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Using Venezia™, physicians at Ludwig-Maximilians University of Munich (LMU Munich) successfully performed the interstitial brachytherapy procedure. Venezia allows clinicians to treat advanced stages of cervical cancer, such as stages IIIA and IIIB with vaginal and parametrial extensions, which previously required very specialized techniques, such as freehand needle placement. "Getting the radiation to reach to disease extensions beyond the cervix has always been challenging with conventional brachytherapy applicators," says Stefanie Corradini, MD, radiation oncologist at LMU Munich. "Venezia incorporates components that enable us to treat the cervix as well as the parametrium and vagina using a single applicator. We were able to treat this first patient's disease extension in these anatomies by placing both parallel and oblique interstitial needles. With Venezia, we will have a very standardized way to treat cervical cancer and interstitial needle placement will be very reproducible." "The design of Venezia makes it intuitive to use," adds LMU Munich's Cornelius Maihöfer, MD. "Within just a few trial runs of applicator assembly, the team became proficient in handling Venezia." The two lunar-shaped ovoids of Venezia form a ring that when clicked together provide the ease of insertion of a tandem and ovoid applicator in combination with the dose distribution provided by a ring applicator. The ring creates a pear-shaped dose distribution, a proven dosimetry shape that matches the cervix and endometrium. To reach tumor extension beyond the cervix, the design also incorporates a perineal template for placement of parallel needles and holes in the ring for insertion of oblique needles. "You could view Venezia as a hybrid brachytherapy applicator that combines the best characteristics of both ring and ovoid devices as well as an array of interstitial catheters that can be customized to each patient´s tumor anatomy," Dr. Corradini says. For further information, please contact: This information was brought to you by Cision http://news.cision.com The following files are available for download: To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/patient-in-germany-is-worlds-first-to-receive-brachytherapy-with-elektas-new-venezia-gynecological-applicator-300446091.html


Using Venezia™, physicians at Ludwig-Maximilians University of Munich (LMU Munich) successfully performed the interstitial brachytherapy procedure. Venezia allows clinicians to treat advanced stages of cervical cancer, such as stages IIIA and IIIB with vaginal and parametrial extensions, which previously required very specialized techniques, such as freehand needle placement. "Getting the radiation to reach to disease extensions beyond the cervix has always been challenging with conventional brachytherapy applicators," says Stefanie Corradini, MD, radiation oncologist at LMU Munich. "Venezia incorporates components that enable us to treat the cervix as well as the parametrium and vagina using a single applicator. We were able to treat this first patient's disease extension in these anatomies by placing both parallel and oblique interstitial needles. With Venezia, we will have a very standardized way to treat cervical cancer and interstitial needle placement will be very reproducible." "The design of Venezia makes it intuitive to use," adds LMU Munich's Cornelius Maihöfer, MD. "Within just a few trial runs of applicator assembly, the team became proficient in handling Venezia." The two lunar-shaped ovoids of Venezia form a ring that when clicked together provide the ease of insertion of a tandem and ovoid applicator in combination with the dose distribution provided by a ring applicator. The ring creates a pear-shaped dose distribution, a proven dosimetry shape that matches the cervix and endometrium. To reach tumor extension beyond the cervix, the design also incorporates a perineal template for placement of parallel needles and holes in the ring for insertion of oblique needles. "You could view Venezia as a hybrid brachytherapy applicator that combines the best characteristics of both ring and ovoid devices as well as an array of interstitial catheters that can be customized to each patient´s tumor anatomy," Dr. Corradini says. For further information, please contact: This information was brought to you by Cision http://news.cision.com The following files are available for download:


