Abeona Therapeutics Announces Pivotal Expansion of ABO-102 Gene Therapy Clinical Trials in Sanfilippo Syndrome Type A
NEW YORK and CLEVELAND, Aug. 16, 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, today announced the pivotal expansion of its gene therapy clinical trials for patients with MPS IIIA in the USA, Europe and Australia.
"We have completed the necessary regulatory and ethical committee approvals and site initiations in Europe and Australia in order to accelerate enrollment," stated Juan Ruiz, M.D., Ph.D., Abeona's Chief Medical Officer. "We remain very encouraged by the improvements observed in clinically relevant biomarkers post-dosing of ABO-102, including durable reductions in heparan sulfate measured in the CNS, reduction of organ disease pathology, and signals of CNS improvement or stabilization at one-year follow-up in Cohort 1 subjects, and look forward to providing a more fulsome clinical update at important clinical conferences, including ESGCT, this fall," he continued.
The planned expansion will enroll an additional eight to ten MPS IIIA subjects, with total enrollment of fourteen to sixteen subjects to be completed by 1Q2018. Per the design of the clinical trial, subjects will receive a single, intravenous injection of ABO-102 to deliver the AAV viral vector systemically 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.
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 proprietary vector platform, AIM(TM), for next generation product candidates. For more information, visit www.abeonatherapeutics.com.
Vice President, Investor Relations
Abeona Therapeutics Inc.
Vice President, Communications & Operations
Abeona Therapeutics Inc.
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, our plans for continued development and internationalization of our clinical programs, that patients will continue to be identified, enrolled, treated and monitored in the EB-101 clinical trial, and that studies will continue to indicate that EB-101 is well-tolerated and may offer significant improvements in wound healing; the addition of two additional global clinical sites 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 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.
The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.
Source: Abeona Therapeutics Inc via Globenewswire
Följ NASDAQ OMX
Abonnera på våra pressmeddelanden.
Senaste pressmeddelandena från NASDAQ OMX
Fura Completes Acquisition of Colombian Emerald Mine, Coscuez22.1.2018 13:00 | Pressmeddelande
TORONTO, Jan. 22, 2018 (GLOBE NEWSWIRE) -- Fura Gems Inc. (TSXV:FURA) is pleased to announce that it has completed its acquisition of 76% of the issued and outstanding shares of Esmeracol S.A. ("Esmeracol"), which owns a 100% interest in mining licence no. 122 - 95M (the "Coscuez Licence"). Dev Shetty, President & CEO of Fura, commented: "Fura has achieved one more important milestone by adding the iconic Colombian emerald mine, Coscuez, to its portfolio. With this acquisition, Fura now has two important silos in its portfolio, Colombian emeralds and Mozambican rubies. Fura will now work towards modernisation of the Coscuez emerald mine and deployment of the best health and safety standards for its employees while keeping the environmental impact minimal. Fura will also closely work with the local community and relevant authorities to promote the social-economic development in the region. We would like to thank our local partner and also the entire team on the ground in the Colombia fo
Agency for the Cooperation of Energy Regulators (ACER) continues to use Nasdaq's SMARTS for Pan-European Market Monitoring of EU Wholesale Energy Markets22.1.2018 12:30 | Pressmeddelande
NEW YORK, Jan. 22, 2018 (GLOBE NEWSWIRE) -- The Agency for the Cooperation of Energy Regulators (ACER) has officially extended its contract with Nasdaq (Nasdaq:NDAQ) to continue to leverage Nasdaq's SMARTS Market Surveillance technology to detect insider trading and market manipulation across the European Union's wholesale energy markets by digesting and analyzing massive volumes of data from hundreds of power producers, exchanges, operators, and brokers across 28 member states. The new agreement contract extension will continue to allow national regulatory authorities to share data, technology, and expertise, with the aim of creating a cooperative regulatory infrastructure. Under the contract, SMARTS technology will continue being leveraged to monitor the European wholesale gas and electricity markets, covering both spot trading and derivative markets across Europe in accordance with the obligations under the REMIT legislation. Further, the surveillance technology will support ACER's
