Selecta hires Lonza to make synthetic vector delivered gene therapy

By Gareth Macdonald contact

- Last updated on GMT

Related tags: Gene therapy candidate, Gene

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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