NEW YORK and CLEVELAND, May 08, 2017 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare diseases, announced today that data from the Company's gene therapy clinical trial and preclinical research programs will be highlighted at the 20th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) being held in Washington, D.C. from May 10-13, 2017. Abeona’s collaborators will present two oral and one poster presentation during the conference. These presentations will detail data from clinical and research programs utilizing gene therapy as a potential treatment for patients with the lysosomal storage disorders Sanfilippo syndrome and Batten Disease, and the Company’s next generation AAV AIM™ Vector platform.  Details of the presentations and poster are listed below. Oral Presentations: Session 243 - Clinical Trials for Neurologic and Neurosensory Disorders:  292. A Phase 1/2 Clinical Trial of Systemic Gene Transfer of scAAV9.U1a.hSGSH for MPS IIIA: Safety, Tolerability, and Preliminary Evidence of Biopotency Presenter: Kevin M. Flanigan, M.D., Principal Investigator and Director, Research Institute of Nationwide Children’s Hospital, Columbus, OH Date/Time: Thursday, May 11th; 4:00pm–4:15pm EDT Location: Marriott Salon 1 Session 344 - Preclinical Progress Towards Therapies for Neurologic Disorders: 537. Intrathecal scAAV9 Gene Therapy Is an Effective Treatment for PPT1-Deficiency in the Preclinical INCL Mouse Model Presenter: Alejandra J. Rozenberg, Ph.D. and Erik A. Lykken, Ph.D., -  University of North Carolina at Chapel Hill, Chapel Hill, NC Date/Time: Friday, May 12th; 4:45pm–5:00pm EDT Location: Delaware AB Poster Session: Session AAV Vectors II: 316. Enhanced CNS Specificity of Adeno-Associated Viral Vectors Created by DNA Shuffling and Directed Evolution Presenter: Daphne Chen, University of North Carolina at Chapel Hill, Chapel Hill, NC Date/Time: Thursday, May 11th; 5:15pm–7:15pm EDT Location: Exhibit Hall A & B South Conference Call Details: Abeona will host a live conference call briefing on Friday, May 12th at 10:00am EDT. Analysts and investors can participate in the conference call by dialing 877-269-7756 for domestic callers and 201-689-7817 for international callers. About Abeona: Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene therapies for life-threatening rare genetic diseases. Abeona's lead programs include ABO-102 (AAV-SGSH), an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome type A (MPS IIIA) and EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB). Abeona is also developing ABO-101 (AAV-NAGLU) for Sanfilippo syndrome type B (MPS IIIB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease (JNCL), ABO-202 (AAV-CLN1) for treatment of infantile Batten disease (INCL), EB-201 for epidermolysis bullosa (EB), ABO-301 (AAV-FANCC) for Fanconi anemia (FA) disorder and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy for rare blood diseases. In addition, Abeona has a plasma-based protein therapy pipeline, including SDF Alpha™ (alpha-1 protease inhibitor) for inherited COPD, using its proprietary SDF™ (Salt Diafiltration) ethanol-free process. For more information, visit www.abeonatherapeutics.com. This press release contains certain statements that are forward-looking within the meaning of Section 27a of the Securities Act of 1933, as amended, and that involve risks and uncertainties. These statements are subject to numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the impact of competition; the ability to develop our products and technologies; the ability to achieve or obtain necessary regulatory approvals; the impact of changes in the financial markets and global economic conditions; and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and other reports filed by the Company with the Securities and Exchange Commission. The Company undertakes no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.


Cerami M.,IIIA | Straccia U.,CNR Institute of Neuroscience
Information Sciences | Year: 2013

Recently there have been some unexpected results concerning Fuzzy Description Logics (FDLs) with General Concept Inclusions (GCIs). They show that, unlike the classical case, the DL ALC with GCIs does not have the finite model property under Łukasiewicz Logic or Product Logic, the proposed reasoning algorithms are neither correct nor complete and, specifically, knowledge base satisfiability is an undecidable problem for Product Logic. In this work, we show that knowledge base satisfiability is also an undecidable problem for Łukasiewicz Logic. We additionally provide a decision algorithm for acyclic ALC knowledge bases under Łukasiewicz Logic via a Mixed Integer Linear Programming (MILP) based procedure (note, however, that the decidability of this problem is already known). While similar MILP based algorithms have been proposed in the literature for acyclic ALC knowledge bases under Łukasiewicz Logic, none of them exhibit formal proofs of their correctness and completeness, which is the additional contribution here. © 2012 Elsevier Inc. All rights reserved.


