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News Article | May 10, 2017
Site: www.prweb.com

Flash Global, a leading provider of end-to-end service supply chain solutions on a global scale, recently hosted a group of graduate students from The Ohio State University Fisher College of Business, giving them the opportunity to gain real-world experience in addressing logistics and service supply chain business needs. The event took place April 26 at the company’s state-of-the-art Global Service Center in Lockbourne, Ohio. “As Flash Global continues to define the service supply chain and drive the strategic value it brings to companies of all sizes in the international marketplace, we’re always looking for new, innovative ways to refine operations and processes to meet customers’ needs. Partnering with best and brightest students enrolled Masters of Business Logistics Engineering (MBLE) program has enabled us to design and execute on six engineering-related projects that have led to streamlined efficiencies in aspects of our business,” said Ted Waggoner, Sr. Director, IT PMO for Flash. The prestigious MBLE program is designed to enable students to enter the job market equally comfortable with logistics strategy, the management of logistics operations, and engineering tasks. This internship-like collaboration with Flash, which started in 2016, has afforded opportunities for 35 students to work on projects such as reducing cycle times in the Repair and Configure to Order lines, creation of Value Stream Mapping, Receiving Area Efficiency Improvement Analysis and more. “Flash has benefited from the energetic and innovative approach of these international students, while providing them with access to a world-class facility and the opportunity to develop real-world, real-time strategic analysis for service supply chain solutions through collaboration with our expert teams. We could not be more thrilled with the relationship we are developing with The Ohio State University and its MBLE program. Although we just completed our second engagement, our teams are already looking forward to next year’s group of students and the projects we will work on together,” said Sam Mikles, President and CEO of Flash Global. To learn more about custom service supply chain solutions designed from an integrated suite of services that are flexible and scalable to meet customer demands today and in the future, connect with Flash Global today. ABOUT FLASH GLOBAL Headquartered in New Jersey (USA), Flash Global provides the industry’s most comprehensive end-to-end suite of global service supply chain solutions that support many of the top OEMs in the world with either emerging or established technologies. Committed to a relentless pursuit of excellence, Flash offers an immense global infrastructure that enables companies to instantly scale in 140+ countries, creating consistency, predictability and visibility into their service supply chain. Flash has in-region and in-country expertise across its Global Command Centers, Global Service Centers, and immense network of global stocking and import/export locations to service OEMs’ customer bases.


--Positive dose response in central nervous system with 60.7% +/- 8.8% reduction of disease-causing heparan sulfate GAG observed in Cohort 2 --Reduction of disease manifestation observed in decreased liver volume of 14.81% (+/- 1.2%) --ABO-102 well-tolerated in six subjects through 1100 days follow up with no Serious Adverse Events --Cohort 1 demonstrated stabilized or improved Leiter Nonverbal IQ scores at six months --Conference call today at 10:00am EDT; 877-269-7756 for domestic callers and 201-689-7817 for international callers NEW YORK and CLEVELAND, May 12, 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 updated data from the ongoing gene therapy clinical trial for Sanfilippo syndrome Type A (MPS IIIA) at the American Society Gene and Cell Therapy (ASGCT) 20th Annual Meeting. 