FDA Grants Fast Track Designation to ABO-202 Gene Therapy for Infantile Batten Disease

Abeona Therapeutics’ one-time gene therapy candidate ABO-202 has been granted fast track designation by the U.S. Food and Drug Administration (FDA) for treating children with infantile Batten disease, also known as CLN1 disease.

A fast track designation by the FDA is intended to accelerate the review of new compounds that potentially could fill unmet medical needs.

The company is preparing to initiate a Phase 1/2 clinical trial to evaluate ABO-202 in children with CLN1 disease, with dates expected by the end of this year.

ABO-202 uses a modified viral vector called AAV9 to deliver a correct copy of the PPT1 gene. Mutations in this gene lead to a lack of the PPT1 enzyme — key for proper function of cellular structures called lysosomes. Lysosomes are the “trash compactors” of the cell, clearing waste and recycling the components. When the PPT1 enzyme is absent or not working properly, fatty residues known as lipofuscins accumulate inside cells, causing neuroinflammation and neurodegeneration.

This investigational gene therapy can be delivered either as an injection into the bloodstream (intravenous), an injection into the fluid surrounding the spinal cord (intrathecal injection), or both. Once the therapy is delivered, a healthy copy of the PPT1 gene can then produce enough PPT1 enzyme to keep the cell healthy.

In preclinical studies, a single dose of ABO-202 was effective to deliver a functional copy of PPT1 to cells throughout the body. In a mouse model of the disease, ABO-202 led to expanded lifespan and improved neurological function. These studies also demonstrated that using the two delivery routes at the same time enhanced the therapy’s effectiveness compared to either administration route alone.

“Receiving Fast Track designation acknowledges the urgency for developing a therapy for children suffering from this rapidly-progressing and fatal disease and highlights the significant potential of ABO-202 to address this unmet need,” João Siffert, MD, CEO at Abeona, said in a press release.

ABO-202 has been granted both orphan drug and rare pediatric disease designations by the FDA, and orphan medicinal product designation by the European Medicines Agency.

Orphan drug status is intended to encourage therapies for rare and serious diseases, through benefits such as seven years of market exclusivity and exemption from FDA application fees.