Abeona Therapeutics Inc. (ABEO) Q4 2016 Earnings Report, Transcript and Summary

Good morning and welcome to the Abeona Therapeutics Inc.’s Quarterly Earnings and Business Update Conference Call. [Operator Instructions] As a reminder, this conference is being recorded. It is now my pleasure to introduce your host Christine Silverstein, Vice President of Investor Relations at Abeona Therapeutics. Christine?

Thank you. Good morning and welcome everyone. On the call today we have Dr. Timothy Miller, President and CEO; and Jeffery Davis, COO of Abeona Therapeutics. Dr. Miller will begin the call with an overview of the fourth quarter and more recent highlights and developments at Abeona. After, Jeff will provide additional comments on the quarter, a brief overview of summary financials and provide a snap shot of our financial position and review the upcoming investor conference calendar. Following that we will open the floor up to questions. Before I turn the call over to them, I need to remind our listeners that remarks made during this call may 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 differ 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 website at www.abeonatherapeutics.com. With that said, it is now my pleasure to introduce Dr. Timothy Miller. Tim, you have the floor.

Thanks Christine and thanks for everyone for joining us this morning. 2016 and the fourth quarter especially were marked by notable achievements and advancements for Abeona and our goals for expanding and building ourselves as a rare disease leading gene therapy company. We’ve expanded our program pipeline with the addition of a clinical stage asset. We reported on positive clinical data in our lead program for Sanfilippo syndrome type A, as well as our programs for Epidermolysis Bullosa, and we’ve achieved multiple regulatory designations in multiple programs. Furthermore, we have stated that we are further developing our work in the library of next generation AAV viral vectors, our AIM viral vector platforms, which we have global exclusive rates with the rights to sublicense. To go over some of the milestones that we achieved, particularly in the clinical domain, we will talk a little bit about our lead program ABO 102, which is a treatment, gene therapy treatment for the patients with Sanfilippo syndrome type A. We have met many of our goals for advancing this lead gene therapy program, it is the only one in the clinic in 2016 and currently in 2017 that is enrolling patients for patients with Sanfilippo syndrome. For those that don't know, this is a lysosomal storage disease, these children have problems with an enzyme in breaking down sugars. 70% of these children don't reach the age 18, and it is rather notable to note that recently there have been a lot of popularized deaths from children with this disease. Right now, Abeona is the only program in the world that has a gene therapy muscles and the other therapy in development and in clinical trials. Some of the regulatory achievements in this program were that the EMA or the European Medicinal Agency has granted Orphan Drug Designation, and we also received Fast Track designation from the FDA from this program, which enables us to file [indiscernible] biologics licensing application. We are already building on the granted rare disease Pediatric Designation and the Orphan Drug Designation in the United States. The significance and of these designations is not to be really undermined, it is certainly as we look to the future going to be helpful for us looking to file potentially in late 2018. We do have a dosing and complete reporting of the low dose cohort, which will be -- some of which will be reported tomorrow at the New York Academy of Sciences and otherwise reported at the American Society for Gene & Cell Therapy Conference coming up in May. It is important that we do note that the priority review voucher, we are eligible for this program and look forward to updating more as we go forward with our regulatory strategies. We have recently reported positive data from the low dose, demonstrating central nervous system and peripheral organ disease bio-potency. As a review of the data that was reported out, we did report out two patients worth of data, although we do have much of the third patients dose data to report up soon. ABO 102 is well tolerated in all subjects to date with three low dose and two high dose patients tested. We are through over 750 days of follow-up with no serious adverse events, which is rather remarkable for gene therapy program as delivered by intravenous delivery. And we have seen a significant reduction in the heparan sulfate, which is the actual GAG or glycosaminoglycan in the cerebrospinal fluid, over 60% in - over three patients. Continued evidence of bio-potency has been demonstrated with reduced liver and spleen volumes and decreased urinary GAGs, as well as total GAGs. To the subject that says that the six-month time point showed evidence for stabilization or improvement, an average of 60% over two subjects in multiples - the Mullen subdomains, and the adaptive behavior ratings on the Vineland stabilized. Importantly, subject showed improved ability to complete individual items in the Leiter-R non-verbal IQ assessment resulting in improved raw scores. This is particularly important as these first three subjects had all been in rewarding the Natural History Study and so we had a long term longitudinal data on them able to demonstrate that severe disease progression over the ages of 5 to 6, and then as they came in for their baseline visit, they actually had gone through the floor of their ability to be able to scored. Why this is important is because six months post injection, these children have improved to a point where they could be scored again on their non-verbal IQ. Looking at, as we go into the high dose cohort, where we are going to be completing the enrolment, the planned enrolment of three patient at Nationwide Children's Hospital in April, and we will most likely be expanding the enrolment either at this dose or at a higher dose at some point in the near future. As important to note that we have – are expanding our enrolments in Spain and Australia, the sites are both being initiated in April, so we will be set to screen starting late April or beginning of May, so as we go into the end of the second quarter, we will be enrolling 2 to 3 patients a month. And we as we start to set some of our enrolment timelines, this is important because we will be moving towards an enrolment goal of 15 patients in the next few months, I’m sorry throughout the end of 2017. This is extremely valuable to the company as we seek to become a leader in