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Spark Therapeutics, Inc. (ONCE)

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was nog geen draadje over, dus bij deze.

Ik denk dat het goed is om ook Spark te volgen om een bredere blik te verkrijgen op gentherapy..

Vandaag dit bericht:

finance.yahoo.com/news/spark-therapeu...



* het interessante aan dit bericht vind ik, dat leaders in gentherapy vaker uitgenodigd kunnen worden om trials te starten met derden, omdat zij pioniers zijn in de gentherapy, dus vooruitlopen op vele concurrenten.

Deals zoals die met BMY of Pfizer kunnen zich vaker voordoen en zorgen voor een brede basis met vele kansen in de toekomst. Met de fabriek van QURE lijkt QURE zich ook al meer dan anderen voorbereid te hebben op -big business-. QURE, Spark e.d. kunnen zich opmaken voor grote investeringen van andere grote biotechs/pharma's nu gentherapy echt van de grond lijkt te komen.
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Spark Therapeutics and Clearside Biomedical Announce Exclusive Option to License Technology for Potentially Differentiated Delivery of Gene Therapy to the Eye
Companies to explore potential of Clearside's microinjector technology for use in delivering gene therapeutics to the retina and choroid through the suprachoroidal space
PR Newswire Spark Therapeutics, Inc.
17 hours ago

????

PHILADELPHIA, April 28, 2015 /PRNewswire/ -- Spark Therapeutics, Inc. (ONCE) and Clearside Biomedical, Inc. announced today that they have entered into an option agreement under which Spark acquired exclusive rights to license Clearside's microinjector technology to deliver gene therapies to the back of the eye. Under the agreement, the companies will explore the feasibility of using Clearside's microinjector technology to deliver viral vectors to the choroid and the retina through the suprachoroidal space (SCS).

If successful, this technique could create a differentiated alternative to sub-retinal or intravenous administration for future gene therapy applications, potentially broadening the range of conditions treatable through gene therapy.

"We believe gene therapy has tremendous potential to provide one-time, life-altering treatments to patients with debilitating, monogenic blinding conditions, and development of an alternative delivery approach that broadly covers the retina would be an important step in further unlocking this potential," said Jeffrey D. Marrazzo, co-founder and CEO of Spark. "As we build a pipeline of gene therapies to treat inherited retinal dystrophies, we believe Clearside's proprietary technology and intellectual property estate for delivery of therapeutics to the SCS may allow us to target new diseases. If successful, the microinjector technology may provide an alternative to current routes of administration, provide ease of delivery for physicians and further expand on our leadership in the ocular gene therapy field."

Using a proprietary microinjector, Clearside's technology delivers therapeutic agents to the retina and choroid through the SCS and without substantial diffusion to the vitreous of the eye. Clearside is currently conducting a Phase 2 clinical trial in macular edema associated with uveitis and a Phase 2 clinical trial in macular edema associated with retinal vein occlusion (RVO). In a recently concluded Phase 1/2 trial, subjects with non-infectious uveitis treated with a single suprachoroidal injection of a commercially available formulation of triamcinolone acetonide, using Clearside's proprietary microinjector, showed improvement in best corrected visual acuity ranging between one and five lines (or up to 25 letters). The procedure was generally well tolerated in this trial and no meaningful intraocular pressure (IOP) increases were observed in any subjects through the six month duration of the trial.

"Clearside is delighted to be working with Spark, a clear leader in applying gene therapy to treat blinding diseases," said Daniel White, CEO and president of Clearside. "We believe this agreement highlights the important potential of precision delivery to propel the field of ocular gene therapy forward. By reaching ocular tissues that we would otherwise not reach, we are creating the potential to treat a range of debilitating ocular conditions."