Company CEO and COO Presentations to be Held on Wednesday, February 22nd NEW YORK and CLEVELAND, Feb. 21, 2017 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO) a leading clinical-stage biopharmaceutical company focused on developing therapies for life-threatening rare genetic diseases, today announced that members of management will present at the following upcoming investor and industry conferences: Investor Conference: 2017 RBC Capital Markets Global Healthcare Conference Presenter:  Jeffrey B. Davis, Chief Operating Officer Date/Time: Wednesday, February 22nd at 3:35 pm EST Location: The Lotte/Palace Hotel, New York City, NY Room: Kennedy II, 4th Floor Live Webcast Link: http://www.veracast.com/webcasts/rbc/healthcare2017/58114482008.cfm Industry Conference: 3rd Annual CRISPR & Precision Genome Editing Congress 2017 Presenter: Timothy J. Miller, Ph.D., Chief Executive Officer Presentation Title: Application of CRISPR Technology in the Development of Therapeutics for Blood Disorders Date/Time: Wednesday, February 22nd at 11:05 am EST Location: Sheraton Boston Hotel, Boston, MA Website: http://crispr-congress.com/ About Abeona: Abeona Therapeutics Inc. is a leading clinical-stage biopharmaceutical company developing gene therapies for life-threatening rare genetic diseases. Abeona's lead programs include ABO-102 (AAV-SGSH) and ABO-101 (AAV-NAGLU), adeno-associated virus (AAV) based gene therapies for Sanfilippo syndrome (MPS IIIA and IIIB, respectively). Abeona is also developing EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB), EB-201 for epidermolysis bullosa (EB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease (JNCL), ABO-202 (AAV-CLN1) gene therapy for treatment of infantile Batten disease (INCL), and ABO-301 (AAV-FANCC) for Fanconi anemia (FA) disorder and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy for rare blood diseases. In addition, Abeona has a plasma-based protein therapy pipeline, including SDF Alpha™ (alpha-1 protease inhibitor) for inherited COPD, using its proprietary SDF™ (Salt Diafiltration) ethanol-free process. For more information, visit www.abeonatherapeutics.com. This press release contains certain statements that are forward-looking within the meaning of Section 27a of the Securities Act of 1933, as amended, and that involve risks and uncertainties. These statements are subject to numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the impact of competition; the ability to develop our products and technologies; the ability to achieve or obtain necessary regulatory approvals; the impact of changes in the financial markets and global economic conditions; our belief that initial signals of biopotency and clinical activity, which suggest that ABO-102 successfully reached target tissues throughout the body, including the central nervous system; our belief that the data demonstrate an early and robust systemic delivery of ABO-102, and the increased reductions in CNS GAG support our approach for intravenous delivery for subjects with Sanfilippo syndromes, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and other reports filed by the Company with the Securities and Exchange Commission. The Company undertakes no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.


News Article | February 23, 2017
Site: globenewswire.com

Securities Class Action Lawsuit lacked any valid basis and was voluntarily dismissed NEW YORK and CLEVELAND, Feb. 23, 2017 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq:ABEO),  a leading clinical-stage biopharmaceutical company focused on developing therapies for life-threatening rare genetic diseases, announced today that on February 14, 2017, the plaintiff voluntarily dismissed the putative securities class action lawsuit he had recently filed against the company and certain members of its management, following the Company’s demand that the case be dismissed because it lacked a valid legal and factual basis. The plaintiff based his Complaint, in its entirety, on allegations that been cut and pasted from an internet blog article.  No payment or any other consideration was paid by, or on behalf of, the Company or its management in connection with the lawsuit’s dismissal. “We are gratified to have brought about the prompt dismissal of this meritless case just two months after it began, as it should never have been filed at all,” said Jordan D. Hershman of Morgan, Lewis & Bockius LLP, lead counsel for the Company. About Abeona: Abeona Therapeutics Inc. is a leading clinical-stage biopharmaceutical company developing gene therapies for life-threatening rare genetic diseases. Abeona's lead programs include ABO-102 (AAV-SGSH) and ABO-101 (AAV-NAGLU), adeno-associated virus (AAV) based genetic therapies for Sanfilippo syndrome (MPS IIIA and IIIB, respectively). Abeona is also developing EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB), EB-201 for epidermolysis bullosa (EB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease (JNCL), ABO-202 (AAV-CLN1) gene therapy for treatment of infantile Batten disease (INCL), and ABO-301 (AAV-FANCC) for Fanconi anemia (FA) disorder and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy for rare blood diseases. In addition, Abeona has a plasma-based protein therapy pipeline, including SDF Alpha™ (alpha-1 protease inhibitor) for inherited COPD, using its proprietary SDF™ (Salt Diafiltration) ethanol-free process. For more information, visit www.abeonatherapeutics.com. This press release contains certain statements that are forward-looking within the meaning of Section 27a of the Securities Act of 1933, as amended, and that involve risks and uncertainties. These statements are subject to numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the impact of competition; the ability to develop our products and technologies; the ability to achieve or obtain necessary regulatory approvals; the impact of changes in the financial markets and global economic conditions; our belief that initial signals of biopotency and clinical activity, which suggest that ABO-102 successfully reached target tissues throughout the body, including the central nervous system; our belief that the data demonstrate an early and robust systemic delivery of ABO-102, and the increased reductions in CNS GAG support our approach for intravenous delivery for subjects with Sanfilippo syndromes, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and other reports filed by the Company with the Securities and Exchange Commission. The Company undertakes no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.


