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.