If Spark exercises its option, in return for exclusive, worldwide rights to use Clearside's microinjection technology and related intellectual property in the field of gene therapy, Spark will pay to Clearside an upfront licensing fee, development related milestones and commercial royalties on sales of Spark's products covered by the licensed technology.
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4:52 pm Spark Therapeutics and Clearside Biomedical announce entrance into an option agreement, for Spark to acquire exclusive rights to license Clearside's eye gene microinjector technology Briefing.com 17 hrs ago
Secondary Offerings Outnumber IPOs by 9 to 1 in 2015 Q1 24/7 Wall St. q 18 days ago

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About Spark Therapeutics

Spark is a gene therapy leader seeking to transform the lives of patients suffering from debilitating genetic diseases by developing one-time, life-altering treatments. Spark's initial focus is on treating orphan diseases where no, or only palliative, therapies exist. Spark's most advanced product candidate, SPK-RPE65, which has received both breakthrough therapy and orphan product designation, is in a fully enrolled pivotal Phase 3 clinical trial for the treatment of rare blinding conditions. Spark is leveraging the experience and technology utilized in the development of SPK-RPE65 to address a broad spectrum of blinding conditions, starting with the development of SPK-CHM for the potential treatment of choroideremia, currently in a Phase 1/2 clinical trial. Spark also is establishing a pipeline of gene therapy candidates to treat hematologic disorders and neurodegenerative diseases, including through a global collaboration with Pfizer Inc. around the development and commercialization of its SPK-FIX program for the treatment of hemophilia B. Spark's integrated gene therapy platform builds on two decades of research, development and manufacturing at The Children's Hospital of Philadelphia, including human trials conducted across diverse therapeutic areas and routes of administration. To learn more, please visit www.sparktx.com.

About Clearside Biomedical

Clearside Biomedical, Inc., headquartered in Alpharetta, GA, is a clinical-stage biopharmaceutical company developing first-in-class drug therapies to treat chronic, blinding diseases of the eye. Clearside's product candidates focus on diseases affecting the retina and the choroid, especially diseases associated with macular edema. Visit www.clearsidebio.com for more information.

Prof. Dollar
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Recentelijk met niks naar beneden, nu weer met niks omhoog? Het volume stelt ook niks voor.
DrMedicalValue
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Eens. Wellichtmorgen een big sell-off? Gaat het aandeel $QURE achterna? Daytraders & swingers?

Andere verklaring: vorige week een ongerust stukje in The Street over komende ONCE data read-out en door de CEO aangekondigd overleg met FDA over de resultaten. Dit heeft ongerustheid + scherpe koersdaling gegeven (samen met QURE dat die dag ook 8% dipte na 28,14 geraakt te hebben). Vandaag ruim hersteld. Een tweetje van Feuerstein met wat handelaren?

QURE heeft zich nog niet helemaal hersteld, maar staat wel boven zijn 50 daags gemiddelde.....
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Prof. Dollar
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quote:

Dogder schreef op 12 jan 2016 om 17:55:


ir.sparktx.com/phoenix.zhtml?c=253900...

Wat vinden jullie hier van als het gaat om Hemofilie b data?

Ik kan er nog niet echt wijs van worden.

Het geheel vind ik overigens wel overtuigend en concreet gepresenteerd.
DrMedicalValue
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Hemofilie data nog allemaal preklinisch.
ALS het Hemofilie B product werkt geeft het hogere % FIX dan $QURE, maar dat is niet zo relevant, want tot 40% xpressie heb je nog steeds milde hemofilie B.
Grote RISICO is Padua gen en het geoptimaliseerde capside.
Dit geeft (zoals $BXLT heeft laten zien) groot risico op immuunreacties, en wel van het type waar T-cellen bij betrokken zijn en die niet goed te bestrijden zijn met Prednison en de FIX productie beinvloeden.
Ook FIX levels die alle kanten opschieten door het Padua gen.

ONZIN is verder dat 60% van HemoB patienten antistoffen tegen $QURE's AAV5 heeft. Tot nu toe heeft 1/14 patienten antistoffen. Dichter bij 10% dus.