Sanfilippo gene therapy, but in gene therapy overall. Our second lead gene therapy program is in the form of autologous keratinocyte corrective gene therapy for a horrifying disease called epidermolysis bullosa, otherwise known as butterfly skin syndrome and we are treating the most severe form of this, the recessive dystrophic version. In this program, children come in or adolescents come in or adults come in and they have a biopsy taken, the keratinocytes, which are actual skin cells are taken out and purified and expanded. Using a gene therapy, they are corrected, and the kids have an underlying deficit of a protein called collagen. Collagen is really the zipper that holds your skin onto your body, and because these patients don't have that they have these blistering diseases that open and form chronic wounds that don't heal for years. We are now partnered with the Stanford University, a very large natural history study showing that none of the therapy that have already been tried to date have worked to help close these wounds, including improved dermatologic programs, the products. Right now, what we do is, we -- while we go up and make these sheets -- they are off the size of an iPhone 7 and each subject gets six of them, placed on top of their wounds. To date, we have - again we have continued to expand our programs with this particular clinical stage asset. We have received EMA Orphan Drug Designation and reported positive Phase 1 clinical results for ABO 101, which are published last year in the Journal of American Medical Association or JAMA, which highlighted that the therapy was well-tolerated in these patients and demonstrated clinical efficacy in 67% of the healed wound in six months, some of which have continued to last 12 months, and importantly has seen biomarker expression of collagen in these wounds. This is significant as we look to have regulatory meetings in the second and third quarter to talk about registrable endpoints for this program, particularly looking at what will be the amount of wound closure and biomarker expression in a one-time point. So accordingly we have treated the sixth patient and the seventh patient has recently been treated as well. So this is a total of 42 wounds that have been treated, and the next steps the company anticipates providing a clinical update at the upcoming annual meeting in Society for Investigative Dermatology in April 2017. Additional regulatory is we have US Orphan Drug Designation and the Rare Pediatric Disease Designation hoping to come in later this year, as well as additional enrolment targets to try and hit 10 to 12 patients by the end of the year. Next, we have, to talk about is our second Sanfilippo program. So recall that for those that are not very familiar with Sanfilippo syndrome or four types, Type A, B, C, and D. We are focusing on the two most common types, Type A, which is our ABO 102 program and then we have any ABO 101 program, which is for Sanfilippo syndrome type B. This is again another adeno-associated virus gene therapy program. We have received the IND to go ahead and start the clinical trial. And it is on track and we are anticipating that we will help patients enrolled in that very soon. The IND has been granted and we will be seeking initial regulatory approval in Europe. Currently, we have two of the three necessary steps completed in Europe to start the clinical trial there as well. We have been granted Orphan Drug Designation in Europe and continue to look forward to announcing additional regulatory designations in this program later this year, as well as reporting our later in the second half of 2018. Moving on to our Proprietary AIM Vector, so we haven't given a lot of guidance yet on how well we are approaching this AIM Vector program, but it does encompass the Vectors that have tissue tropisms from multiple targets, including the hematologic and dermatology phases. We have partnered with University of North Carolina in developing the AIM Vector system. That is a novel AAV-based Vector technology that may also target additional tropisms such as muscle tissue, the liver and or syndrome nervous system. So, right now we are looking forward to reporting out some data on the platform with the American Society for Gene Therapy in collaboration with our partners at the University of North Carolina. It is a very exciting program. We are looking forward to how - this is really how we see approach to second generation products for our lead programs, as well as additional de novel or new programs to be brought in-house at Abeona. We do have two additional programs that I will comment on today. We have our Batten disease programs. So Batten disease is another lysosomal storage disease, so when you look at the top of our pipeline at metabolic and you will see four AAV programs that are all targeting lysosomal storage disease, so the Sanfilippo programs are currently clinical stage and the Batten disease programs at preclinical, but we anticipate that these will be clinical stage by the end of the year. For our juvenile Batten program, which is ABO 201 or the CLN3 version of the disease, the EMA has granted Orphan Drug Designation. We announced preclinical data supporting clinical trials for ABO 201 were published in September, the Journal of Neuroscience. We have completed a lot of the preclinical studies necessary to get into the NIDA and conducted a pre-IND meeting last year, which was very successful. The IND enabling studies for this have been initiated and we are engaging in the FDA an additional comments. Things are looking very, very good in the animals to date, there is no doubt and things look, unlike they are on track for this program to be - again the IND by the end of the year. For ABO 202 the sealant one version of the disease are infantile. We are continuing to advance this towards IND enabling study and we again anticipate that human clinical trials will be commenced by the end of the year. So it has been a very exciting year and really beginning of the year in 2017 for Abeona. Our gene therapy programs, you have three of them that are clinical stage. There are very few companies in the world that have actual clinical data in gene therapy much less a broad enough pipeline. We are setting ourselves up well with the inductor platform to enhance our programs and essentially you for any additional programs for use of the AIM Vector platform, and look forward to announcing that data at the upcoming conferences later on in the year. So with that, I’ll turn this over to Jeff Davis to give our fourth quarter and year-end summary financial results, and look forward to answering any questions coming up at the end of the call. Jeff?

