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Abeona Therapeutics to Present Data for ABO-401 in Cystic Fibrosis and Retinal Disorders at the American Society of Gene and Cell Therapy Annual Meeting

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Monday, April 15, 2019

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NEW YORK and CLEVELAND - Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy,

announced that new data demonstrating the capabilities of the AIM™ capsid library in cystic fibrosis and retinal disorders will be presented at the American Society of Gene and Cell Therapy 22nd Annual Meeting, being held April 29 – May 2, 2019 in Washington, D.C. Additional preclinical data for ABO-202, the Company’s AAV9-based gene therapy for the treatment of patients with CLN1 disease, will also be presented.

“The data to be presented at the ASGCT meeting will highlight study results on the delivery and expression of hCFTR gene in cystic fibrosis and the potential of the AIM™ AAV vector platform in the treatment of multiple retinal disorders. New data from IND-enabling studies of our program in CLN1 disease, expected to enter the clinic later this year, will also be presented,” said João Siffert, M.D., Chief Executive Officer.

“The AIM capsid library has demonstrated significant versatility with the ability to deliver genes to target tissues with enhanced specificity,” added Timothy J. Miller, Ph.D., President and Chief Scientific Officer. “This next-generation AAV capsid platform has the potential to transform how we can target pulmonary, retinal and neurological disorders with gene therapy.”

Identification of AAV Developed for Cystic Fibrosis (CF) Gene Therapy That Restores CFTR Function in Human CF Patient Cells
Presenter: Paul T. Wille, Ph.D., Case Western Reserve University, Cleveland, OH; Abeona Therapeutics Inc.
Date/Time: Tuesday April 30, 5:00 – 6:00 p.m. ET
Poster Board Number: 92
Session title: Cardiovascular and Pulmonary Diseases
Room: Columbia Hall
Abstract number: 528

Novel AAV Capsids Demonstrate Strong Retinal Expression in Non-Human Primates After Intravitreal Administration
Presenter: Brian Kevany, Ph.D., Abeona Therapeutics Inc.
Date/Time: Monday April 29, 5:00 – 6:00 p.m. ET
Session title: Neurologic Diseases
Room: Columbia Hall
Abstract number: 225

Intrathecal and Intravenous Combination Gene Therapy in the Mouse Model of Infantile Neuronal Ceroid Lipofuscinosis Extends Lifespan and Improves Behavioral Outcomes in Moderately Affected Mice
Presenter: Erik A. Lykken, Ph.D., University of Texas Southwestern Medical Center, Dallas, TX
Date/Time: Monday April 29, 11:45 a.m. – 12:00 p.m. ET
Session title: Tools, Delivery and Neuro Capsids
Room: Monroe

About Abeona Therapeutics 
Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing gene and cell therapies for serious diseases. The Company’s clinical programs include EB-101, its autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, as well as ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo syndrome types A and B (MPS IIIA and MPS IIIB). The Company’s portfolio of AAV9-based gene therapies also features ABO-202 and ABO-201 for CLN1 disease and CLN3 disease, respectively. Its preclinical assets include ABO-401, which uses the novel AIM™ AAV vector platform to address all mutations of cystic fibrosis. Abeona has received numerous regulatory designations from the FDA and EMA for its pipeline candidates and is the only company with Regenerative Medicine Advanced Therapy designation for two investigational therapies (EB-101 and ABO-102). For more information, visit www.abeonatherapeutics.com.

Investor Contact:
Sofia Warner
Senior Director, Investor Relations
Abeona Therapeutics
+1 (646) 813-4710
swarner@abeonatherapeutics.com

Media Contact:
Scott Santiamo
Director, Corporate Communications
Abeona Therapeutics
+1 (718) 344-5843
ssantiamo@abeonatherapeutics.com

Source: Abeona Therapeutics
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