSD is individually rare but collectively they affect many individuals, with an estimated incidence of around 1:6,000 live births. Approximately 70% of these LSD cases represent unique highly progressive neurodegenerative diseases, highlighting the sensitivity of the central nervous system to lysosomal dysfunction.
ERT works by replacing a missing or deficient lysosomal enzyme. It is relatively safe and effective in prolonging life in high-risk patients, but it is not a cure. Patients typically need lifelong treatment, either weekly or biweekly intravenously over several hours.
Some tissues, such as cardiac muscle, cartilage, and especially skeletal muscle, have low levels of the receptors needed for ERT uptake. This results in low ERT uptake by the cells in these tissues. If any of these also represent the target tissue for the therapy, then proportionately high doses of the ERT are needed with more frequent dosing, resulting in much higher costs and greater risk of side effects.
Sutura is designing cell-penetrating peptides (CPPs) to overcome the cell entry barrier in order to greatly reduce the large amounts of drug required. Our unique approach has the potential to significantly improve the efficacy of ERT while reducing the dosage needed, ultimately benefiting patients afflicted with LSDs. We look forward to sharing more about our progress and breakthroughs in this crucial area of biomolecular medicine.