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Spark Therapeutics, Inc. (ONCE)

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$ONCE One of the 9 needed an infusion to control a suspected knee bleed even though the p.has a FIX level of 36%.
Spark Therapeutics with exclusive worldwide rights to Selecta’s proprietary Synthetic Vaccine Particles (SVP™) platform technology for co-administration with gene therapeutics directed at the FVIII gene for hemophilia A as well as exclusive options for up to four additional undisclosed genetic targets.

George does say one huge asterisk remains: about 40 percent of hemophiliacs can’t be helped yet. That’s because the type of virus used in this therapy is similar to one that naturally infects people. The result: many people have virus-grabbing antibodies in their blood that would intercept Spark’s treatment on its way to the liver. Such patients have been excluded from the study. “It's an obstacle to calling this an off-the-shelf treatment,” says George. High is working on ideas to overcome the problem, including releasing decoy viruses to soak up the antibodies.

Het management van Spark verkoopt lekker hun eigen aandelen,,AB,JB*,OB,OE*,OE,S,AS,JS*,OS,OS*,3,JB,JS&o=-1&a=
Hemophilia B Gene Therapy - Spark $ONCE AAV8-hFIX19: This study has been terminated.

AAV8-hFIX19 terminated. SPK9001 recruiting.

Dogder schreef op 20 dec 2016 om 18:29:

Het management van Spark verkoopt lekker hun eigen aandelen,,AB,JB*,OB,OE*,OE,S,AS,JS*,OS,OS*,3,JB,JS&o=-1&a=

Gevalletje van voorkennis?
Ik zou het wel denken want steriods werken niet tegen een immuunrespons, als ik kijk naar de Baxalta trail
Impact of AAV Capsid-Specific T-Cell Responses on Design and Outcome of Clinical Gene Transfer Trials with Recombinant Adeno-Associated Viral Vectors: An Evolving Controversy $ONCE (+ $QURE etc.)

$ONCE #HemophiliaB SPK-9001 update: Friday, April 7.
Spark Therapeutics Presents Updated Preliminary Data from Hemophilia B Phase 1/2 Trial Suggesting Consistent and Sustained Levels of Factor IX Activity at the Hemostasis and Thrombosis Research Society (HTRS) 2017 Scientific Symposium
As of data cutoff, the annualized bleeding rate (ABR) has been reduced by 96 percent and the annualized infusion rate (AIR) reduced by 99 percent
Both participants who began a tapering course of steroids have completed their regimen
PHILADELPHIA, April 06, 2017 (GLOBE NEWSWIRE) -- Spark Therapeutics (NASDAQ:ONCE) announced updated preliminary data today from 10 infused participants in the ongoing Phase 1/2 clinical trial of investigational SPK-9001 for hemophilia B. All participants have experienced consistent and sustained increases in factor IX activity following administration of the investigational therapy. These data will be presented at the Hemostasis and Thrombosis Research Society (HTRS) 2017 Scientific Symposium in Scottsdale, Arizona on Friday, April 7, by Adam Cuker, M.D., assistant professor of medicine at the Perelman School of Medicine of the University of Pennsylvania and a clinical investigator at Children’s Hospital of Philadelphia.
Data as of March 24, 2017 will be presented on 10 participants in the study, who were dosed with a single administration of 5 x 1011 vector genomes (vg)/kg body weight. All participants have discontinued routine infusions of factor IX concentrates. Based on individual patient history prior to the study, ABR was reduced by 96 percent to a mean of 0.39 annual bleeds, compared with 9.2 bleeds before SPK-9001administration. AIR was reduced 99 percent to a mean of 0.98 annual infusions, compared with 68.5 infusions before SPK-9001administration. 
As of the data cutoff, nine of the 10 infused participants have not taken factor IX concentrates to prevent or control bleeding events since vector administration. As previously reported, one participant with severe joint disease has self-administered precautionary infusions for persistent knee pain. The mean steady-state factor IX activity level post 12 weeks treatment for the 10 participants was a sustained 33 percent (range as of the data cutoff: 14 to 81 percent). In the study to date, no serious adverse events have been reported, including no factor IX inhibitors and no thrombotic events.These data represent more than 2,400 cumulative patient days of exposure from the start of the trial.
Two of the 10 participants experienced an asymptomatic, transient elevation in liver enzymes, or decline in FIX activity, potentially indicative of an immune response to the Spark100 vector capsid, that occurred several weeks post infusion. Both participants received a tapering dose of oral corticosteroids, after which their alanine aminotransferase (ALT) levels returned to baseline. The activity level of one of these participants has stabilized at approximately 15 percent for more than nine weeks post corticosteroid use. The other participant had a factor IX activity level between 70 to 80 percent at completion of steroid use.
“The additional preliminary data continue to support our initial observations that a single intravenous administration of SPK-9001 has resulted in consistent and sustained levels of factor IX activity for trial participants,” said Katherine A. High, M.D., president and chief scientific officer at Spark Therapeutics.  “Notably, all participants to date have consistently achieved our targeted therapeutic range of FIX activity. As we continue to glean more insights from these preliminary data, our analysis suggests that a tapering course of oral corticosteroids has been well-tolerated and may help control potential capsid immune responses following SPK-9001 infusion.”
These data from the Phase 1/2 clinical trial of SPK-9001 will be presented during a poster session on Friday, April 7, from 5:15-6:15 p.m. MST.
$ONCE announces a new 5-year installment payment plan for #Luxturna at #BIOCEO to pave the way for the next gen of gene therapies, commercial chief Ron Philip says. 50% collected upfront, 25% about 30-60 days post procedure. Remaining 25% over five year increments.
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