Skip to main content

Abeona Therapeutic’s ABO-102 get Fast Track Designation from FDA

 

Clinical courses

Abeona Therapeutics Inc. Announced that the U.S. Food and Drug Administration (FDA) granted Fast Track designation for ABO-102, a single intravenous injection of AAV gene therapy for subjects with MPS IIIA (Sanfilippo syndrome type A), a rare autosomal recessive disease affecting every cell and organ in the body causing neurocognitive decline, speech loss, loss of mobility, and premature death in children.

"Fast Track designation underscores the importance that the FDA places on developing new treatments for life-threatening disorders, such as MPS IIIA, and reinforces our mandate of accelerating the development of ABO-102 to market," stated Timothy J. Miller, Ph.D., President and CEO of Abeona Therapeutics. "We look forward to providing additional updates for our ongoing Phase 1/2 clinical trial."

"The Fast Track designation comes with an increase in interaction and feedback from the FDA during the development process of a drug and signifies that the FDA may be able to expedite the review and approval of the ABO-102 gene therapy product which, in preclinical and initial clinical work, has shown encouraging signals of biopotency," stated Steven H. Rouhandeh, Executive Chairman. "This designation also demonstrates to the children and families afflicted with MPS IIIA, the FDA's recognition of the severity and importance of addressing this rare orphan disease."

Per the design of the clinical trial, subjects in the low-dose cohort 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 were evaluated at multiple time points over the initial 30 days post-injection for safety assessments and initial signals of biopotency, which suggest that ABO-102 successfully reached target tissues throughout the body, including the central nervous system, to reduce GAG content that underlies the lysosomal storage pathology central to MPS IIIA (Sanfilippo syndrome type A).

ABO-102 is an adeno-associated viral (AAV)-based gene therapy for subjects with MPS IIIA (Sanfilippo syndrome), that is delivered as a one-time intravenous injection. ABO-102 delivers a functioning, corrective copy of the SGSH gene to cells of the central nervous system (CNS) and other organs with the goal of correcting the underlying deficits caused by the inborn genetic errors that are the cause of the disease.

<< Pharma News

Subscribe to PharmaTutor News Alerts by Email