Lysogene, (Paris:LYS) (the “Company” FR0013233475 – LYS) a leading, clinical-stage biotechnology company specializing in gene therapy for rare central nervous system diseases, today announces that the European Medicines Agency (EMA) has granted orphan drug designation to LYS-GM101, the Company’s gene therapy drug candidate for treatment of GM1 Gangliosidosis (GM1). The U.S. Food and Drug Administration also granted an orphan drug designation and a rare pediatric disease designation to LYS-GM101 earlier this year. “The EMA Orphan Drug Designation for LYS-GM101 is a key regulatory milestone further validating the medical plausibility of our approach,” stated Karen Aiach, Founder and Chief Executive Officer of Lysogene. “This designation will further facilitate and accelerate clinical development of our treatment. It is good news for patients suffering from this severe neurodegenerative disease and we look forward to studying this therapy further as we approach our upcoming Phase I/II clinical trial (LYS-GM101) in 2018.” LYS-GM101 is designed to replace a defective gene in the cells of GM1 patients, in order to allow for production of the functional enzyme and to prevent the progressive nature of the neurological damage caused by GM1 in humans. An ODD by the EMA allows a pharmaceutical company to benefit from incentives from the EU to develop a medicine for a rare disease. Applications for ODD are examined by the Committee for Orphan Medicinal Products (COMP), which adopts an opinion that is forwarded to the European Commission (EC). The EC then decides whether to grant an orphan designation for the medicine in question within 30 days of receipt of the COMP opinion. Pharmaceutical companies that obtain ODD benefit from a number of incentives, including protocol assistance, a type of scientific advice specific for designated orphan medicines, and market exclusivity once the medicine is on the market. Fee reductions are also available, depending on the status of the sponsor and the type of service required GM1 is an extremely severe, autosomal recessive disease caused by a mutation in the GLB1 gene encoding for the lysosomal acid beta-balactosidase (ßgal) enzyme. The resulting enzymatic deficiency leads to accumulation of GM1-ganglioside in cells. Clinical presentation is mainly neurological with rapidly progressive impairment (motor, cognitive and behavioral) leading to premature death, mostly in early childhood. It is a devastating disease for patients and families. There is currently no disease modifying treatment available. Lysogene is a clinical stage biotechnology company pioneering the basic research and clinical development of AAV gene therapy for CNS disorders with a high unmet medical need. Since 2009, Lysogene has established a solid platform and network, with lead products in Mucopolysaccharidosis Type IIIA and GM1 Gangliosidosis, to become a global leader in orphan CNS diseases. Lysogene has also obtained ODD by the EMA and FDA and rare pediatric designation by the FDA for its MPS IIIA program. Lysogene is listed on the Euronext regulated market in Paris (ISIN code: FR0013233475)


« La désignation de médicament orphelin par l’EMA pour LYS-GM101 est une importante étape réglementaire. Elle valide la crédibilité de notre approche médicale et facilitera et accélérera le développement clinique de notre traitement. C’est une bonne nouvelle pour les patients atteints de cette maladie neurodégénérative grave et nous avons hâte de démarrer l’étude clinique de phase I/II (LYS-GM101) en 2018 », déclare Karen Aiach, Fondatrice et Directrice Générale de Lysogene. Lysogene est une société de biotechnologie au stade clinique, pionnière dans la recherche fondamentale et le développement clinique de thérapies géniques utilisant des vecteurs dérivés de virus adéno-associés pour traiter des maladies rares et mortelles du SNC de l’enfant, pour lesquelles il n’existe aujourd’hui, à la connaissance de la Société, aucun traitement. Depuis 2009, Lysogene a mis en place une solide plateforme et un réseau important, avec des produits innovants dans la MPS IIIA et dans la gangliosidose à GM1. Lysogene a obtenu la désignation de médicament orphelin par la FDA et l’EMA, et la désignation de maladie rare pédiatrique par la FDA, pour le programme MPS IIIA.


