The acquisition will add Regenxbio with two product candidates, including DTX301 and DTX401.
DTX301 is being developed for the treatment of ornithine transcarbamylase (OTC) deficiency. It will use the NAV AAV8 vector to deliver a copy of the OTC gene to liver cells.
Under an active investigational new drug (IND) application, DTX301 is being assessed for Phase I/II clinical trial.
DTX401 is being developed for the treatment of glycogen storage disease type Ia (GSDIa). It is designed to use the NAV AAV8 vector to deliver a copy of the glucose-6-phosphatase (G6Pase) gene to liver cells.
An IND application is expected to be submitted for DTX401 in early 2018. Both DTX301 and DTX401have secured orphan drug status in the US and Europe.
Regenxbio will also acquire DTX201 for the treatment of hemophilia A. It is designed to use the NAV Technology to deliver a copy of the Factor VIII gene to liver cells.
DTX201 is being developed under the global development and commercialization collaboration with Bayer. An IND application is expected to be submitted for DTX201 in early 2018.
In addition, Regenxbio will acquire preclinical product candidates for phenylketonuria (PKU), Wilson disease and citrullinemia type I, manufacturing technology and other intellectual property developed by Dimension.
Subject to customary regulatory approvals and other closing conditions, the deal is expected to complete by the end of this year.
Regenxbio president and CEO Kenneth Mills said: “This acquisition confirms Regenxbio’s leadership in the field of AAV gene therapy and expands our pipeline in metabolic diseases using NAV Technology with a clinical asset and several preclinical assets.
Dimension CEO Dr Annalisa Jenkins said: “Regenxbio has strong leadership, understanding of technology, capabilities, and financial resources that provide significant synergies with Dimension’s R&D portfolio.”
Image: Regenxbio to acquire Dimension Therapeutics. Photo: courtesy of adamr / FreeDigitalPhotos.net.