Greetings, and welcome to Abeona Therapeutics Third Quarter 2017 Financial Results and recent business highlights. At this time, all participants are in a listen-only mode, and a brief question-and-answer session will follow the formal presentation [Operator Instructions]. As a reminder, this conference is being recorded. I would now like to turn the conference over to, Christine Silverstein. Thank you. You may begin.

Thank you. Good morning, and welcome, everyone. On the call today we have, Dr. Tim Miller, President and CEO and Jeff Davis, COO of Abeona Therapeutics. Dr. Miller will begin the call with an overview of the third quarter and more recent highlights and developments at Abeona. After, Jeff will provide an additional commentary on the quarter, a brief overview of financial summary and provide a snapshot of our financial position, and review the upcoming investor conference schedule. Following that, we will open the floor to a short Q&A session. Before I turn the call over, I need to remind our listeners that remarks made during the call can contain forward-looking statements that involve risks and uncertainties. Forward-looking statements on this call are made pursuant to the safe harbor provisions of the federal securities laws. Information contained in the forward-looking statements is based on current expectations and is subject to change and actual results may vary materially from forward-looking statements. Some of the factors that could cause actual results to differ are discussed in the reports filed with the SEC. These documents are available on our Web site at www.abeonatherapeutics.com. With that said, it is now my pleasure to introduce Dr. Timothy Miller. Tim, you have the floor.

Good morning, everyone. And thank you for joining us on this morning on our earnings call. The third quarter was marked by notable advancements in our goal of building a leading rare disease company with a focus on gene therapies. Importantly, we initiated enrollements in our global clinical trial sites for ABO-102, for treatment of patients with MPS IIIA, and reported additional data to underscore the durability and clinical benefit on this gene therapy. Our Epidermolysis Bullosa program achieved a very nice milestone with FDA Breakthrough Therapy designation, completed its Phase 1/2 clinical trial and is now continuing to advance as we finalize the clinical protocol before the initiation of the pivotal Phase 3 trial next year. With regard to our MPS IIIB program, we recently initiated screening and look forward to commencing enrollment shortly. In addition, work in optimizing our AIM vector platform demonstrated exciting progress, including demonstration of enhanced tissue tropisms compared to other naturally occurring AAV capsids. So to talk a little bit about some of our corporate achievements, we had a really great R&D Day. We held our inaugural R&D Day on October 11th, in New York City, where Abeona management, together with key opinion leaders and clinical experts, presented an update on our clinical program to and over 80% crowd of institutional investors and equity analysts. We next completed an equity financing on October 19th where we announced the closing of $92 million underwritten public offering and full exercise of the underwriters’ option to purchase additional shares. That same week, we announced a grant of up to $13.85 million from leading Sanfilippo syndrome or MPS III foundations from around the world, further supporting the clinical development of our MPS III and IIIB gene therapy programs. We had announced a groundbreaking of the manufacturing facility here…

Thank you, Tim. Our cash and cash equivalents as of September 30, 2017 were $56.5 million compared to $58.3 million as of June 30, 2017. Cash used in operating activities in the nine months ended September 30th was $17.6 million as compared to $9.6 million in the same period of 2016. There were a couple of items that impacted our cash balance both in the third quarter and subsequent to the quarter. As reflected in the current Form 10-Q, the Company received roughly $5.1 million in the third quarter from the proceeds of exercises of outstanding warrants. Additionally, as mentioned by Tim previously, Abeona closed on a public offering of common stock and subsequent exercise of the over-allotment option in the gross amount of $92 million. We thank our bankers, the underwriters and most importantly syndicate of leading investments funds that participated in the financing. As reflected in the most recent press release, as of October 31, 2017, Abeona’s total cash and cash equivalents was $142.6 million. With respect to revenues, revenues were $219,000 for the third quarter of 2017 compared to $184,000 in the second quarter of 2016. Nine months revenues, the first nine months of this year were $622,000 compared to $633,000 in the comparable period last year. Revenues consisted of a combination of royalties from marketed products, primarily MuGard, and the recognition of deferred revenues related to upfront payments from early licensing agreements. Loss per share was $0.13 in the third quarter of 2017 compared to a loss per share of $0.08 in the comparable period in 2016. The total number of common shares outstanding as of the day of the filing of the Form 10-Q on November 13, 2017, which would include the shares in the recently mentioned public offering, as well as shares issued in the exercise of warrants in the third quarter, is 46.7 million shares. Before turning it over or opening up for Q&A, I just will mention some upcoming events and conferences in the week after Thanksgiving on November 30th, Abeona will participate in the Barclays Gene Editing and Gene Therapy day in New York City. Our presentation slot is 10:40 AM Eastern Time. On December 4th, we will be participating in Mizuho’s Global Healthcare Conference in New York and on the following on December 5th, Abeona will participate in Oppenheimer and Co's Orphan and Rare Disease Day New York City. And I believe the format for that meeting is all one-on-one meetings. And so with that, I think I will open it up or have the monitor open it up to Q&A.