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 Sanfilippo syndrome (MPS IIIA), a rare autosomal-recessive lysosomal storage disease. “Abeona continues to advance gene therapy for MPS IIIA patients and we are excited about the positive dose response in the CNS seen in Cohort 2. The observation of a dose response supports our clinical approach, and we are encouraged to observe further reductions in central nervous system (CNS) heparan sulfate, reduction in liver volume, and preliminary evidence of slowed neurocognitive decline, are very encouraging. We look forward to accelerating enrollment with the recently initiated global sites (Spain and Australia) and reporting additional clinical data in the ABO-102 global MPS IIIA trial later this year,” stated Timothy J. Miller, Ph.D., President and CEO of Abeona Therapeutics. 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. Highlights reported data on five (n=3 Cohort 1, n=2 Cohort 2) out of the six patients treated to date in the gene therapy trial included: Biopotency: positive dose response observed in Cohort 2. --At 30 days post-injection, two patients in the Cohort 2 demonstrated 60.7%(+/- 8.8%) reduction in cerebral spinal fluid (CSF) heparan sulfate (HS). Hepatosplenomegaly: consistent reduction in liver volume observed. --At 30 days post-injection, Cohort 2 subjects demonstrated reductions in liver volumes of 14.81% (+/- 1.2%). --The natural history study in 25 subjects with MPS III (Truxal et. al., 2016, Mol. Genet. Metab.) demonstrated that subjects had increased liver volumes averaging 116% at baseline that did not change over a year of follow-up. Cognitive Assessments: evidence of cognitive stabilization at six months in Cohort 1. --Cognitive assessments, taken at baseline, at the six-month timepoint for the Cohort 1 (n=3), subjects showed evidence of stabilization or improvement in the Leiter-R non-verbal IQ and Vineland (adaptive behavior) scales. --Cognitive assessments are taken at six-month and twelve-month follow-up visits. --Leiter Nonverbal IQ assessments in Cohort 1 subjects demonstrated stabilized or improved scores at six-months post-injection. Notably, one subject improved +10 (+/-6) points, while age-matched controls in the Natural History study would have predicted a decrease of -11.1 (+/-2.7) points over 6 months. --Vineland assessments in Cohort 1 at six months post-injection suggest stabilization in adaptive behavior scores. Safety: well-tolerated in all subjects through 1100 days cumulative post-injection. --No serious adverse events (SAEs) reported in subjects in either cohort receiving ABO-102 (Cohort 1: 5E12 vg/kg and Cohort 2: 1E13 vg/kg). “We remain encouraged by signs of tolerability and biological effects that we have observed in Cohort 1 and in the initial two subjects of Cohort 2,” stated Kevin M. Flanigan, M.D., principal investigator, Director of the Center for Gene Therapy at Nationwide Children’s Hospital and Professor of Pediatrics and Neurology at The Ohio State University College of Medicine. “We are pleased to see decreases in CSF HS compared to the Cohort 1 at 30 days post-injection, and we look forward to enrolling additional high-dose patients.” The ongoing Phase 1/2 clinical trial, which has received FastTrack designation, Orphan Product Designation, and Rare Pediatric Disease designation by the FDA, is designed to evaluate safety and efficacy of ABO-102 in patients with MPS IIIA.  The global clinical study is supported by a 25-subject MPS III Natural History Study, which included potential efficacy assessments consisting of neurocognitive evaluations, biochemical assays and MRI data generated over one year of follow-up assessments. Company Conference Call Details: Abeona will host a live conference call briefing today 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. 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), 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 include without limitation the statement that the addition of two additional global clinical site will accelerate our ability to enroll and evaluate ABO-102 as a potential treatment for patients with Sanfilippo syndrome type A, or MPS IIIA.  Such 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 secure licenses for any technology that may be necessary to commercialize our products; 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 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 quarterly reports on Form 10-Q 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.


