Andre'a Lucca - Director, Communications Tim Miller - President and CEO Jeff Davis - COO.

James Liverman - Wells Fargo Advisors.

Greeting and welcome to the Abeona Therapeutics Second Quarter Conference Call. At this time all participants are in a listen-only mode. A brief question-and-answer session will follow the formal presentation. [Operator Instructions]. As a reminder, this conference is being recorded.

It is now my pleasure to introduce you to our host, Andre'a Lucca, Director of Communications at Abeona Therapeutics.

Andre'a?.

Thanks Adam. Good morning and welcome everyone. On the call today are Dr. Tim Miller, President and CEO; and Jeff Davis COO of Abeona Therapeutics. Tim will begin the call with an overview of the second quarter and more recent developments at Abeona and then Jeff will provide additional comments on the quarter.

A brief overview of recent financings and provide a snapshot of our financial position and review upcoming investor conference calendar. Following that we will open the floor to a few 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 and 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 Web site, www.abeonatherapeutics.com. With that said it is now my pleasure to introduce Dr. Tim Miller. Tim you have the floor..

Thank you Andre'a. And welcome all the investors, analysts and other stakeholders that maybe all including any of our supporting foundations apparent to the call today. The second quarter of 2015 was one of significant growth and transformation for Abeona Therapeutics from a number of perspectives.

First and foremost, Abeona announced and closed the acquisition of the private company Abeona Therapeutics LLC, which was one of three partnering therapeutic companies that initially spun out of Nationwide Children's Hospital in Columbus Ohio around 2013.

With that acquisition Abeona acquired two leading systemic AAV gene therapeutic programs for the treatment of patient with Sanfilippo syndromes type A and type B.

We believe as do our generous shareholders, Sanfilippo foundation that have been critically important for the development on these programs, that we have two best in class programs for this devastating genetic disease.

And that our unique approach offers the highest probability of success providing these children with a viable treatment option to treat both the central nervous system deficits and peripheral manifestations of this deadly disease.

We have initiated the manufacture of the clinical and the trial material in the past quarter, for the clinical trials in both Sanfilippo type A and type B to start in the United States, and expect it to be in a position to dose our patients in both trials towards the end of the fourth quarter of this year.

The Abeona transaction closed in mid-May of this year. On June 08 of this year we announced that Abeona has signed a license for exclusive worldwide rights to strategically complimentary AAV gene therapy program for the treatment of Juvenile Batten disease another lysosomal storage disease. This program comes from the laboratory of Dr.

Tammy Kielian of University of Nebraska Medical Center. Juvenile Batten disease is like Sanfilippo syndromes, a devastating lysosomal storage disease with no FDA approved treatment options.

We are optimistic that we can take advantage of some of the synergies we have observed and both the safety and efficacies in our AAV gene therapies for treating lysosomal storage disease and move the Juvenile Batten disease program into human clinical trials by this time next year.

We very much look forward to keeping you upraised of the developments in this program. Also as another milestone on June 15, Abeona licensed exclusive worldwide rights to take CRISPR-Cas9 Gene Editing program that also uses the AAV gene therapy technology from the laboratory of Dr. Jakub Tolar, at the University of Minnesota.

As a part of this very important license agreement, Abeona received the rights to Dr. Tolar’s unique application of the CRISPR/Cas9 Gene Editing Technology and a broad range of rare non-oncology blood disorders.

This groundbreaking platform will enable us to develop new CRISPR-based therapies in a verity of blood disorders including diseases like hemophilia, sickle cell anemia, beta thalassemia as well as many other inherited forms of neutropenia, thrombocytopenia and other anemias. Initially, our lead product using CRISPR/Cas9 is Fanconi anemia Dr.

Tolar and his team have demonstrated very promising efficacy and repairing the FANCC gene defect and human fibroblasts from [technical difficulty] patient. Importantly with no off target effects and we look forward to working with Dr.

Tolar and his team the FDA and the dedicated community of 10 anemia patients in their families to accelerate this program into human clinical trials.

Additionally on June 19, we announced that we have changed our corporate name to Abeona Therapeutics Incorporated to better reflect our broader commitment to delivering gene therapy and plasma-based products for the treatment of severe and life-threatening rare diseases.

As part of this process, Abeona began trading on NASDAQ at Monday June 22 under the ticker symbol ABEO and Abeona’s extended leadership and management team have had the opportunity to bring that NASDAQ closing bell that day.

Now I’d like to turn it over to Jeff Davis, our COO for a brief update on the SDF plasma program and review of the quarter’s financial..

Thank you, Tim. And I would also like to thank all investors and analysts and stakeholders of Abeona for participating in the call today.

As Tim mentioned, we’re focused on rare and orphanage diseases in the first one that we in-licensed while September was a program inherited COPD which was a new technology to extract and purify proteins from human blood plasma.

And our first program is in alpha-1 antitrypsin, which is an old name it’s often called and alpha-1 protease inhibitor today.

We en-licensed the technology in September since February of this year we have been optimizing and finalizing the downstream chromatography steps of that program of that technology with the aim of scaling it up and being in clinical trials in 2016.

Our process relative to the Cohn process, the Cohn cold fractionation process is a process by which substantially all of the human plasma proteins are created today and that’s a process that was developed in 1940s with a little tweaks but no sort of revolution in the process of moving those proteins.

