Wired News – European Medicines Agency Granted Orphan Drug Designation for Abeona’s ABO-202 Gene Therapy Program in Batten Disease

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LONDON, UK / ACCESSWIRE / April 24, 2018 / Active-Investors.com has just released a free research report on Abeona Therapeutics Inc. (NASDAQ: ABEO). If you want access to this report all you need to do is sign up now by clicking the following link www.active-investors.com/registration-sg/?symbol=ABEO as the Company’s latest news hit the wire. On April 20, 2018, the Company announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products has granted Orphan Drug Designation for Abeona`s gene therapy program ABO-202 for the treatment of subjects with neuronal ceroid lipofuscinosis, also known as Batten Disease, a rare, fatal, autosomal recessive (inherited) disorder of the nervous system. Register today and get access to over 1,000 Free Research Reports by joining our site below:

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The orphan drug designation provides Abeona with incentives and benefits in the European Union, including protocol assistance, reduced fees, and protection from market competition once ABO-202 is approved for patients with CLN1 disease.

Compelling Pre-Clinical Data Underscore Viability of ABO-202 as a Potential Treatment for CLN1 Disease

Carsten Thiel, Ph. D, Chief Executive Officer of Abeona, stated that this designation is an important step in the Company’s CLN1 program as it encourages to proceed towards clinical development.

Carsten added that compelling pre-clinical data underscore the viability of ABO-202 as a potential treatment for CLN1 disease in humans.

FDA Granted Orphan Drug Designation for ABO-202 Gene Therapy Program in Batten Disease

On February 12, 2018, the ABO-202 program was granted Orphan Drug Designation (ODD) by the US Food and Drug Administration (FDA) for the treatment of infantile Batten disease. On March 15, 2018, the FDA granted Rare Pediatric Disease Designation for the ABO-202 program for the treatment of CLN1 disease (infantile and late infantile onset Batten disease).

Abeona Reported Top-Line Data at the WORLDSymposium

Preclinical data from the program were recently presented at the WORLDSymposium held in San Diego. Key findings include that the data demonstrate that a single intravenous, single intrathecal (IT) or combination therapy utilizing both administration routes of a self-complementary adeno-associated virus encoding the human CLN1 gene to CLN1 mice significantly increased their survival, improved behavior and reduced motor deficits. It was also reported that higher doses further improved these observations, suggesting that methods increasing total CNS exposure may be beneficial and provided some survival and behavioral benefit to symptomatic mice. A combination approach delivering ABO-202 by both intravenous and intrathecal routes of administration further increased survival efficacy and improved potential treatment options for older animals with advanced disease manifestations.

About CLN1 Disease

Infantile and late infantile neuronal ceroid lipofuscinosis is a severe lysosomal disease caused by mutations in the CLN1 gene, which encodes the soluble lysosomal enzyme Palmitoyl-Protein-Thioesterase-1 (PPT1) and result in osmiophilic granules accumulating in lysosomes and leading to neuroinflammation, neurodegeneration, and death. CLN1 disease is neurodegenerative, manifests shortly after birth, and is fatal in its classic form by 6 to 12 years of age. In the classic form, aggressive clinical features appear, including rapid speech and motor deterioration, refractory epilepsy, ataxia, myoclonus, and visual failure between the ages of 6 and 24 months. By 5 years of age, CLN1 disease patients with the classic infantile form are typically poorly responsive and are no longer communicative. These patients subsequently die in the following few years.

About ABO-202

ABO-202 is an adeno-associated virus developed to introduce a functional copy of the CLN1 gene into cells in order to restore the enzyme activity that is needed to break down certain lipopigment proteins that are deficient in patients with CLN1 disease.

About Abeona Therapeutics Inc.

Founded in 1989 and headquartered in Dallas, Texas, Abeona Therapeutics is a leading clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare genetic diseases.

Stock Performance Snapshot

April 23, 2018 – At Monday’s closing bell, Abeona Therapeutics’ stock dropped 4.31%, ending the trading session at $20.00.

Volume traded for the day: 1.60 million shares, which was above the 3-month average volume of 1.23 million shares.

Stock performance in the last month – up 35.14%; previous three-month period – up 34.68%; past twelve-month period – up 284.62%; and year-to-date – up 26.18%

After yesterday’s close, Abeona Therapeutics’ market cap was at $886.40 million.

The stock is part of the Healthcare sector, categorized under the Biotechnology industry. This sector was up 0.1% at the end of the session.

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