Zou goed kunnen werken, maar er is een groot risico op slechte resultaten zoals Baxalta heeft.
Prof. Dollar
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quote:

DrMedicalValue schreef op 13 jan 2016 om 11:53:


[..]
ONZIN is verder dat 60% van HemoB patienten antistoffen tegen $QURE's AAV5 heeft. Tot nu toe heeft 1/14 patienten antistoffen. Dichter bij 10% dus.
[..]

@DrMedicalValue: Zo las ik het ook maar dacht er wat anders bij: doet QURE er niet verstandig aan dit zelf beter te communiceren? Want is er geen verschil tussen AAV5 en AAV5? De AAV5's van QURE zijn baculo-based en die van anderen mammalian-based. Of heb ik het mis?
DrMedicalValue
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Ja dat is lastig want ik zie niet zo 1,2,3, de relatie tussen de mate waarin je antilichamen hebt en het baculovirus proces.
$QURE heeft op de press conference wel degelijk hierop gewezen, maar idd niet heel duidelijk.
Weet gewoon niet zo goed waar de verschillen vandaan komen en wellicht weet $QURE dat ook niet.....
Spark99
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Interessante webcast (Questions and answers) van Spark vooral de vragen over hemophilia.
flosz
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Question-and-Answer Session
Operator
Thank you. [Operator Instructions] And our first question comes from Cory Kasimov from JPMorgan. Your line is now open.
Cory Kasimov
I guess, first of all, can you talk a little bit about the keys we should be watching for in the first look at the Factor 9 data from your FIX program, and how much follow up there will be when the results come out in the next few months?
Jeff Marrazzo
Sure. I think you heard me say sort of from the beginning, one of the things that we're looking to accomplish is not just of course in long-term expression, we also believe that there is important natural history data from the Netherlands, suggests that wells and above 10%, 10% to 15%, that range change the natural history sufficiently, reducing the prospect of leads. We are obviously looking for consistency in the results. And of course, we are cognizant of the potential for immune responses, and are looking to identify ways to manage that. Further from just an overall principal perspective, it's a dose escalating trial, so we're looking for identifying the dose that we believe can be most effective, and obviously in our view, in our mind lowers better, not only potentially from immune response, but certainly also from a manufacturing perspective.
Cory Kasimov
And then my second question, I understand it's probably a little difficult at this point. But is there anything you can say regarding the similarities or maybe more importantly, the differences of your factor 8 program relative to others out there, especially Biomarin's?
Jeff Marrazzo
So let me take a step back and say we were encouraged by the data that they shared. I think importantly for us they are using a V domain deleted transgene, and the fact that did confer expression is an important principal we are using that same transgene. We also were encouraged that they did not see any inhibitors to that, which is another key point, so those two pieces of information we believe are important pieces of information that directly relate to what we're doing. Obviously it's not lost on you all, they did see elevated ALPs or LFTs in some subjects. I think that question of how that proceeds will be an important one to follow. What we have been focusing on particularly with Factor 8 has been looking at optimized the number of aspects of our specific candidates in order to again for the principal I just said, factoring on Factor 9, we go in with the lowest possible dose.
We believe that has the best chance to either avoid immune responses or to manage them more easily. And also confers an important principle when it comes to manufacturing. So later today what Xavier Anguela as Kathy mentioned is presenting, is data on our lead preclinical candidate. We presented data at ASH in December on an earlier candidate. And we've subsequent made enhancements to that candidate, that actually from the data in ASH, are showing about a two-fold step-up from the data that we showed at ASH, which at that time still we believe is one of the highest levels of people were achieving with an unmodified Factor 8 gene. So I think importantly what we're seeing from that data, and I don't have all the data from all the other preclinical studies off the top of my head.

