A route of administration study of BBP-812, an AAV9-based gene therapy for the treatment of Canavan disease, in juvenile cynomolgus macaques

Scott DW, Rouse JL, Romero KB, Eclov R, Lewis TEW, Kapadia M, Mansfield G, and Beard CW
Aspa Therapeutics, BridgeBio, Raleigh, NC , USA

Aspa Therapeutics is pleased to share the news that a poster was presented on October 22, 2019 at the European Society of Gene and Cell Therapy meeting, describing an important development in gene therapy for Canavan disease.

Genetics expert and microbiologist, Dr. Guangping Gao, details the experiences that led to him to devoting his scientific career to tackling Canavan disease, and illuminates the successes
he and his collaborators have achieved in this area.

Your participation in this study will help us better understand Canavan disease and why patients with the same disease often have a quite different course. Such knowledge is a prerequisite for evaluating new experimental therapies and assessing the effectiveness of palliative measures. Be sure to sign and send the informed consent as well which you can download here as well.

Seemin S. Ahmed • Guangping Gao

Canavan Review Article

Canavan disease was first described in 1931, and this review traces the progress made in the past 8 decades to understand the disease by enumerating current hypotheses and ongoing palliative measures to alleviate patient symptoms in the context of the latest advances in the field.

A research summary on the Canavan disease.

A Research Project Summary (Feb 2012)

The progress report on the effectiveness of acetate supplementation on mice/rats.

A progress report on the successful rescue of early fatality of Canavan mice by single IV injections of novel gene therapeutics.

The final progress report, focusing on maximizing the efficiency of gene delivery to the CNS using the AAV9 capsid by
two aims.