--Positive dose response in central nervous system with 60.7% +/- 8.8% reduction of disease-causing heparan sulfate GAG observed in Cohort 2 --Reduction of disease manifestation observed in decreased liver volume of 14.81% (+/- 1.2%) --ABO-102 well-tolerated in six subjects through 1100 days follow up with no Serious Adverse Events --Cohort 1 demonstrated stabilized or improved Leiter Nonverbal IQ scores at six months --Conference call today at 10:00am EDT; 877-269-7756 for domestic callers and 201-689-7817 for international callers NEW YORK and CLEVELAND, May 12, 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 updated data from the ongoing gene therapy clinical trial for Sanfilippo syndrome Type A (MPS IIIA) at the American Society Gene and Cell Therapy (ASGCT) 20th Annual Meeting. 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 Sanfilippo syndrome (MPS IIIA), a rare autosomal-recessive lysosomal storage disease. “Abeona continues to advance gene therapy for MPS IIIA patients and we are excited about the positive dose response in the CNS seen in Cohort 2. The observation of a dose response supports our clinical approach, and we are encouraged to observe further reductions in central nervous system (CNS) heparan sulfate, reduction in liver volume, and preliminary evidence of slowed neurocognitive decline, are very encouraging. We look forward to accelerating enrollment with the recently initiated global sites (Spain and Australia) and reporting additional clinical data in the ABO-102 global MPS IIIA trial later this year,” stated Timothy J. Miller, Ph.D., President and CEO of Abeona Therapeutics. 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. Highlights reported data on five (n=3 Cohort 1, n=2 Cohort 2) out of the six patients treated to date in the gene therapy trial included: Biopotency: positive dose response observed in Cohort 2. --At 30 days post-injection, two patients in the Cohort 2 demonstrated 60.7%(+/- 8.8%) reduction in cerebral spinal fluid (CSF) heparan sulfate (HS). Hepatosplenomegaly: consistent reduction in liver volume observed. --At 30 days post-injection, Cohort 2 subjects demonstrated reductions in liver volumes of 14.81% (+/- 1.2%). --The natural history study in 25 subjects with MPS III (Truxal et. al., 2016, Mol. Genet. Metab.) demonstrated that subjects had increased liver volumes averaging 116% at baseline that did not change over a year of follow-up. Cognitive Assessments: evidence of cognitive stabilization at six months in Cohort 1. --Cognitive assessments, taken at baseline, at the six-month timepoint for the Cohort 1 (n=3), subjects showed evidence of stabilization or improvement in the Leiter-R non-verbal IQ and Vineland (adaptive behavior) scales. --Cognitive assessments are taken at six-month and twelve-month follow-up visits. --Leiter Nonverbal IQ assessments in Cohort 1 subjects demonstrated stabilized or improved scores at six-months post-injection. Notably, one subject improved +10 (+/-6) points, while age-matched controls in the Natural History study would have predicted a decrease of -11.1 (+/-2.7) points over 6 months. --Vineland assessments in Cohort 1 at six months post-injection suggest stabilization in adaptive behavior scores. Safety: well-tolerated in all subjects through 1100 days cumulative post-injection. --No serious adverse events (SAEs) reported in subjects in either cohort receiving ABO-102 (Cohort 1: 5E12 vg/kg and Cohort 2: 1E13 vg/kg). “We remain encouraged by signs of tolerability and biological effects that we have observed in Cohort 1 and in the initial two subjects of Cohort 2,” stated Kevin M. Flanigan, M.D., principal investigator, Director of the Center for Gene Therapy at Nationwide Children’s Hospital and Professor of Pediatrics and Neurology at The Ohio State University College of Medicine. “We are pleased to see decreases in CSF HS compared to the Cohort 1 at 30 days post-injection, and we look forward to enrolling additional high-dose patients.” The ongoing Phase 1/2 clinical trial, which has received FastTrack designation, Orphan Product Designation, and Rare Pediatric Disease designation by the FDA, is designed to evaluate safety and efficacy of ABO-102 in patients with MPS IIIA.  The global clinical study is supported by a 25-subject MPS III Natural History Study, which included potential efficacy assessments consisting of neurocognitive evaluations, biochemical assays and MRI data generated over one year of follow-up assessments. Company Conference Call Details: Abeona will host a live conference call briefing today 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. 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), 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 include without limitation the statement that the addition of two additional global clinical site will accelerate our ability to enroll and evaluate ABO-102 as a potential treatment for patients with Sanfilippo syndrome type A, or MPS IIIA.  Such 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 secure licenses for any technology that may be necessary to commercialize our products; 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 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 quarterly reports on Form 10-Q 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.


