StemCell Therapy

The FDA designates Orphan Drug-tagged ProQR Therapeutics' (NASDAQ:PRQR) QR-110 for Fast Track review for the treatment of Leber's Congenital Amaurosis Type 10 (LCA 10), a genetic disorder characterized by the progressive loss of vision in the first few years of life. It is caused by a mutation that results in abnormal splicing of messenger RNA (mRNA) which results in the non-functioning of a key protein called CEP920.

QR-110, an RNA-based oligonucleotide, is designed to restore the normal splicing of messenger RNA to enable fully functioning CEP920 to be produced. It is administered via intravitreal injections in the eye.

Fast Track status provides for more frequent interactions with the FDA review team and a rolling review of the New Drug Application (NDA).

Top-line results from an early-stage, open-label study, PQ-110-001, should be available next year.

Shares are up3%premarket but only on 70 shares.

More:
ProQR's QR-110 Fast Track'd for rare pediatric blindness; shares ahead 3% premarket - Seeking Alpha

This entry was posted on May 31, 2017 at 7:42 pm and is filed under Blindness. You can follow any responses to this entry through the RSS 2.0 feed. Both comments and pings are currently closed. |