GlobeNewswire

Abeona Announces FDA Grants RMAT Designation to ABO-102 Gene Therapy in MPS IIIA

Dela

First Gene Therapy Using AAV Approach Granted Regenerative Medicine Advanced Therapy Designation

NEW YORK and CLEVELAND, April 23, 2018 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel cell and gene therapies for life-threatening rare genetic diseases, announced today that the US Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ABO-102, the Company's AAV-mediated gene therapy for the treatment of Sanfilippo syndrome Type A (MPS IIIA), a rare autosomal-recessive lysosomal storage disease.

"We are encouraged to have received the first gene therapy RMAT designation in MPS IIIA and look forward to further collaborating with the FDA to determine next steps in the development pathway for ABO-102," said Carsten Thiel, Ph.D., CEO of Abeona Therapeutics. "This action further reinforces the clinical significance in the data observed in the ongoing Phase 1/2 trial and the high unmet need for effective treatment options for patients suffering from MPS IIIA."

Established under the 21st Century Cures Act, RMAT designation is an expedited program for the advancement and approval of regenerative medicine products. A regenerative medicine is eligible for the designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such a disease or condition. RMAT allows companies developing regenerative medicine and gene therapies to work more closely and frequently with the FDA and grants all of the benefits of Breakthrough Therapy Designation.

The Company continues to engage the FDA on its ongoing Phase 1/2 trial. In the trial, subjects receive a single intravenous injection of ABO-102 to facilitate systemic delivery of a functional copy of the gene (SGSH) associated with onset and progression of MPS IIIA. Subjects are evaluated at multiple time points post-injection for safety, tolerability and efficacy. To date, 11 subjects have been enrolled.  An update on results from the trial will be presented at the American Society for Gene and Cell Therapy (ASGCT) in May this year.  ABO-102 has been granted Rare Pediatric Disease Designation, Fast Track Designation. and Orphan Product Designation in the U.S. and Orphan Drug Designation in the European Union.

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. 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 cell and gene therapies for life-threatening rare genetic diseases. Abeona's lead programs include EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB), ABO-102 (AAV-SGSH), an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome type A (MPS IIIA) and ABO-101 (AAV-NAGLU), an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome type B (MPS IIIB).  Abeona is also developing ABO-201 (AAV-CLN3) gene therapy for CLN3 disease, ABO-202 (AAV-CLN1) for treatment of CLN1 disease, 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 is developing a proprietary vector platform, AIM(TM), for next generation product candidates.  For more information, visit www.abeonatherapeutics.com 

Investor Contact:
Christine Silverstein
SVP, Finance & Investor Relations 
Abeona Therapeutics Inc.
+1 (646) 813-4707
csilverstein@abeonatherapeutics.com  

Media Contact:
Lynn Granito
Berry & Company Public Relations
+1 (212) 253-8881
lgranito@berrypr.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, statements about our ability to develop our products and technologies; our plans for continued development of our products and technologies and internationalization of our clinical programs; that patients will continue to be identified, enrolled, treated and monitored in the ABO-102 clinical trial, and that studies will continue to indicate that ABO-102 is well-tolerated; our expectation that certain of our products may be eligible for priority review and accelerated approval by the FDA; and our expectation that we will continue to advance our gene therapy for MPS IIIA patients.  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, risks associated with data analysis and reporting, 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.




This announcement is distributed by Nasdaq Corporate Solutions on behalf of Nasdaq Corporate Solutions clients.
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

Om

GlobeNewswire



Följ GlobeNewswire

Abonnera på våra pressmeddelanden.

Senaste pressmeddelandena från GlobeNewswire

Arcoma AB: Investerarmöten våren 201915.2.2019 08:46:00Pressmeddelande

15 februari 2019 PRESSRELEASE Arcoma har som ambition att fortsätta öka sitt aktieägarfokus och kontinuerligt förbättra kommunikationen med aktieägare. Under våren 2019 kommer Arcoma medverka på flertalet investerarmöten där aktuella händelser och aktiviteter presenteras. Här ges en ökad inblick i bolagets vision och strategier samt en möjlighet till att ställa frågor till bolagets ledning. SvD Börsplus Temadag "Dubblare" Dag, tid och plats: 5 mars, kl. 13.00, GT 30, Grev Turegatan 30, Stockholm Stockholm Corporate Finance Life Science event Dag, tid och plats: 14 mars, kl. 07.30, Berns, Kammarsalen, Stockholm Redeyes Medtech & Diagnostics event Dag, tid och plats: 14:e maj, Redeye, Mäster Samuelsgatan 42, Stockholm För ytterligare information, vänligen kontakta: Arcomas VD Jesper Söderqvist, Telefon: +46 470 70 69 81, E-mail: jesper.soderqvist at arcoma.se Om Arcoma Arcoma, med lång erfarenhet av branschen, är en ledande leverantör av integrerade digitala röntgensystem med hög kvalite

Corline Biomedical AB: Första patient doserad med Renaparin®15.2.2019 08:42:00Pressmeddelande