--Positive dose response in central nervous system with 60.7% +/- 8.8% reduction of disease-causing heparan sulfate GAG observed in Cohort 2 --Reduction of disease manifestation observed in decreased liver volume of 14.81% (+/- 1.2%) --ABO-102 well-tolerated in six subjects through 1100 days follow up with no Serious Adverse Events --Cohort 1 demonstrated stabilized or improved Leiter Nonverbal IQ scores at six months --Conference call today at 10:00am EDT; 877-269-7756 for domestic callers and 201-689-7817 for international callers NEW YORK and CLEVELAND, May 12, 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 updated data from the ongoing gene therapy clinical trial for Sanfilippo syndrome Type A (MPS IIIA) at the American Society Gene and Cell Therapy (ASGCT) 20th Annual Meeting. 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 Sanfilippo syndrome (MPS IIIA), a rare autosomal-recessive lysosomal storage disease. “Abeona continues to advance gene therapy for MPS IIIA patients and we are excited about the positive dose response in the CNS seen in Cohort 2. The observation of a dose response supports our clinical approach, and we are encouraged to observe further reductions in central nervous system (CNS) heparan sulfate, reduction in liver volume, and preliminary evidence of slowed neurocognitive decline, are very encouraging. We look forward to accelerating enrollment with the recently initiated global sites (Spain and Australia) and reporting additional clinical data in the ABO-102 global MPS IIIA trial later this year,” stated Timothy J. Miller, Ph.D., President and CEO of Abeona Therapeutics. 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. Highlights reported data on five (n=3 Cohort 1, n=2 Cohort 2) out of the six patients treated to date in the gene therapy trial included: Biopotency: positive dose response observed in Cohort 2. --At 30 days post-injection, two patients in the Cohort 2 demonstrated 60.7%(+/- 8.8%) reduction in cerebral spinal fluid (CSF) heparan sulfate (HS). Hepatosplenomegaly: consistent reduction in liver volume observed. --At 30 days post-injection, Cohort 2 subjects demonstrated reductions in liver volumes of 14.81% (+/- 1.2%). --The natural history study in 25 subjects with MPS III (Truxal et. al., 2016, Mol. Genet. Metab.) demonstrated that subjects had increased liver volumes averaging 116% at baseline that did not change over a year of follow-up. Cognitive Assessments: evidence of cognitive stabilization at six months in Cohort 1. --Cognitive assessments, taken at baseline, at the six-month timepoint for the Cohort 1 (n=3), subjects showed evidence of stabilization or improvement in the Leiter-R non-verbal IQ and Vineland (adaptive behavior) scales. --Cognitive assessments are taken at six-month and twelve-month follow-up visits. --Leiter Nonverbal IQ assessments in Cohort 1 subjects demonstrated stabilized or improved scores at six-months post-injection. Notably, one subject improved +10 (+/-6) points, while age-matched controls in the Natural History study would have predicted a decrease of -11.1 (+/-2.7) points over 6 months. --Vineland assessments in Cohort 1 at six months post-injection suggest stabilization in adaptive behavior scores. Safety: well-tolerated in all subjects through 1100 days cumulative post-injection. --No serious adverse events (SAEs) reported in subjects in either cohort receiving ABO-102 (Cohort 1: 5E12 vg/kg and Cohort 2: 1E13 vg/kg). “We remain encouraged by signs of tolerability and biological effects that we have observed in Cohort 1 and in the initial two subjects of Cohort 2,” stated Kevin M. Flanigan, M.D., principal investigator, Director of the Center for Gene Therapy at Nationwide Children’s Hospital and Professor of Pediatrics and Neurology at The Ohio State University College of Medicine. “We are pleased to see decreases in CSF HS compared to the Cohort 1 at 30 days post-injection, and we look forward to enrolling additional high-dose patients.” The ongoing Phase 1/2 clinical trial, which has received FastTrack designation, Orphan Product Designation, and Rare Pediatric Disease designation by the FDA, is designed to evaluate safety and efficacy of ABO-102 in patients with MPS IIIA.  The global clinical study is supported by a 25-subject MPS III Natural History Study, which included potential efficacy assessments consisting of neurocognitive evaluations, biochemical assays and MRI data generated over one year of follow-up assessments. Company Conference Call Details: Abeona will host a live conference call briefing today 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. 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), 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 include without limitation the statement that the addition of two additional global clinical site will accelerate our ability to enroll and evaluate ABO-102 as a potential treatment for patients with Sanfilippo syndrome type A, or MPS IIIA.  Such 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 secure licenses for any technology that may be necessary to commercialize our products; 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 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 quarterly reports on Form 10-Q 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 | May 12, 2017
