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Today, the American biotechnology company Delani announced some patient data for its Hunter syndrome (also known as MPS II) drug DNL310 Phase I clinical trial
.
A total of 17 patients were enrolled in this trial, and today released data on biomarkers such as heparin sulfate and neurofilament protein in 5 patients in group A at 24 weeks
Drug source analysis
Hunter's disease is one of the lysosomal storage diseases.
It is a rare genetic disease with an incidence of about one in 100,000
.
The patient is unable to degrade complex polysaccharides because of the IDS gene mutation, which causes these substances to accumulate in a variety of tissues
The Denali product line is all drugs for senile degenerative diseases, and the target search mainly relies on human genetic data
.
The targets of degenerative diseases are mostly in the center.
The decrease in the level of CSF heparin sulfate indicates that the drug may indeed partially enter the center.
This in itself is an important technological breakthrough for enzyme replacement therapy, but whether this level can be converted into benefits remains to be verified
.
Neurofilament protein is a protein that leaks to the outside of the cell after a cell is injured.
Denali was founded by former Genentech executives.
It is a very high-profile biotechnology company that raised a record IPO that year
.
Their most mature project is the Parkinson's disease drug LRRK2 inhibitor.