Corline Biomedical AB ("Corline" eller "Bolaget") meddelar idag att man inkluderat första patienten i fas 1-studien RENAPAIR 01. Patienten inkluderades vid transplantationsavdelningen på Uppsala Akademiska sjukhus. Renaparin® är en produkt under utveckling för att förbättra njurtransplantation. I Corlines studie RENAPAIR 01 utvärderas produkten med avseende på tolerabilitet och säkerhet. Studien öppnades för patientrekrytering i slutet av 2018 och första patient är nu doserad och inkluderad i studien. Totalt skall 18 patienter rekryteras och när de 4 första patienterna är utvärderade i Uppsala, öppnas också Karolinska Universitetssjukhuset i Huddinge för rekrytering till studien. Henrik Nittmar, VD i Corline Biomedical AB, kommenterar "Det händer mycket i Corline för tillfället och alla arbetar hårt för att målbilden i bolaget skall uppfyllas. Det gäller såväl vår interna organisation som våra externa samarbetspartners. Därför är det extra roligt när vi når avgörande milstolpar i utvec

Corline Biomedical AB: Rekryterar Lars Olsson som Head of CMC14.2.2019 12:44:00Pressmeddelande

Corline Biomedical AB ("Corline" eller "Bolaget") meddelar idag att man rekryterat Lars Olsson till den nyinrättade tjänsten som Head of CMC (Chemistry, Manufacturing & Control). Lars Olsson hade tidigare samma tjänst hos Wilson Therapeutics AB, som under 2018 förvärvades av amerikanska Alexion. Lars Olsson kommer att ansvara för Corlines samtliga aktiviteter inom tillverkning och kontroll relaterat till bolagets läkemedelsprodukter, med särskilt fokus på Renaparin® inför kommande fas 2- och fas 3-utveckling. I slutet av 2018 meddelade Corline att patientrekryteringen startar i bolagets fas 1-studie RENAPAIR 01, där produkten Renaparin®, som är under utveckling för att förbättra utfallet av njurtransplantation, utvärderas med avseende på tolerabilitet och säkerhet. Därmed tog en ny utvecklingsfas vid i bolaget i vilken alltmer resurser fokuseras på att förbereda den fortsatta kliniska utvecklingen av produkten genom fas 2 och fas 3 i syfte att slutligen kunna registrera produkten för k

Arcoma AB: Canon och Arcoma tecknar distributörsavtal för Turkiet14.2.2019 10:38:00Pressmeddelande

14 februari 2019 Canon Medical Systems i Turkiet och Arcoma har tecknat ett distributörsavtal som gör att Canon kommer att kunna sälja Arcomas produkter i Turkiet från och med den 15 februari 2019. Canon är sedan tidigare återförsäljare för Arcomas produkter i Nordamerika och utvalda länder i Europa. Detta samarbete utökas nu till Turkiet som är det första land i Canons Asien organisation att välja Arcoma som en formell partner. "Att vi stärker och formaliserar samarbetet med Canon även i Turkiet är ännu ett erkännande av Arcoma som leverantör av högkvalitativa röntgensystem. Canon i Turkiet förbereder ett flertal större upphandlingar till sjukhus där Arcomas lösningar ingår. Avtal som detta ingår i Arcomas strategi att utöka med nya försäljningskanaler och att expandera till nya länder för att skapa framtida tillväxt." kommenterar Arcomas VD, Jesper Söderqvist. För ytterligare information, vänligen kontakta: Arcomas VD Jesper Söderqvist, Telefon: +46 470 70 69 81, E-mail: jesper.soder

Karolinska Development's portfolio company OssDsign raises SEK 64m and aims for listing on Nasdaq First North12.2.2019 09:00:00Pressmeddelande

STOCKHOLM, February 12, 2019. Karolinska Development's portfolio company OssDsign announces today the closing of a private placement of SEK 64 million. Swedish private investors and the French investment management company Alto Invest have taken part in the private placement. OssDsign will now begin the process towards a listing on Nasdaq First North in 2019. The proceeds from the private placement will be used for continued sales expansion in Europe and the US of OssDsign regenerative implant solutions for improved healing of bone defects. Moreover, funds will be used for entering new markets, primarily preparing for market introduction in Japan where the company in parallel to the regulatory process will initiate pre marketing activities. With ethical committee approval already in place, leading surgeons in Japan can start building experience with the OssDsign technology prior to regulatory approval and launch. OssDsign prepares for a listing on Nasdaq Stockholm First North in 2019.

Karolinska Development's portfolio company OssDsign raises SEK 64m and aims for listing on Nasdaq First North12.2.2019 09:00:00Pressmeddelande

STOCKHOLM, February 12, 2019. Karolinska Development's portfolio company OssDsign announces today the closing of a private placement of SEK 64 million. Swedish private investors and the French investment management company Alto Invest have taken part in the private placement. OssDsign will now begin the process towards a listing on Nasdaq First North in 2019. The proceeds from the private placement will be used for continued sales expansion in Europe and the US of OssDsign regenerative implant solutions for improved healing of bone defects. Moreover, funds will be used for entering new markets, primarily preparing for market introduction in Japan where the company in parallel to the regulatory process will initiate pre marketing activities. With ethical committee approval already in place, leading surgeons in Japan can start building experience with the OssDsign technology prior to regulatory approval and launch. OssDsign prepares for a listing on Nasdaq Stockholm First North in 2019.

I vårt pressrum kan du läsa de senaste pressmeddelandena, få tillgång till pressmaterial och hitta kontaktinformation.

Besök vårt pressrum