About the Company
Abeona Therapeutics Inc. (NASDAQ: ABEO) is a clinical-stage biopharmaceutical company focused on developing novel therapies for life-threatening rare genetic diseases. Developing therapies for rare disease requires new approaches and strong collaboration between researchers, industry, regulators and patient groups. Abeona was forged from the company’s close collaborations with key stakeholders all dedicated to transforming new biotechnology insights into breakthrough treatments for rare diseases.
Advancing treatments for rare diseases…Working together to find a cure.
cureA rare disease is one that affects fewer than 200,000 people in the United States. There are nearly 7,000 rare diseases, which may involve chronic illness, disability, and often, premature death. More than 25 million Americans and 30 million Europeans have one.
While rare diseases can affect any age group, about 50% of people affected are children (15 million); and rare diseases account for 35% of deaths in the first year of life.
These rare diseases are often poorly diagnosed, very complex, and have no treatment or not very effective treatment—over 95% of rare diseases do not have a single FDA or EMA approved drug treatment. However, most rare diseases are often caused by changes in genes—80% are genetic in origin and can present at any stage of life.
We believe emerging insights in genetics and advances in biotechnology, as well as new approaches and collaboration between researchers, industry, regulators and patient groups, provide significant opportunities to develop breakthrough treatments for rare diseases.
Abeona Announces $13.85M Grant from Leading Sanfilippo Syndrome Foundations for Clinical Development of MPS III Gene Therapies
Nine global foundations collaborate to grant $13.85 million for the continued advancement of lead Sanfilippo gene therapy programs Company receives infusion of an additional $5 million from exercise of outstanding warrants, totaling $18.85 million in proceeds NEW YORK and CLEVELAND, Oct....
Abeona Therapeutics Enrolls First Two Patients in Pivotal Expansion of Phase 1/2 Clinical Trial in MPS IIIA
ABO-102 Patients Enrolled in Cohort 3 at 3E13 vg/kg Accelerated enrollments begin with first Patient Enrolled at clinical site in Australia; Patient Screening at Spain site underway Day 30 results demonstrate 67% Heparan sulfate reduction in the CNS and...
Abeona Therapeutics Announces Dedication of Commercial Gene Therapy Manufacturing Facility in Cleveland, Ohio
— Facility to support development of advanced gene and cell therapies for treatment of life-threatening rare diseases — Leaders from local government, life sciences and medical technology to join on October 4 for dedication of first anticipated gene...
–Strategic alignment with Brammer Bio for commercial AAV process development, scale up and assay validation –Phillip B. Maples, Ph.D., to lead Abeona manufacturing, as VP of Therapeutics Development and Quality Management CLEVELAND, Sept. 28, 2017 (GLOBE NEWSWIRE) — Abeona...