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BOCA RATON, FL, May 12, 2017-- John Samuel Faulkner is a celebrated Marquis Who's Who biographee. As in all Marquis Who's Who biographical volumes, individuals profiled are selected on the basis of current reference value. Factors such as position, noteworthy accomplishments, visibility, and prominence in a field are all taken into account during the selection process.Marquis Who's Who, the world's premier publisher of biographical profiles, is proud to name Dr. Faulkner a Lifetime Achiever. An accomplished listee, Dr. Faulkner celebrates many years' experience in his professional network, and has been noted for achievements, leadership qualities, and the credentials and successes he has accrued in his field.A respected and long-standing figure in his field, Dr. Faulkner currently is an emeritus professor in the department of physics at Florida Atlantic University, where he has served for over 30 years.In addition to his status as a Lifetime Achiever, Dr. Faulkner has previously been recognized as a Dupont Postgraduate Fellow at The Ohio State University, visited the University of Sheffield, England, as a Senior Fulbright Research Scholar, and was given the Outstanding Achievement Award and Professorial Excellence Program Award at Florida Atlantic University. Furthermore, Dr. Faulkner earned recognition for Best Sustained Research from the United States Department of Energy, an Outstanding Referee Award from the Editors of American Physical Society journals, and was a featured listee in Who's Who in America and Who's Who in the South and Southwest.In recognition of outstanding contributions to his profession and the Marquis Who's Who community, John Samuel Faulkner has been featured on the Marquis Who's Who Lifetime Achievers website. Please visit http://wwlifetimeachievement.com/2017/04/19/john-samuel-faulkner/ to view this distinguished honor.About Marquis Who's Who :Since 1899, when A. N. Marquis printed the First Edition of Who's Who in America , Marquis Who's Who has chronicled the lives of the most accomplished individuals and innovators from every significant field of endeavor, including politics, business, medicine, law, education, art, religion and entertainment. Today, Who's Who in America remains an essential biographical source for thousands of researchers, journalists, librarians and executive search firms around the world. Marquis publications may be visited at the official Marquis Who's Who website at www.marquiswhoswho.com


CLAREMONT, Calif.--(BUSINESS WIRE)--The Pitzer Family Foundation donated $1 million to Pitzer College to support the growth and expansion of science facilities at the W.M. Keck Science Department (KSD), a joint program of Pitzer College, Claremont McKenna College and Scripps College. Since 2002, the number of Pitzer students majoring in sciences at KSD has increased by more than 200 percent. The College also saw a 147 percent increase in students receiving science degrees since 2004. In 2016, biology and human biology were among the 10 top majors within Pitzer’s graduating class. More Pitzer students now major in one of the biological sciences, which include biochemistry, biology, biophysics, human biology, molecular and organismal biology, than in any other discipline. “With this gift, the Pitzer family is pleased to continue its support for science at the Claremont Colleges,” said Russell M. Pitzer, Pitzer College emeritus trustee and emeritus professor of chemistry at The Ohio State University, on behalf of the Pitzer Family Foundation. “Kenneth S. Pitzer, son of the founder of Pitzer College, was not only involved with the original plans for the Keck Joint Science building, but also consulted at various times with Pomona College and Harvey Mudd College about their programs. He and his wife, Jean M. Pitzer, endowed three professorships at Pitzer College related to scientific fields. The Pitzer family is delighted that interest in science has increased to the point that the Keck Science Center must be expanded and is happy to support that expansion with this gift.” Due to increased student interest and enrollment in science courses at each of the KSD colleges, this gift provides significant and much-needed support for the department’s continued growth. “The College is grateful to the Pitzer Family Foundation for believing in the value of science education at Pitzer and the Keck Science Department. Support for faculty and students as they pursue their teaching, learning and research in the sciences is an invaluable gift,” said Pitzer College President Melvin L. Oliver. The W.M. Keck Science Department focuses exclusively on undergraduate science education and provides instruction in small-classroom and lab settings while offering numerous opportunities for students to conduct research. KSD is administered cooperatively by the three participating colleges and is a national leader in the development of interdisciplinary science courses and programs. Pitzer College was founded in 1963 by citrus grower and philanthropist Russell K. Pitzer. The Pitzer Family Foundation (PFF) has continued his tradition of generous support of the College. In 2007, the PFF provided $5 million for the construction of Sanborn Hall in Phase I of the College’s Residential Life Project in memory of Flora Sanborn Pitzer, the founder’s wife. The foundation also gave $500,000 to establish the Pitzer Archive and Conference Center in Residential Life Project Phase II in 2012. The founder’s grandchildren, Ann E., Russell M. and John S. Pitzer, contributed to the development of the Jean M. Pitzer Archaeology Laboratory. In addition, members of the Pitzer family have established numerous endowed scholarships, professorships and an endowed directorship of international programs at the College. Pitzer College is a nationally top-ranked undergraduate liberal arts and sciences institution. A member of The Claremont Colleges, Pitzer offers a distinctive approach to a liberal arts education by linking intellectual inquiry with interdisciplinary studies, cultural immersion, social responsibility and community involvement. For more information, please visit www.pitzer.edu.


