Sanfilippo syndrome gene therapy could be filed in US next year, Abeona

By Dan Stanton contact

- Last updated on GMT

Image: iStock/ktsimage
Image: iStock/ktsimage
Abeona Therapeutics says it plans to file its single injection adeno-associated virus (AAV) gene therapy for Sanfilippo syndrome in the US in late 2018.

There is no available treatment for the rare autosomal recessive lysosomal storage disease Sanfilippo syndrome.

But while pharma firms such as Shire, Alexion and BioMarin are investigating enzyme replacement therapies, Abeona says its gene therapy candidate ABO-102 could offer patients a cure delivered by a single injection.

In February, Abeona presented positive Phase I/II data for the candidate and during a call to discuss Q4 earnings yesterday CEO Timothy Miller said with last year’s fast-track US Food and Drug Administration (FDA) designation​, the firm is looking to potentially file the product in late 2018.

“We are already building on the [previously] granted rare disease Pediatric Designation and the Orphan Drug Designation in the United States,”​ he told stakeholders.

“As we look to engage the FDA – we do have some meetings coming up – we will be talking about what we believe are registrable endpoints and yes we do hope that this [will be] our registration study where we could potentially file this in the second half of 2018.”

ABO-102 is based on Abeona’s gene therapy platform comprising of DNA that encodes a therapeutic protein delivered by a genetically-modified adeno-associated virus (AAV).

According to the firm, the modified virus is capable of delivering therapeutic DNA across the blood brain barrier and into the central nervous system (CNS).

Abeona has three other gene therapies in the clinic, including candidates for juvenile batten disease (JNCL) and infantile batten disease (INCL).

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