Thank you, Tim. In terms of the financials, I’ll talk a little bit about some of the specifics. Our cash position at the end of the year, cash and cash equivalents at December 31, 2016 was $69.1 million, compared to $31.2 million as of the end of September 30, 2016. The net cash used in operating activities for the 12 months of 2016 was $13 million, as compared to $10.4 million in the same period in 2015, so just a modest increase there. The increase in net cash, the strengthening of our balance sheet occurred in the fourth quarter, we closed underwritten public offering of 6 million shares of common stock at an offering price of $7 per share that closed on November 1, 2016 and then there was a modest partial exercise of the show later in that month that occurred. In terms of revenues, revenues were $256,000 in the fourth quarter compared to $215,000 in the fourth quarter of 2015, and the 12-month revenues were approximately $900,000, compared to approximately $1 million in 2015. Again, these revenues consisted, mainly a combination of royalties from a marketed product MuGard, which is licensed out in several countries, as well as deferred revenue related to upfront payments from early licensing agreements. The loss per share was $0.19 in the fourth quarter, compared to $0.06 in the comparable quarter in 2015 and that’s a fairly concise summary of what went on. If anyone has any questions with respect to the financials in the Form 10-K that was recently filed, feel free to give me a call directly to talk about it. Before summary, I will just talk a little bit about some of the upcoming events and conferences. These by the way, as well as links to our financial statements and our investor presentation can all be found on our website at abeonatherapeutics.com. Tomorrow, as Tim alluded to in his prepared talks, we are - we have work to help organize, together with the New York Academy of Sciences and Dr. George Zavoico who covers us on the sell side research side from JonesTrading helped organize a really good gene therapy for rare disease that is happening here in Manhattan tomorrow. We have a bunch of key opinion leaders in the gene therapy space, including Dr Kevin Flanigan, the principal investigator on our Sanfilippo trial. Dr. Katherine High from Spark Therapeutics, Dr. Brian Kaspar Nationwide Children’s Hospital, Dr. Barry Byrne from Florida, Dr. Maria Escolar from the Children's Hospital in Pittsburgh, Dr. David Pearce from Sanford Research, Jude Samulski from Bamboo and Pfizer now, as well as Dr. Jakub Tolar from University of Minnesota with whom we are working on some of the crisper activities that we are doing. So we look forward to a very full day and an informative day tomorrow here in New York. We have a couple other meetings that were alluded to in the call. It is the society for investigative dermatology, later. This is believe around April 6 to 28 where we believe we will be providing some updates on our EB programs and we have a ASCCT in may where I think there will be abstracts on many of our technologies and discussions and presentations there, as well as a few sort of smaller ones arm and Boston and a few other things. So, we will be very active in the next couple of months with a bunch of conferences where we hope to be able to provide some updates on our clinical programs and in our preclinical programs as well. So I think I'll turn it back to, well, maybe we can open it up for questions now, and then Tim will have some closing remarks.