"NCCN Foundation is proud to support this important resource which indeed will empower patients with thyroid cancer and their caregivers to make informed choices about their care," said Marcie R. Reeder, MPH, Executive Director, NCCN Foundation. "We are incredibly grateful for the generous support from our sponsors, ThyCa and Rockin' for the Cure, as their sponsorship for these resources gives patients access to the same treatment information that their doctors use." "Thyroid Cancer is challenging because it affects people of all ages from young children through seniors, is especially life-disrupting and stressful when diagnosed in younger patients, and needs lifelong monitoring and management even in low-risk patients," said Gary Bloom, ThyCa Executive Director, and Thyroid Cancer survivor of 21 years. "ThyCa is very pleased to support these important guidelines, which will be helpful resources for both patients and their caregivers." NCCN Guidelines for Patients are easy-to-understand adaptations based on the same clinical practice guidelines used by health care professionals around the world to determine the best way to treat a person with cancer. Each resource features unbiased expert guidance from the nation's leading cancer centers designed to help people living with cancer understand and discuss their treatment options with their providers. NCCN Guidelines for Patients and NCCN Quick Guide™ sheets—one-page summaries of key points in the patient guidelines—are written in plain language and include patient-friendly tools, such as questions to ask your doctor, a glossary of terms, and medical illustrations of anatomy, tests, and treatment.  NCCN Guidelines for Patients and NCCN Quick Guide™ sheets DO NOT replace the expertise and clinical judgment of the clinician. NCCN currently offers NCCN Guidelines for Patients for the following: Brain, Breast, Colon Distress, Esophageal, Kidney, Non-Small Cell Lung, Ovarian, Pancreatic, Prostate, Stomach, and Thyroid Cancers; Acute Lymphoblastic Leukemia; Adolescents and Young Adults with Cancer; Chronic Lymphocytic Leukemia; Chronic Myelogenous Leukemia; Hodgkin Lymphoma; Lung Cancer Screening; Malignant Pleural Mesothelioma; Melanoma; Multiple Myeloma; Myelodysplastic Syndromes; Nausea and Vomiting; Non-Hodgkin's Lymphomas; Soft Tissue Sarcoma; and Waldenström's Macroglobulinemia. The NCCN Guidelines for Patients and NCCN Quick Guide™ sheet for Thyroid Cancer are available to download for free at NCCN.org/patients and on the NCCN Patient Guides for Cancer mobile app. NCCN Foundation® was founded by the National Comprehensive Cancer Network® (NCCN®) to empower people with cancer and advance oncology innovation. NCCN Foundation supports people with cancer and their caregivers at every step of their treatment journey by delivering unbiased expert guidance from the world's leading cancer experts through the library of NCCN Guidelines for Patients® and other patient education resources. NCCN Foundation is also committed to advancing cancer treatment by funding the nation's promising young investigators at the forefront of cancer research, initiating momentum in their careers and furthering the betterment of patients through their groundbreaking innovations. For more information about NCCN Foundation, visit NCCNFoundation.org. ThyCa: Thyroid Cancer Survivors' Association, Inc., an international nonprofit organization founded in 1995 and advised by thyroid cancer specialists, educates and supports patients and families through its website, online and face-to-face support groups, one-to-one support, over 50 videos with experts on its YouTube Channel, handbooks on all thyroid cancer types, downloadable