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In total, there are 6,700 rare diseases worldwide, forming a community of more than 300 million people.
Do you know what's behind these beautiful names?
Butterfly Baby: Hereditary epidermolysis bullosa. People with this disease are called butterfly babies because their skin is as fragile as their wings.
Mermaid Syndrome: A rare congenital deformity of the lower limbs in which patients are born with adhesions on the inside of their legs that look much like the tail of a mermaid.
Glycosaminoglycan baby:a rare X-linked recessive genetic disorder. It is caused by a mutation in the gene responsible for producing iduronate-2-sulfatase (IDS), an enzyme critical for breaking down glycosaminoglycans (GAGs). Without this enzyme, GAGs accumulate in tissues and organs, leading to multi-organ dysfunction. It’s also called Mucopolysaccharidosis type II (MPS II).
Especially, we notice that the frequency of MPS II is approximately 1 in 100,000 births in China, which has a very high incidence rate compared with other countries. And based on China's population base, the population of MPS II patients in China is extremely large.
MPSII type patients are more likely to develop the disease in childhood, and they will experience joint and bone lesions, facial changes, growth and development problems, ear, nose, and throat problems, etc.
And the youngest sick child is only 1.83 years old, and the patient usually dies within the age of 20.
An engineered exosome, carrying the IDS enzyme, can enter the tissues of patients with MPSII to reduce the storage of GAGs, thereby relieving the patient's pain. ExoCureIDS have a higher efficiency and a stronger ability to penetrate the biofilm barrier.
On the basis of pLVX-IDS-Exosignal, I carry a TAT that can
penetrate the BBB
and alleviate damage to the central nervous system.
Our Design
Learn more ......
May the future sing with melody, children's lives bloom like poetry, and health be the timeless rhythm that guides us all.
Protocol
Engineering
Results
Parts
Human Practices