In February, Dutch rare diseases biotech firm ProQR Therapeutics (Nasdaq: PRQR) lost 74% of its market value in a single day after the failure of its pivotal Phase II/III Illuminate trial of sepofarsen.
In the CEP290-mediated Leber congenital amaurosis 10 (LCA10) study, the investigational RNA therapy did not meet the primary endpoint of best corrected visual acuity (BCVA) at month 12.
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