Thank you [Operator Instructions]. Our first question comes from the line of Matthew Eckler, with RBC. Please go ahead with your questions.

When thinking about cadence for enrollment of the MPS IIIA trial going forward. How long until you think you could have around another 15 patients enrolled, 15 total.

So we’re anticipating to get next five or six patients in through the next quarter so with three sites trying to target one and two a month. We’re looking to have that 15 patients mark, it's right around that March, April, May timeframe.

And building on that then, what are your current thoughts around engaging FDA on the ability to file an NDA?

We think about engaging the FDA a lot, and the EMA. We’re currently on track I think to have regulatory meetings with the FDA and the EMA in the first half of next year where I think that we’ll be talking about the data and talking about what looks like registration end points, or to talk about where they think the trials were undergoing. The data is very robust to this point. And as we see the reductions in key biomarkers, such as heparan sulfate, no one else has really been able to do that. And if you can't reduce that heparan sulfate, you’ll then have a therapy. So from our perspective, when we line up the heparan sulfate reductions with the organ changes, changes in the brain structure and then changes with the neurocognitive improvements and stabilization, I think we have a pretty strong case to-date. So we’ll be having those discussions in really in the first half of next year.

And then last question from me, any updates around efforts to secure license for [AAB9] IP?

We’re certainly looking forward to continuing discussions. And we’ll have an update sometime in 2018.

Thank you. Our next question comes from the line of Liav Abraham with Citigroup. Please go ahead with your questions.

Just a question on timelines regarding the data for ABO-102. Can you just update us where you are in the connection of the six months neurocognitive data from the second cohort of the trial? And when is the earliest for these to be released?

Well, so we’re looking at all the data really together right now we have and really with the Cohort 1 follow up, Cohort 2 and Cohort 3, we’ll be presenting those data at conferences in the first half of 2018.

Can you be any more specific on that?

I think that we will probably be looking at that probably at either the World Conference or the American Society for Gene & Cell Therapy.

And then you guys have a lot going on in terms of clinical programs. At this point are you considering [partnering] any of these or is it too early to be talking about that?

Obviously, as really looking at this as a third generation type of development on the AAV, there are many companies that are looking for -- and many groups that are looking for ways to find additional transduction targets, specific for different organ systems. So we’re continuing to advance internally as well as with our academic partners and I think that’s, just like anyone else out there, there is certainly interest in the programs.

Thank you. Our next question comes from the line of Maury Raycroft with Jefferies. Please go ahead with your questions.

To start, can you comment on the age of the patients in Cohort 3? And given the de-risking with the safety data from the initial two cohorts, do you think the next six to nine patients you enroll maybe even younger age?

So the average aging Cohort 1 was about 6.5, the average age in Cohort 2 was about I think 3.5. As we look to Cohort 3, the average age around those patients is also between ages I think of 3 and 4 so about 3.5. We're certainly very interested and have discussed this with the DSMB and the clinical investigators about dropping the enrollment age. So that's certainly something that we are considering at all three clinical sites, and look forward to providing an update with that again and then probably the first or second quarter. But it seems like a logical expansion of where this therapy would go. We're also looking to solidify some relationships and working on new born screening tests. Now as we look out to the three year horizon with an approved product just like this really what you want to try and do would get this with the new born screening test, because again with that Monitoring Gene Therapy it's treated earlier and treated with a significant dose. So we're looking to try and I think push that frontier as well.

And then for EB-101 for the Phase 3. So you mentioned that you’re screening patients. And as far as enrollment goes and the timelines for the process go, is harvesting keratinocytes part of the process at this point? And then what's the timeline for harvesting the keratinocytes manufacturing grafts and then administering the grafts?

So that all happens over about a four week process, four to five weeks. So again, as we look to finalize the protocol with the FDA not just from the clinical protocol but also on -- again as a Phase III trial, you’re working on CMC, you’re working on assay development, you’re working on the commercial facility itself. So we're looking to try to accelerate that as much as possible. And that includes working with the FDA on ways to speed that process up. So when we purify out the keratinocytes from the moment the biopsy occurs to when they get treated is again about four or five weeks.

And so are you at the point where you’re harvesting keratinocytes sites from patients yet?

Well, we certainly do that as part of the Phase 3 prep. We do engineering runs, I think, through that. And so again, we've been in that for a while now. And the great thing about it is again when you see this in the four GMP facility things have been working very-very well we're on track.

