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VX983
- Question
- Hello
After VX770, VX809 and VX661, VERTEX announces a clinical trial with VX983 (in combination with Kalydeco™-VX770). This clinical trial concerns patients homozygous for deltaF508. Could you tell me more?
Could you tell me if VERTEX has made the deltaF508 mutation its "workhorse" or if there are other researches on other mutations (except the G551D of course) ongoing?
Thank you for answering me
- Answer
- Hello
VX983 is a corrector like VX661 and VX809. VX983 acts on CFTR protein in order to allow it to reach the cellular surface.
Currently, according to information available on the site Vertex (investors.vrtx.com/releases.cfm?hdr02=press) VX983 is evaluated in healthy volunteers.
Indeed, the company plans for the second half of 2013 a test that combines VX770 and VX983 in patients homozygous for deltaF508. No additional information is available to date on the new test.
VERTEX has not made the deltaF508 mutation its "workhorse".
The laboratory realizes clinical trials regarding patients with gating mutation (as G551D): G178R, G551S, S549N, S549R, G970R, G1244E, S1251N, S1255P, or G1349D.
There is also a clinical trial for patients with R117H mutation.
Those tests are done with Kalydeco® (VX770).
Best regards,
Anna Ronayette
- 10.05.2013