Thank you. [Operator Instructions] Thank you. Our first question comes from the line of Elemer Piros with Cantor Fitzgerald. Please proceed with your question.

Yes, good morning gentlemen. What I would like to ask qualitatively Tim is the data that you would disclose tomorrow versus the data that you plan to unveil at ASGCT in May, what will they be, and what the difference between the two might be?

Sure. Nice to hear your voice Elemer as always. So the New York Academy of Sciences meeting tomorrow is really more of about gene therapy in general and many of the leaders in the field are really going to be discussing multiple parts of their individual programs. Dr. Flanigan is going to present I think a few pieces of updates for the ABO 102 trial, I’d keep talking and comment on the CSF/heparin sulfate which has again continued to maintain its reduction at day 180 through the high-dose cohort and then probably comment on some of the urinary GAGs as well, you know we are continuing to process a lot of the data that has come out of the MRIs, for example, through both the natural history study and the clinical trial. One of the things that we are looking forward to providing a very robust update at some point in the near-term future is really, what the MRI data has shown us, as well as how we're linking that prospectively towards the doses that have been involved in the high-dose cohort. So I think that tomorrow you will see some small bit additional bite of the apple from CNS, CNS data, as well as some of the urinary data, I think that Kevin wants to hold a little bit of powder for the American Society of Gene & Cell Therapy Conference, which is certainly going to be a bit more clinically focused and more experts in the field for example on the clinical side.

Okay, okay. Did I hear it correctly that you already dosed the second high-dose patient in the III-A study?

Yes, we will be enrolling or finishing the cohort very, very soon.

Okay. And so do you think some of that data from the high-dose cohorts at least the 30 days would make it to ASGCT?

Yes, that is the current goal. Again, it does take time or we try very hard to maintain a level of clinical equipoise in the data, so that it really enables the patients be identified in the social media or public forum that individual data points can be tied back to them. Again, to both protect them and really just to make sure that we have more than an n of 1 going into these reports. So, and consequently we do have to wait a certain amount of time just to get the data because we try to get at least through day 30, sometimes all the ay up to day 90 before we start reporting things out. So, we are looking forward to at least hopefully reporting out the first two patients worth of data at ASGCT.

Thank you Tim. So the III-B IND, I thought that it was allowed probably last May in the US, but did I hear you correctly that now you are moving to Europe and start a European trial instead of in the US or that is just a two-step process?

No, no, actually, so we will be starting and Nationwide Children’s Hospital in the next few months, but we are also planning to open an additional clinical site in that trial in Europe. So there will be two clinical sites, not just one, so we can double up on our enrollments a little bit earlier than we have on the Sanfilippo type A syndrome program. Thank you for allowing me the opportunity to clarify.

Okay, okay. And so is there a shortage of III-B patients that the study hasn’t started yet or …?

Due to the natural history study in Nationwide Children’s Hospital you know, allow me to just recall, you know we enrolled 10 patients. 10-B patients at Nationwide Children’s Hospital and certainly many of those are crossing over into other programs, but there is no lack of III-B patients to be sure. What is interesting to actually note about it is that there has been an uptick in the number of diagnoses in the past two years, I think because more clinicians are becoming more familiar with these diseases. So, certainly as, I think many of these programs are looking to push enrollments to, again the mantra in this disease state is treat early, treat it with as much as possible and certainly looking to continue fulfilling on that model.

Yes, yes. and one last question on this front Tim, did - you talked about the European process that two out of the city steps that needs to be taken is - has been crossed or checked, what is the third step because I am somewhat unfamiliar with the European regulatory landscape?