And I don't believe all of them are disclosed, but if you just look at a benchmark like 50%, which I don't think necessarily is a benchmark you need to get to have a viable product. You look at that benchmark and our preclinical data, we're getting there with the dose of about 3.2 E12 vector genomes, and obviously what Biomarin's preclinical data showed, and now their clinical data showed, is getting there with about 20-fold-plus higher dose. Again that gives you some sense of what we're seeing in our preclinical data, and why we're very encouraged by it.
Operator
Thank you. Our next question comes from Mark Schoenebaum from Evercore ISI. Your line is now open.
John Scotti
It's John Scotti on for Mark. Thanks for taking my questions. Just a few, I guess, I wanted to clarify what you said earlier in response to Cory's question. Is your goal for the Factor 9 brand, would you say the goal is 10% to 15% expression? I want to make sure I heard you right.
Jeff Marrazzo
Keep going.
John Scotti
No. Go for it. I can ask later.
Jeff Marrazzo
Well, I think what we said in general is that what you're really looking for is a certain level that is disease modifying. What those natural history data suggest is that getting above that 12% level reduces the risk to nearly zero spontaneous bleeds. So that's what I would say today. I think just as importantly is the consistency in which you're getting those types of results. As I said I think in the past, and I don't know, John, if we've talked about this directly. But I know I said in the past, getting a 50% level or a 75% level for 20% of the population may or may not be a product. I think you kind of have to look at what you have in your hand at that point. Our overall goal is to get a level that meets and exceeds this modifying bar and does so consistently across many, if not 100% of the population that you can treat and does so ideally without having to manage the immune response.
John Scotti
I guess on RP65 can you characterize a bit more what happened at the pre BLA meeting in March specifically on the labeling issue? Do you think the FDA is medical labeling language that includes all RP65 mutations, or as opposed to just sort of restricted it to LCA? Has the dialogue changed a bit, or is it still consistent?
Jeff Marrazzo
I would say at a high level that the dialogue has continued to advance in a positive construct. But I have shared even as far back as last year, you may recall, that even probably January of last year, we had not yet broached this topic with the FDA. So subsequently now in most type C meetings, and subsequent set of informal calls and then even other discussions along the way, and now this meeting, that I would say that their openness to understanding and discussing the way of science is encouraging to us.
John Scotti
And then my last question is, I guess on the R&D spend in the quarter, the $18 million, should we think about that as a run rate for the remainder of the year? I guess more importantly long-term? Can you run the business while spending under perhaps $80 million to $100 million in R&D annually? Or is it too early to tell?
Jeff Marrazzo
So I think in terms of this year, we haven't given specific guidance quarter by quarter. But as a run rate I think it's probably close. I think the following year is just going to be the function, even maybe late this year, certainly other years, we have a function of how many studies are running that are later in clinical development. Right. So while we do believe that there is, as you all well characterize in your initiation report, that these types of products, product candidates we are going after, have the ability to run relatively small trials. When you go from these early Phase 1 or 2 trials, when you're doing a handful of patients and now you're doing 20, 30 to 50 or something like that, clearly the cost escalates. When we're doing two or three of those in subsequent years, just going back to the way we laid out our vision for 2018, our goal is to be getting the multiple late stage or pivotal trials over the next couple of years. So I would say that you might characterize it as this sort of steady state component is there, but depending upon what trials we're running, there will be obviously increases driven by those trials.
flosz
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Philip Nadeau
And then one last question just a follow up on Cory's. On the hemophilia B data, when it's released, do you have a sense of how long the follow-up will be, and whether in particular you're going to have people who are more than 12 weeks post administration?
Jeff Marrazzo
Yes. So the short answer is yes. And the slightly longer answer is of course, you will have a range of time points. There will be some that are out past that, there may be a few that are not quite at that point. But you'll have a range of time points. But obviously one of the things you do learn in the early part of these trials is the sort of unique pharmacokinetics of the vector. So you can draw certain extrapolation to subjects that are earlier, but the straight answer is yes.
Spark99
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Dit is wat ik er uit opmaak over het bovenstaande artikel. Als ik het goed begrijp kunnen ze maar 20 procent fix behalen om iedereen te kunnen behandelen. Tevens merk ik dat de ceo het wel heel vaak over een immuunrespons heeft bij te hoge dosis en te hoge fix levels boven de 50%. Dat betekend naar mijn mening dat ze op dit moment geen 50% fix levels kunnen behalen zonder een immuunrespons en dat ze het product niet makkelijk kunnen verbeteren. Ik denk dat het door de padua fix komt wat we dus ook zagen bij Baxter. En dat ze productie problemen hebben bij te hoge dosis wat ik ook hoorde bij Biomarin. Iemand hier nog een opmerking over...
flosz
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twitter.com/andybiotech/status/733323...
twitter.com/andybiotech/status/733332...