And for ABO-101, given that it's not self complimentary like ABO-102. How will this translate to efficiency of expression and ultimately efficacy and can you compensate on some ways by starting the initial Cohort at a higher dose?

So the first -- let me answer the last question first. So yes, we are dosing on the first cohort at a higher dose just under our Cohort 3 dose in the MPS IIIA program. And this is all tracking to our preclinical data of what these doses were about dosing older animals where you saw a complete correction of survival, neuromuscular function and cognitive ability. It's just as a single stranded DNA, just like using the same caps and so we're getting into the same tissues. We also know that we're getting very-very high levels of expression. So one of the nice things is about this particular vector is it produces a bit higher than the self complimentary vectors. So from a manufacturing standpoint, there is a -- it's a bit more of a win there, because we can produce more of it and we produced enough to treat roughly 15 or 18 patients to-data. And again as we look forward to announcing the enrollment of that first patient, are coming up very-very soon.

Our next question comes from the line of Ram Selvaraju with H.C. Wainwright. Please go ahead with your questions.

Firstly, could you comment on what is likely to be the most appropriate outcome measure with respect to measuring neurocognitive function in the [indiscernible] pivotal program and may be specifically discuss the use of the Bayley versus the Leiter and Vineland scales?

I think that the key clinical parameters are going to start with the measurement of heparan sulfate. Again, as we’ve been able to demonstrate, seeing 70% reduction in CNS, as well as in urine going out through one-year even at the lowest dose, this is the key pathological substance that’s being used by many of these -- again in these patients; and so first and foremost, you’ve got to start there; second, looking at multiple organ changes that go on as a result of after treatment; so seeing reductions in the liver and spleen volumes, seeing preservation of deep brain architecture. And recall this is an intravenous administration so you don’t see this, you don’t see those types of benefits in many other ways of the treatment due to the access of the intravenous administration that can go through into the deep brain architecture with the 600 miles of using veins and arteries. And so we have a 25 patient natural history study that was used to support really the clinical development of these programs. The FDA was brought on in conjunction and collaboration in the design of that clinical trial. And so we use the Leiter and the Vineland scales, in particular, where we’ve been able to demonstrate that level -- some of levels of benefit but we’re very comfortable at those. And again, in our interactions with multiple regulatory agencies, they’ve been using those as well. It’s important to note Ram that these scales are actually applicable when looking at six months olds, up through eight, nine, 10 year olds, some of the other scales that are in consideration actually have ceiling of 42 months. So when you got to go back and back calculate through your natural history study where many patients are actually inappropriate to be used by that scale, those tend to make a little bit more of a challenge. So at least our interactions with the regulators so far have demonstrated that we’re on track.

And a couple of other quick things. Do you anticipate being able to utilize similar peripheral biomarkers to what has so far been used and what has demonstrated apparently statistically significant impact in the MPS IIIA program with the ABO-102 versus what you anticipate being able to do with ABO-101 going forward? If you could give us a way to correlate what you might be looking at with respect to peripheral biomarkers of the ABO-101 program based on what you’ve learnt so far with ABO-102? And then secondly, just with respect to the potential for utilizing a gene therapy approach in EB going forward as opposed to the skin grafting approach. Can you give us a sense of what you are thinking about in terms of the administration paradigm for gene therapy specifically in EB? Thank you.

Well, for the ABO-101 program, much of what we are doing in the ABO-102 program will directly transfer over. So we will be looking at some very similar biomarkers. We will be looking at liver and spleen and brain volumetric changes. We will be using a neurocognitive scale. So I think one of the nice things about that program is that we anticipate the results will track very similarly of the MPS IIIA program. For the EB program, the EB-101 is applicable to very large areas. So you can imagine covering a number of rooms of our EB patient body with iPhone sized grafts, that’s an important differentiator in this particular product. That being said, there are some areas that can be challenging for multiple reasons for durability, for accessibility, looking at the -- for example, between the areas between your fingers. So we have the EB-201 program, which is an AAV delivered way of delivering collagen 7A1. This is an AAV vector that has very high tropism for epidermis and dermal tissue and this is about 95% of the cells in those regions. And we already have proof-of-concept where we already have in human skin the demonstration of collagen 7A1 production. And so we see these eventually being a combination approach. It's important to note that even with this type of delivery the AAVs we are seeing long-term expression, in those areas and certainly much longer than some other different types of viruses that tend to be very quickly removed out of the skin. So it's important thing to note on where we are going with those programs, but thanks for asking that.

And then the last thing is based on what you have learned so far with EBITDA-101. Do you expect, especially now that you have these AAV vectors that have skin specific tropism qualities, that there might be the possibility of your strategically moving into other rare dermatological disorders beyond EB per se given especially the ease with which you can monitor efficacy outcomes in those kinds of conditions?

