Rare Daily Staff

Selecta Biosciences and Asklepios BioPharmaceutical have entered into a strategic partnership advance next-generation adeno-associated virus (AAV) gene therapies for patients with rare genetic diseases.

The partnership will leverage the proprietary technology platforms of both companies with a human proof-of-concept trial to validate the potential for re-dosing in patients with genetic diseases.

Currently the ability to re-administer systemic AAV gene therapy is limited by the development of neutralizing antibodies. Selecta, however, has shown preclinical evidence to support the potential for re-dosing patients receiving gene therapy.  When used in combination with AAV gene therapy vectors, Selecta’s ImmTOR inhibits the immune response to the vector.

The ability to safely re-dose AAV should help achieve therapeutic benefit in patients who are under-dosed. It should also help restore transgene expression in patients, particularly growing pediatric patients, who may lose expression over time. In addition, the companies said integrating ImmTOR into a gene therapy protocol provides a first dose benefit by enhancing liver-directed transgene expression in preclinical models.

“We expect that Selecta’s ImmTOR technology, in combination with AskBio’s AAV technology and clinical leadership, will allow us to rapidly advance a portfolio of new combination therapies through proof of concept and into the clinic,” said Carsten Brunn, CEO of Selecta.

AskBio was one of the first gene therapy companies and has been an innovator in the field. It holds more than 500 patents and applications for AAV technology and processes. Its platform was used to develop the only the FDA-approved gene therapies Zolgensma and Luxterna.

Photo: Carsten Brunn, CEO of Selecta

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