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August 08, 2019
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Regenxbio’s wet AMD gene therapy shows positive phase 1/2a results

RGX-314, Regenxbio’s gene therapy using NAV vectors for AAV-mediated antibody delivery, has shown positive phase 1/2a results in patients with wet age-related macular degeneration, continuing to be well tolerated across five dose cohorts, according to a press release.

Forty-two subjects have been treated in the trial, and dose-independent increases in RGX-314 protein expression levels, measured from aqueous samples by electrochemiluminescence immunoassay, have been observed at all dose levels after 1 month of treatment, the release said.

“We are encouraged by the continued positive interim data from the phase 1/2a RGX-314 trial and the potential of NAV gene therapy as a one-time treatment for wet AMD, and look forward to sharing 6-month results from all five cohorts in the trial in October 2019,” Kenneth T. Mills, president and CEO of Regenxbio, said in the release.

The company also announced second quarter revenues of $7.9 million compared with $40 million in the second quarter of 2018. A net loss of $1.5 million was also recorded for the quarter compared with a net income of $10.6 million a year ago.

As of June 30, Regenxbio had cash, cash equivalents and marketable securities totaling $449.7 million.