That’s a fun one. So gene therapy is all about delivery. And if you’ve got right vector delivered by the right disease for the right method then you know how you want to try and approach different diseases. And so we’ve demonstrated really the proof of concept in the skin. And so now using this type of AAV delivery certainly opens up hundreds of rare dermatological diseases. Certainly, we’re looking at that opportunistically since again we think we have the vector to be able to do that. So thanks.

Our next question comes from the line of Elemer Piros with Cantor. Please go ahead with your questions.

Tim, is it okay to assume than in Australia once you get that site up and running, you would also start with the high-dose in the IIIA patients?

Yes, so that’s correct. So when we started -- so we enrolled the first patient already in Australia and that patient was at the high dose at the Cohort 3.

I've confused it with Spain, you are correct.

Yes, also in Spain.

So thinking about the younger population 3.5 and maybe even younger, as you I am sure maybe even going down to six months of age. Have you determined whether an improvement or a change and let it be neurocognitive or some other measures, is not due to the early pretty good natural history or normal development of the chart?

Yes, so that’s been a very interesting thing for us discussed internally and with some of our DSMB and KOLs. Basically you’re talking about if you enroll like a one year old, how do you attribute neurological benefits to drug treatment rather than incremental improvements that would have normally happened before the disease really took hold. Is that correct?

That is correct.

So really where a lot of the fallback for demonstration of efficacy in support of the neurological scores really comes down to brain volumetric mapping. So we know from the natural history study where there's been significant volumetric loss, whether it’s a six month old or five-year-old. And so in support of the neurological finding, we will be able to back that up by looking at the brain volumetrics volume a lot.

So you would think that if there is a normal development, but no volumetric damage yet then that drug is probably still in the normal course as opposed to having a drug effect. But on the other hand, if there is an improvement in both neurocog and brain volume that would be much better to ascribe to an effect due to the therapy. Is that -- am I getting it right?

That’s correct. Also, it's important to note Elemer that like the Leiter and the Vineland score, they’re applicable down to that age group. So we’ll be looking at that as well as the Mullen scores in those younger patients. So we think that those are good ways to be able to assess that.

But is it safe to say that for the next six to nine IIIA patients, you would stay in this three to four year old category, or it's….

That's certainly -- I think it's probably more important to say that we're seeing with patients that have scores in the developmental IQ range or developmental score range, and in really a certain window, we want them to be above the floors. So that again, you can have a quantitative measurement of assessment.

And our next question comes from the line of Matthew Cross with Jones Trading. Please go ahead with your questions.

So at you R&D Day in October, you went into some detail on the use of both the IV and intrathecal route of administration in patent disease programs pre-clinically. And I was wondering if you could remind us what about this indication or the vector may require this approach to get above the maximum feasible dose for a single route of administration compared to your other programs utilizing an AAV vector?

So just to clarify, the CLN 3 program as a systemic administration but the CLN 1 program is the combination dosing. And I think that as we look to the future of gene therapy, what you’re going to end up seeing from more programs than just is more groups will be using combination dosing going in with what you got one of our third generation AIM vectors or you’re using one from a one of the older by generations that you may have a little less of a tropism. The idea is that you want to hit as many systems that are affected by the disease it's hard as possible with the gene therapy. The general mantra here is that over expression or increasing the expression as a replacement strategy has really been the way to go with the CPs level that we've seen particularly with our AAV vectors and again now working into the AIM vectors that's really where I think the field has headed. So we’re excited in the infantile Batten program for CLN 1 to try and champion that. And the reason why we are going that route is because again this is infantile, this presents if you catch it early enough, it presents very, very fast with very, very high mortality usually before the kids hit 10 and then very, very severe disease progression. So pre-clinically, what the preclinical research even in gray has shown is that there is also a significant amount of spinal damage in this particular disease. So while again all part of the CNS and it was really then his model to try and approach this from a combination route to say we want to go intravenous, we want to go intrathecal, let's see how far we can really demonstrate the efficacy. And really in the end and you may actually be able to reduce some of your manufacturing burden by going both routes. So certainly we're looking at patients first and what's going to be the best way to target their disease manifestations and really how to alleviate those.

Thank you. This concludes our question-and-answer session. I'd like to turn the floor back to management for any closing comments.

Thanks everyone for joining us today and we look forward to meeting with many of you in January and going on into the annual conference that’s coming up. So thanks again for everyone. Christine if you have any closing comments.

That wraps it up Tim. Thank you everyone for joining the call.

Thank you. This concludes today's conference. You may disconnect your lines at this time. And thank you for your participation.