Bell Canada Reaches Milestone in Network 3.0 Vision with Open Network Automation Platform (ONAP) and Strategic Partnership with Amdocs22.1.2018 12:00 | Pressmeddelande
Bell's leadership in bringing open source ONAP into production enables innovative new offerings to customers faster and at lower cost ST. LOUIS, Jan. 22, 2018 (GLOBE NEWSWIRE) -- Amdocs (NASDAQ:DOX), a leading provider of software and services to communications and media companies, today announced that Bell, Canada's largest communications company, has successfully implemented the first network automation use case in production leveraging the Linux Foundation's Open Network Automation Platform (ONAP). As Bell's strategic partner, Amdocs brings unparalleled ONAP expertise and DevOps scrum team agility and delivery capabilities with the goal of driving innovation faster and reducing the cost of building and operating the data center of the future. With clear benefits such as a more elastic network and increased business agility to be gained from taking advantage of network functions virtualization (NFV), Bell is driving its network transformation by leveraging the ONAP-based network serv
Alvogen CEO Robert Wessman speaks exclusively to World Finance about new opportunities in generic pharmaceuticals22.1.2018 11:51 | Pressmeddelande
LONDON, Jan. 22, 2018 (GLOBE NEWSWIRE) -- Industry disruptor Robert Wessman, the CEO of rapidly growing generic pharmaceuticals company Alvogen, shares his thoughts on the latest market trends in the new edition of World Finance magazine. Only different in name and price, generic pharmaceuticals in the US are a whopping 50 to 70 percent less expensive than their branded equivalents. Unsurprisingly, in an era of ever-rising medical costs, a growing number of governmental entities and corporations are now choosing generics over branded pharmaceuticals. For some time, just a handful of big-name organisations dominated this nascent market. But, following its peak in 2015, over-valuations have prompted vulnerability, opening the door for new, more nimble competitors to join and disrupt the market. Among this innovative group of 'next generation' pharmaceutical companies is Reykjavik-based Alvogen. Though it was only established in 2009, this global-facing organisation now boasts an annual t
Hat-trick for LeoVegas at EGR Nordics Awards 201822.1.2018 08:00 | Pressmeddelande
The most successful and innovative gaming companies are noticed at the EGR Nordic Awards. In fierce competition, LeoVegas won three awards, "Nordics Operator of the Year", "Casino Operator of the Year" and "Sports Betting Operator of the Year". "We are extremely pleased to have won the Casino Operator and the Sports Betting Operator awards as these are the two main categories that define our product. Being declared as the Nordics Operator of the Year is really outstanding! To receive these awards is a receipt that we are an extremely talented team at LeoVegas that is constantly delivering. It also shows that we are the GameTech company that is leading the way within the Nordic region," says Gustaf Hagman, Group CEO. Nordics Operator of the Year LeoVegas won the award for the Nordics Operator of the Year because LeoVegas has been the driving force for innovation in all parts of the company and clearly distinguished from its competitors through a mix of high growth, innovation and strong
Immunicum AB (publ) meddelar kommande presentationer och deltagande i konferenser under januari och februari22.1.2018 08:00 | Pressmeddelande
Pressmeddelande 22 januari 2018 Immunicum AB (publ) meddelar kommande presentationer och deltagande i konferenser under januari och februari Immunicum AB (IMMU.ST), ett bioteknikbolag som utvecklar nya immunostimulerande cancerbehandlingar för ett antal solida tumörformer, meddelar idag att Immunicums ledning kommer att presentera bolaget och delta under flera kommande branschkonferenser och forskningskonferenser under januari och februari. ASCO/SITC - Clinical Immuno-Oncology Symposium Datum: 25-27 januari 2018 Plats: San Francisco Marriott Marquis, San Francisco, Kalifornien, USA Aktiedagen Lund Datum: 30 januari 2018 Presentation: tisdag den 30 januari, 10:15 CET Plats: Medicon Village, Hörsalen Scheelevägen 2, Lund För att delta, registrera dig här https://www.aktiespararna.se/aktiviteter/aktiedagen-lund-30-januari Cholangiocarcinoma Foundation Annual Conference Datum: 31 januari-2 februari 2018 Presentation: Poster Session: torsdag den 1 februari, kl. 19:00-21:00 Mountain Standard
I vårt pressrum kan du läsa de senaste pressmeddelandena, få tillgång till pressmaterial och hitta kontaktinformation.Besök vårt pressrum