Spark Adds To Early But Promising Data For Hemophilia Gene Therapy


The race to develop a gene therapy for hemophilia just moved another few feet forward. Spark Therapeutics, which is one of several companies developing a treatment, is the latest to provide its first glimpse of clinical data in human patients.
As with other snippets from rivals like UniQure, of Amsterdam, and BioMarin Pharmaceutical, of San Rafael, CA, the results—interim data from an early-stage clinical trial—look promising. Investors have bumped Spark (NASDAQ: ONCE) shares up 13 percent as of mid-day. But many questions remain.
No fewer than seven groups are working on a long-lasting treatment for hemophilia using gene therapy. The field has been on a roller-coaster ride for more than two decades, but the lure for drug developers is strong. Hemophiliacs, especially those with a severe form who need frequent infusions of blood-clotting drugs to prevent dangerous bleeding episodes, would benefit from a new standard of care.
That benefit will only arrive if gene therapies safely produce meaningful levels of the clotting factor that patients lack. “Meaningful” doesn’t necessary mean normal. In fact, many in the field believe that simply boosting Factor IX (in severe hemophilia B patients) or Factor VIII (in severe hemophilia A patients) to a fraction of normal levels—5 percent of normal is a threshold commonly cited—would make a big impact. And the higher the number, the better the potential result.
Spark reported today that its hemophilia B treatment, SPK-9001, showed early promise toward that goal. Spark reported on three patients: One had Factor IX levels of 28 percent of normal after 18 weeks, another had 30 percent after seven weeks, and a third had 16 percent at three weeks.
Spark said their levels rose consistently through the first four weeks after treatment, and that while one patient got a precautionary infusion two days after treatment because of a suspected ankle bleed, the other patients haven’t needed additional treatment since the gene therapy has kicked in.
Spark also noted that the patients have not required immunosuppressive steroids to counteract the immune reaction that other gene therapy programs in hemophilia have triggered.
The data come with a number of caveats, starting with the tiny sample size of three patients. The results will have to hold up for a long time, without complications, to be truly meaningful. Such tiny samples would typically not merit as much attention, but in a field of new medical exploration, like gene therapy, every data point is notable, and taken with early but promising data from similar programs, the overall message is “so far, so good.”
Over the past year, UniQure (NASDAQ: QURE) and Bannockburn, Ill-based Baxalta (NYSE: BXLT) have also presented early clinical data on their hemophilia B programs. And BioMarin (NASDAQ: BMRN) last month provided the first clinical data for its hemophilia A gene therapy. (A majority of patients with the chronic blood disease have hemophilia A.)
The differences between all of these companies are very technical, concerning the viral “vectors” they use to deliver their gene therapies into patients’ cells, or with the genetic material itself. Those differences could eventually result in a range of clinical benefits. Perhaps one therapy eventually will prove safer, or longer-lasting, or beneficial to a certain genetic subtype of patients.
Spark will provide more details from the data at a medical meeting next month. Bloomberg has more on the data here. And check out this story for more on gene therapy, hemophilia, and Spark’s scientific founder Katherine High, a key figure in gene therapy’s renaissance. Spark is also developing a gene therapy for hemophilia A that should begin clinical testing next year.
* ?
www.xconomy.com/national/2016/05/19/s...
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