ProQR Raises $90m To Advance Eye Disease Pipeline
Lead Candidates For LCA10 And Usher Syndrome
The Dutch biotech has firmed up its finances as its promising pipeline of RNA therapies for rare genetic eye disease starts to move through the clinic.
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The company is pinning hopes on its Usher syndrome and retinitis pigmentosa programs, but the disconnect between Phase I/II success and Phase II/III failure dented analysts’ enthusiasm.
While rivals Intellia impressed with their first in vivo CRISPR gene-editing data, Editas’s first results in just two patients are not enough to convince investors.
ProQR’s Axiomer platform can enable single nucleotide RNA editing, an approach that could address thousands of disease-causing genetic mutations, the company says.