FDA applies brakes on gene therapy trial

[Farooqji]

Neurocrine Biosciences & Voyager Therapeuticsa announcethat the FDA has put a clinical hold on their gene therapy RESTORE-1 trial (evaluating NBIb-1817/VY-AADC) in Parkinsons, pending DSMB request for review of additional data in early 2021

ir.voyagertherapeutics.com/...

[jimcaster]

That's not encouraging, but I don't know how discouraged to be. 🤔

[kaypeeoh]

Not directly applicable, but last year I needed surgery to place a stent in a coronary artery. Before the operation they gave me a flu shot. I imagine similar attitude with the covid vac.

[MarionP]

Well that's curious. I wonder what the deal is. This is one of the most promising candidates.

[sharoncrayn]

To those very few who are interested in this CT:

" The clinical hold follows the submission by Neurocrine Biosciences of an IND Safety Report related to the observation of MRI abnormalities in some RESTORE-1 study participants. The clinical implications of this observation are currently unknown and are being evaluated."

Pretty obvious the AEs were serious (MRI defined!!) and probably increasing. "Some" is the undefined operative word. I never saw this gene therapy model (the so-called AADC theory) as "one of the most promising" unless every PD CT is "most promising". Very few are because PD is a multiple variable disease.

Until they find out why the AEs really occurred, this CT is DOA.

Sharon

[Parkinsonjisung in reply to sharoncrayn]

Sharon, what do you consider the most promising CT for parkinson?

[Farooqji]

This is not the first bump in the road for the RESTORE-1 study. The trial had previously been paused due to the ongoing COVID-19 pandemic and was also paused at the request of the independent Data Safety Monitoring Board seeking additional time to review additional data. The DSMB has requested additional patient level data from the trial and now plans to review these data in early 2021, Voyager said in its announcement.biospace.com/article/voyage...