As it pertains to alpha-1, the Cohn process is not a very efficient process and the yields are very-very small. Our process which we called salt diafiltration which is just a two step salt precipitation followed by chromatography is optimized around both alpha-1 antitrypsin and IVIG or Intravenous immunoglobulins.

Those are the two high value proteins we believe in the plasma proteins space. We’re having success in scaling that up, we’re on plan.

We are seeing the vastly improved yields in alpha-1 as well as significant improvements in the yield of IVIG and our plan is to go have a conversation with the FDA about our process as well as our scale up the running of validation batches to make our clinical material and to get into what we hope and believe to be a single phase I/typical trial and alpha-1 sometimes next year.

So it is going as planned and we’re excited about the results that we’re seeing so far. Transitioning to talk a little bit about the financial perspectives and accomplishments, in the second quarter, we’ve gone a very long way to strengthening our balance sheet. At the end of the first quarter, Abeona had approximately $8 million on the balance sheet.

During the second quarter, we executed two direct placements totaling $17 million with institutional investors, healthcare institutional investors. We received approximately $4.6 million in cash due to the exercise of outstanding publicly traded warrants. We have warrants that trade on NASDAQ at ABEOW.

And we consolidated in just under $5 million in cash from the acquisition of Abeona Therapeutics LLC. So as of June 30, 2015 as reflected in our recently filed 10-Q, we had roughly $30.5 million in cash and prepaid expenses.

After the end of the quarter, we executed an additional $15.5 million financing in a direct financing with the current institutional investors. And given the information in those public filings, investors can approximate that our current cash balance is roughly $45 million.

I’d like to remind the investor community that Abeona has roughly 1.4 million in grants for the development of clinical material for the upcoming Sanfilippo A and B trials and that will obviously defer a cash burn on the development of drug for those two trials. So that's really the highlight in the financial.

I’ll turn it back to Tim to comment on our upcoming conference schedule and then we can take some Q&A..

Thanks Jeff. It’s been an exciting quarter and it is reflected, I think, in lot of the interest in Abeona. We’re going to be presenting at a number of Investor and Rare Disease conferences in the near future.

During the week August 31, we’re presenting at the Sidoti Microcap Equity conference in New York City, it’s actually September 2, at 11.20 AM at the [indiscernible] New York. We’re also presenting on September 10, the Rodman & Renshaw Conference in New York City.

We’ll be presenting at the National MPS Society Family conference in Salt Lake City over September 18th and September 19th. September 24 and 26 is The Global Genes Patient Advocacy Summit and tribute to The Champions of Hope at the Hyatt Regency Resort and Huntington Beach, California.

Abeona had previously received the 2013 Champion of Hope award for Patient Advocacy. We were there to support the community there as well. We are also helping the sponsor, just a table there.

On October 07 to 09 we’ll be presenting at the Alliance for Regenerative Medicine The Stem Cell on the Mesa Meeting, where the company has been selected to join other leaders in the gene and cell therapy space to represent.

In October we’re also be presenting and moderating sessions at SMI Groups Annual Conference on Orphan Drugs and Rare Diseases in London on October 19 and 20. Thank you everyone. I’ll now turn it over to the operator for any questions..

Thank you ladies and gentlemen. We will now be conducting a question and answer session. [Operator Instructions] Our first question comes from the line of James Liverman with Wells Fargo Advisors. Please go ahead with your question..

It’s very exciting to see all the changes that are occurring with your company.

When might we begin to see some of the scientific data, what the first really significant indications be these continuous scaling up of the salt diafiltration technology?.

With respect to the new programs that we all discussed today, Hi Jim by the way, the current plan on Sanfilippo A and B is to dose the first patients in those trials later in the fourth quarter of this year. Those will be open label trials.

So if you look at some of the rare disease open label trials that are going on in gene therapy space, the companies tend to speak about those, how those patients are doing in those trials on an ongoing basis.

So it is likely that we will be talking about clinical data, human clinical data in Sanfilippo A and B prior to human clinical data in the alpha-1 antitrypsin trial..

Okay. That makes sense.

And can you give us some more color on your CRISPR technology?.

Sure. It was licensed out of Dr. Jacob Toller's Lab. There's certainly been and I believe that we have published that in some social media, roughly few days ago, a very nice presentation from Mark Osborne [ph] out of the University of Minnesota describing a lot of the work that's been done with CRISPR-cas9 or their programs out there in fact anemia.

So certainly a very exciting program and we are looking forward to bringing that in the clinical trials we believe towards the end of 2016..

And very sort of naïve about these things, but is there some broad based applications down the road potentially?.

I think that's very true, it's been herald that it's one of the greatest finds in biotechnology in the past 50 years so I mean a very exciting technology that many groups are looking to utilize..

Thank you. [Operator Instructions]. Ladies and gentlemen we have no further questions in queue at this time. I would like to turn the floor back over to management for closing remarks..

Well, thank you. This is Tim Miller. I would like to thank everyone for their attention and participation. We certainly look forward to keeping our investors, analysts and stakeholders abreast of developments, particularly as we are very near towards the clinical trials. We love to see you at upcoming investor and corporate partnering conferences.

Please reach out through Andrea Lucca, our Director of Communications, sitting at the meeting. And to replay information a recorded replay of this call will be available at the following toll free number 877-660-6853 or an international toll number 201-612-7415 and by selecting the conference ID 13617494. Thank you very much, everyone..

Thank you, ladies and gentlemen. This does conclude our teleconference for today. You may now disconnect your lines at this time. Thank you for your participation. And have a wonderful day..