More so, the first few steps are going to be very much in parallel with what would be considered IRB and the recombinant DNA advisory committee submissions in the United States, so we have already gone through the GMO, the genetically modified organism and ethical submissions and reviews and basically have those approved. So the last step is really with the main regulatory agency in staying, which is the country where we are doing this. So I am looking forward to, and we have a very good relationship with the regulatory agency and they have been very, very helpful providing both feedback in our earlier stages and now that we are about to submit for that regulatory approval to treat coming out.

Okay, okay and one quick question to Jeff, the differ in last year of $13 million Jeff I don’t presume that that would be reflective of what you expect in 2017, what sort of ramp do you envision from that level?

Yes, that is an excellent question Elemer. We all along said last year that the burn we anticipated the burn to increase given the fact that we will open two additional side for the A trial, we will get the B trial kicked off and we have multiple enrollments in the EBA trial that are currently sort of scheduled. I think we have telling investment community guiding them that we anticipate burning $20 million to $22 million this year. So we burned $13 million last year and that doesn’t include any sort of, necessarily any kind of one timers that are unanticipated or what have you, but given that we have somewhere between two and three years worth of cash so we don’t have really any current plans to raise money.

Okay. Thank you very much for answering my questions.

Thanks Elemer.

Thanks Elemer.

Thank you. Our next question comes from the line of Jason McCarthy with Maxim Group. Please proceed with your question.

Hi guys, if we could just steer back to EB-101, if you can remind me, you said you had seven patients that were treated so far, maybe you can give us an update on how long those patients that had their wounds grafted for, maybe what we might expect at the general meeting at the end of the month and pending that trial accelerating enrollment to two to three patients per month and your meetings with the FDA is there a possibility that this study could serve as a registration study?

Hey Jason, it is a great question and you know we are looking forward to really kind of bringing a bit more of the spot light under the EB program in the next couple of months. So we would clarify that we are not trying to do two to three patients in the month in the EB program that is more of the ABO 102 programs and these are still going to be successive enrollments just because of the drug is essentially made or the product is made and it is really its location and how we’re approaching this in discussions with the FDA. So currently we do have 7 patients that have been enrolled. What the Society for Investigative Dermatology is going to talk about really is through the almost - it is almost 2 populations from, the first couple of years, so for the first four patients are already through two year with the follow-up. So in this particular disease category that’s rather significant when you look at the fact that many of them had already received other dermatology products that try and close their wounds and none of them actually had stayed close for more than just a few weeks, whereas after two years and after being treated with our product, and these wounds are upwards of 50% staying close and you are seeing expression of that collagen two years out in them. So as we look to engage the FDA, we do have some meetings coming up, we will be talking about what we believe our registratable endpoints and yes we do hope that this is our registration study where we could potentially file this in the second half of 2018 as well. So again it is an exciting program, we're certainly is expanding it with the enrolments and you will hear this at least for different Investigative Dermatology.

Great. And just a follow-up to that can you just give us a sense of the diameter of the wound that you are able to close. I know there are other groups that are in this space as well, but to my knowledge that they are able to close a very, very tiny portion of the wound, can you give us a sense of how EB-101 mechanism of action is different and if you can cover much more sizeable area of these wounds?

Yes, great question. So when you meet a lot of these patients what they end up coming in as is, basically they were wrapped in bandages because they have open wounds over greater than 50% of their body sometimes. And our EB program can actually treat much larger areas. So again if you can imagine that essentially a skin - the product is about the size of an iPhone 7 and each patient gets six iPhone 7’s worth of surface area covered over their skin. So, we can cover a areas like entire arms, the entire size, a lot of skin over the back, chest so certainly able to cover a large surface area. So thank you for that.

Great. Thanks for taking the questions Tim.

Thanks Jason.

Thank you. Our next question comes from the line of George Zavoico with JonesTrading. Please proceed with your question.

Hi, thank you and thanks for the update Time and Jeff. A couple of fairly quick questions, on the AIM platform for the tropism issue that you talked about where you are looking to see tropism to various organs and tissues, do you have a certain criteria in mind as to what is sufficient to go forward, in other words what percent of off target effect is acceptable?

