Encouraging Gene Therapy News

[jimcaster]

The primary headline relates to increased dosage, but also refers to earlier reports which we have previously discussed.

"Early three- and six-month data on that first group was reported to be encouraging, with AXO-Lenti-PD being both well tolerated and patients showing better motor function.

One year after receiving the gene therapy, these patients continued to show gains, including a 37% improvement in off-period motor symptoms (periods when anti-parkinsonian medication wears off) and a better quality-of-life.

These two patients also had a 22-point positive change from baseline (study start) on the Unified Parkinson’s Disease Rating Scale (UPDRS) Part II “Off” score, which assesses daily life activities. This improvement was higher than what was observed with ProSavin, another Parkinson’s gene therapy treatment candidate that Axovant evaluated in a separate Phase 1/2 study (NCT01856439)."

parkinsonsnewstoday.com/202...

[RooJr]

Thanks for posting!

[WinnieThePoo]

Another really interesting and promising area of research

[Hidden in reply to WinnieThePoo]

Sharon in 3,2....

[jeffreyn in reply to Hidden]

Actually, this time it's me!

I was somewhat discouraged by this item in the Study Record on the Clinical Trials website:

"Estimated Study Completion Date: December 2031"

clinicaltrials.gov/ct2/show...

[Hidden in reply to jeffreyn]

You are far too delicate a communicator to step in for sharon.

Gotta be a typo, tho?

[jeffreyn in reply to Hidden]

You would think so, given that the Estimated Primary Completion Date is June 2022. OTOH, they have had more than enough time to correct it.

[MarionP]

I didn't see whether there is a double blind to part B and measurement technicians for part B who are different than part A.