low-iodine cookbook, online newsletter, and materials in 10 languages. ThyCa sponsors seminars, workshops, and the annual International Thyroid Cancer Survivors' Conference, as well as Thyroid Cancer Awareness Month, year-round awareness programs for early detection, and thyroid cancer research funds and research grants. For more information visit http://www.thyca.org. About the National Comprehensive Cancer Network The National Comprehensive Cancer Network® (NCCN®), a not-for-profit alliance of 27 leading cancer centers devoted to patient care, research, and education, is dedicated to improving the quality, effectiveness, and efficiency of cancer care so that patients can live better lives. Through the leadership and expertise of clinical professionals at NCCN Member Institutions, NCCN develops resources that present valuable information to the numerous stakeholders in the health care delivery system. As the arbiter of high-quality cancer care, NCCN promotes the importance of continuous quality improvement and recognizes the significance of creating clinical practice guidelines appropriate for use by patients, clinicians, and other health care decision-makers. The NCCN Member Institutions are: Fred & Pamela Buffett Cancer Center, Omaha, NE; Case Comprehensive Cancer Center/University Hospitals Seidman Cancer Center and Cleveland Clinic Taussig Cancer Institute, Cleveland, OH; City of Hope Comprehensive Cancer Center, Los Angeles, CA; Dana-Farber/Brigham and Women's Cancer Center | Massachusetts General Hospital Cancer Center, Boston, MA; Duke Cancer Institute, Durham, NC; Fox Chase Cancer Center, Philadelphia, PA; Huntsman Cancer Institute at the University of Utah, Salt Lake City, UT; Fred Hutchinson Cancer Research Center/Seattle Cancer Care Alliance, Seattle, WA; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, MD; Robert H. Lurie Comprehensive Cancer Center of Northwestern University, Chicago, IL; Mayo Clinic Cancer Center, Phoenix/Scottsdale, AZ, Jacksonville, FL, and Rochester, MN; Memorial Sloan Kettering Cancer Center, New York, NY; Moffitt Cancer Center, Tampa, FL; The Ohio State University Comprehensive Cancer Center - James Cancer Hospital and Solove Research Institute, Columbus, OH; Roswell Park Cancer Institute, Buffalo, NY; Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine, St. Louis, MO; St. Jude Children's Research Hospital/The University of Tennessee Health Science Center, Memphis, TN; Stanford Cancer Institute, Stanford, CA; University of Alabama at Birmingham Comprehensive Cancer Center, Birmingham, AL; UC San Diego Moores Cancer Center, La Jolla, CA; UCSF Helen Diller Family Comprehensive Cancer Center, San Francisco, CA; University of Colorado Cancer Center, Aurora, CO; University of Michigan Comprehensive Cancer Center, Ann Arbor, MI; The University of Texas MD Anderson Cancer Center, Houston, TX; University of Wisconsin Carbone Cancer Center, Madison, WI; Vanderbilt-Ingram Cancer Center, Nashville, TN; and Yale Cancer Center/Smilow Cancer Hospital, New Haven, CT. [1] Robert I. Haddad, MD, et. al., NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines) for Thyroid Carcinoma, Version 1.2017. © 2017 National Comprehensive Cancer Network, Inc.  Available at NCCN.org. Accessed: May 8, 2017 [2] "Cancer Stat Facts: Thyroid Cancer." Surveillance, Epidemiology, and End Results Program. National Cancer Institute, n.d. Web. 08 May 2017. To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/nccn-publishes-new-patient-education-resources-for-thyroid-cancer----one-of-the-most-commonly-diagnosed-cancers-in-women-in-the-united-states-300456776.html

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