What:  Abeona Therapeutics Inc. (Nasdaq:ABEO), a leading clinical-stage biopharmaceutical company focused on developing therapies for life-threatening rare genetic diseases, will visit the Nasdaq MarketSite in Times Square to raise awareness for Rare Disease Day. In honor of the occasion, Timothy J. Miller, Ph.D., President and Chief Executive Officer, will ring the Closing Bell. Social Media: For multimedia features such as exclusive content, photo postings, status updates and video of bell ceremonies, please visit our Facebook page: http://www.facebook.com/NASDAQ. For photos from ceremonies and events, please visit our Instagram page: http://instagram.com/nasdaq For livestream of ceremonies and events, please visit our YouTube page: http://www.youtube.com/nasdaq/live For news tweets, please visit our Twitter page: http://twitter.com/nasdaq For exciting viral content and ceremony photos, please visit our Tumblr page: http://nasdaq.tumblr.com/ Webcast: A live stream of the Nasdaq Closing Bell will be available at: https://new.livestream.com/nasdaq/live or http://www.nasdaq.com/about/marketsitetowervideo.asx Photos: To obtain a hi-resolution photograph of the Market Close, please go to http://business.nasdaq.com/discover/market-bell-ceremonies and click on the market close of your choice. About Abeona Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene therapies for life-threatening rare genetic diseases. Abeona's lead programs include ABO-102 (AAV-SGSH) and ABO-101 (AAV-NAGLU), adeno-associated virus (AAV) based gene therapies for Sanfilippo syndrome (MPS IIIA and IIIB, respectively). Abeona is also developing EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB), EB-201 for epidermolysis bullosa (EB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease (JNCL), ABO-202 (AAV-CLN1) gene therapy for treatment of infantile Batten disease (INCL), and ABO-301 (AAV-FANCC) for Fanconi anemia (FA) disorder and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy for rare blood diseases. In addition, Abeona has a plasma-based protein therapy pipeline, including SDF Alpha™ (alpha-1 protease inhibitor) for inherited COPD, using its proprietary SDF™ (Salt Diafiltration) ethanol-free process. For more information, visit www.abeonatherapeutics.com. About Nasdaq Nasdaq (Nasdaq:NDAQ) is a leading provider of trading, clearing, exchange technology, listing, information and public company services across six continents. Through its diverse portfolio of solutions, Nasdaq enables clients to plan, optimize and execute their business vision with confidence, using proven technologies that provide transparency and insight for navigating today's global capital markets. As the creator of the world's first electronic stock market, its technology powers more than 85 marketplaces in 50 countries, and 1 in 10 of the world's securities transactions. Nasdaq is home to approximately 3,800 listed companies with a market value of $10.1 trillion and nearly 18,000 corporate clients. To learn more, visit: business.nasdaq.com.


