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Selecta hires Lonza to make synthetic vector delivered gene therapy

By Gareth MacDonald+

17-Feb-2017
Last updated on 20-Feb-2017 at 15:14 GMT2017-02-20T15:14:50Z

Lonza HQ in Switzerland

Lonza HQ in Switzerland

Selecta Biosciences Inc. has hired Swiss contractor Lonza to make a gene therapy candidate for the metabolic disease, Methylmalonic Acidemia (MMA).

The deal – financial terms of which were not disclosed – will see Lonza make the gene therapy at its recently established manufacturing facility in Houston, Texas.

Selecta’s MMA candidate is delivered using a synthetic adeno-associated viral (AAV) vector – called Anc80 – that the US firm licensed from  the Massachusetts Eye and Ear Infirmary and The Schepens Eye Research Institute, Inc – known as MEE - last May .

According to Selecta, the synthetic vector technology achieves higher gene expression levels in the liver than natural AAV vectors.

Lonza licensed rights to sell the vectors to gene therapy developers from MEE in September in an agreement which also saw it tasked with developing a commercial scale production process for Anc80.

At the time, Luk Vandenberghe from MEE told us Anc80 was developed as a result of efforts to “optimize AAVs for therapeutic gene transfer applications on clinically relevant parameters including host immunity, production yields, tissue targeting and specificity.”

Immune tolerance

In addition to utilizing the ANc80 delivery vector, Selecta’s MMA candidate also incorporates SVP‑Rapamycin, the company’s biodegradable nanoparticle encapsulating the immunomodulator rapamycin.

The SVP-Rapamycin technology is designed to minimize the development of anti-drug antibodies (ADA).

Selecta touted the potential benefits of the technology before it went public last year , suggesting that Crealta Pharmaceuticals’ gout drug Krystexxa (pegloticase) had failed to achieve broad commercial adoption partly as a result of the ADA it induced.

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