Yes sure George. So just to clarify, the AIM Vector library possess a number of AAV’s for direct to gene replacement strategy, and so we're looking at these that under a disease specific mindset, so gene therapy as we know is all about delivery and what to deliver the correct copy of gene, under the gene replacement strategies. So we do have vectors that have a significant dermatology or skin tropism. Some of the vectors have very good tropisms for liver, others for the central nervous system which was more part of the library where they actually derived from. So using these gene replacement strategies isn't so much about the target defects, which is really the 13 programs. I didn't comment very much on our Fanconi anemia program at the University of Minnesota, and Stanford. That is certainly moving along. The researchers at the University of Minnesota, Jakub Tolar will be speaking at the New York Academy of Sciences tomorrow George, has certainly help to advance it, but, so for the AIM Vector platform we're looking at these at right tropism deliver the most appropriate way for the right disease. And very similar to many of the other gene therapies programs that are out there trying to [indiscernible] able to include additional disease targets and with these additional disease categories, so hope that answers your question.

Yes it does. In summary, it sounds like you're not really, there is no real criteria for tropism it is just, it is more important to see a satisfactory outcome for the patients in the clinical benefit, as long as you don't have adverse of target effects, is that, in summary is that pretty [indiscernible].

Yes it is a fair to say it again. You are trying to find the right issue tropism for the right [indiscernible] looks like you use a muscular base AAV program for the central nervous system target. So again we look forward to announcing and really showing more about this program in the upcoming months and really those are going to be the ones that additional disease targets will fall under.

Okay. With regard to the central lipo programs, you mentioned - well the trials are set for low-dose and high-dose and then you mentioned that you might go to a higher dose. What would, you need to consider, what kind of results would make you go through a third dose?

Well the prospectively defined dose program has always included the ability to go to higher doses that we chose. We have a very, very broad window within our safety programs from the INDA enabling hospitality study and in Lysosomal Storage Diseases in general the mantra has always been with the gene therapy approach trying to go with treat younger and treat with more gene therapy as possible. So, the investigators have always considered that if things looked safer than they would try and push the does. Just to be clear we are very comfortable with what we are seeing right now, even in the low dose. Recall that when we first started these programs, we started with the minimum efficacious dose to be able to be treated in older animals that showed complete correction of disease which included survival by neuromuscular benefit and really cognitive corrections. And to date when you look at the low dose data, this has been really, I mean a robust data package. No other program in the world has been able to show any form of combination or reductions of gags in the CSF, reductions of gags systemically, organ manifestation changes, and deliverance and seeing volume changes, as well as early indication from a disease stabilization from a neurocognitive benefit. So when we dose escalated it has always been under the idea that look at things look safe, maybe we will continue to dose escalate from there, but again, we would probably speculate to say that things are already working in the clinical program, let’s see how much far that we can do this really for the benefit of the patients because as we enrolled, what we consider the other side of the bell curve on the developmental stage, and now as we speak to enroll some if you are patients to really be able to see if we can extend what will be considered normal or push them into a more normal range of development. So it is a really exciting time for us actually be able to consider just enrolling more patients with a high dose, but really looking to see if we can even accelerate or enhance this program with additional dosing. So, very, very exciting.

You are also decreasing the age in steps of patients who enrolled. First batch I think was - there was an 18 and over and then you - could you just remind us what you are - how you are going to younger patients and what steps you are going to younger patients?

Well the enrolment criteria hasn't changed. It has always been two years and older and relate the natural history study that has come out of this and looked at 25 patients has really shown that it is all about the disease manifestation and an individual patient that really dictates whether they should be enrolled in the clinical trial. We do have a variety of important enrolment criteria, including AAV’s, in the specificity of anti-priorities for AAV. I think the clinicians, really their goal has been to show some initial signs of safety and potential efficacy and now again with the ability to try and target some of the younger kids with this particular product. It is more about, not just destabilization, but potential disease reversal to the point where the kid can develop a bit more normally so.

Thank you. Ladies and gentlemen we have come to the end of our time for questions. I’ll turn the floor back to Mr. Miller for any final remarks.

Well it has been an exciting year and thank you all for your questions. Certainly, as we have spoken to many of you, and look forward to continuing our dialogue, certainly being able to report out the degree to data as we have an exciting time for a company. Now we have three clinical stage programs that are gene therapy programs and two additional programs going into the clinic. Abeona is really continuing its progress to becoming a world leader in gene therapy for rare disease treatments. I like to thank all our investors and partners, researchers and dedicated patients foundations, including the employees and scientific advisers and board members for their continued support, and look forward to meeting with all of you throughout the year. Thank you.

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