NEW YORK and CLEVELAND, Feb. 17, 2017 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq:ABEO): Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused on developing therapies for life-threatening rare genetic diseases, announced updated data from the ongoing gene therapy clinical trial for Sanfilippo syndrome Type A (MPS IIIA), at the 13th Annual WORLDSymposium™ 2017 lysosomal storage disorders conference in San Diego, CA. The ongoing Phase 1/2 trial for ABO-102 (AAV-SGSH) is a first-in-man clinical trial utilizing a single intravenous injection of AAV gene therapy for subjects with MPS IIIA, a rare autosomal recessive disease affecting every cell and organ in the body, which results in neurocognitive decline, speech loss, loss of mobility, and premature death in children. “We remain encouraged by continued signs of tolerability and biopotency in the low-dose cohort, and enrollment of the high-dose cohort is underway,” stated Kevin M. Flanigan, M.D., principal investigator with the Center for Gene Therapy at Nationwide Children’s Hospital and Professor of Pediatrics and Neurology at The Ohio State University College of Medicine. “Additionally, we are pleased to see further decreases in CSF GAG measurements, as well as preliminary evidence for stabilization or improvement of some cognitive functions, at six months post-dosing.” Per the design of the clinical trial, subjects received a single, intravenous injection of ABO-102 to deliver the AAV viral vector systematically throughout the body to introduce a corrective copy of the gene that underlies the cause of the MPS IIIA disease. Subjects are evaluated at multiple time points post-injection for safety assessments and initial signals of biopotency and clinical activity, which suggest that ABO-102 successfully reached target tissues throughout the body, including the central nervous system. Observations reported at the WORLDSymposium™ conference included: “The data demonstrate an early and robust systemic delivery of ABO-102, and the increased reductions in CNS HS GAG support our approach for intravenous ABO-102 delivery for subjects with Sanfilippo syndromes,” stated Timothy J. Miller, Ph.D., President and CEO of Abeona Therapeutics. “We are excited about continued biomarker signals in this trial, as well as early positive signals in the neurocognitive assessments. While we are still very early in the trial, we are extremely encouraged by these early results and look forward to expanding enrollment in this clinical trial with enrollments accelerating at two additional international clinical sites.” Abeona’s MPS IIIA program, ABO-102, has been granted Orphan Product Designation in the USA and in the European Union, has received the Rare Pediatric Disease Designation in the US, and recently received Fast Track designation by the US FDA. Sanfilippo syndromes (or mucopolysaccharidosis (MPS) type III): a group of four inherited genetic diseases each caused by a single gene defect, described as type A, B, C or D, which cause enzyme deficiencies that result in the abnormal accumulation of glycosaminoglycans (GAGs, or sugars) in body tissues. MPS III is a lysosomal storage disease, a group of rare inborn errors of metabolism resulting from deficiency in normal lysosomal function. The incidence of MPS III (all four types combined) is estimated to be 1 in 70,000 births. Mucopolysaccharides are long chains of sugar molecule used in the building of connective tissues in the body. There is a continuous process in the body of replacing used materials and breaking them down for disposal. Children with MPS III are missing an enzyme which is essential in breaking down the used mucopolysaccharides called heparan sulfate. The partially broken down mucopolysaccharides remain stored in cells in the body causing progressive damage. In MPS III, the predominant symptoms occur due to accumulation within the central nervous system (CNS), including the brain and spinal cord, resulting in cognitive decline, motor dysfunction, and eventual death. Importantly, there is no cure for MPS III and treatments are largely supportive care. About Abeona: Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene therapies for life-threatening rare genetic diseases. Abeona's lead programs include ABO-102 (AAV-SGSH) and ABO-101 (AAV-NAGLU), adeno-associated virus (AAV) based gene therapies for Sanfilippo syndrome (MPS IIIA and IIIB, respectively). Abeona is also developing EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB), EB-201 for epidermolysis bullosa (EB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease (JNCL), ABO-202 (AAV-CLN1) gene therapy for treatment of infantile Batten disease (INCL), and ABO-301 (AAV-FANCC) for Fanconi anemia (FA) disorder and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy for rare blood diseases. In addition, Abeona has a plasma-based protein therapy pipeline, including SDF Alpha™ (alpha-1 protease inhibitor) for inherited COPD, using its proprietary SDF™ (Salt Diafiltration) ethanol-free process. For more information, visit www.abeonatherapeutics.com. This press release contains certain statements that are forward-looking within the meaning of Section 27a of the Securities Act of 1933, as amended, and that involve risks and uncertainties. These statements are subject to numerous risks and uncertainties, including but not limited to continued interest in our rare disease portfolio, our ability to enroll patients in clinical trials, the impact of competition; the ability to develop our products and technologies; the ability to achieve or obtain necessary regulatory approvals; the impact of changes in the financial markets and global economic conditions; our belief that initial signals of biopotency and clinical activity, which suggest that ABO-102 successfully reached target tissues throughout the body, including the central nervous system; our belief that the data demonstrate an early and robust systemic delivery of ABO-102, and the increased reductions in CNS GAG support our approach for intravenous delivery for subjects with Sanfilippo syndromes, and other risks as may be detailed from time to time in the Company's Annual Reports on Form 10-K and other reports filed by the Company with the Securities and Exchange Commission. The Company undertakes no obligations to make any revisions to the forward-looking statements contained in this release or to update them to reflect events or circumstances occurring after the date of this release, whether as a result of new information, future developments or otherwise.

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