Biogen's antisense oligonucleotide (ASO) development and manufacturing has been, until recently, carried out at our partner Ionis Pharmaceuticals (e.g., Spinraza). In 2015, this new modality was established within Biogen’ Technical Development group to support the growing ASO portfolio. In 2016, a development lab was established in Cambridge MA for making ASOs at both lab (1/100th) and pilot scale (1/10th MFG scale) using our new platform process.<br/>In the fall of 2017, our first full scale GMP batches for the lead clinical development compound was manufactured in the newly built synthesis suite within the oral solid manufacturing facility in North Carolina. A four-step purification process was then carried out in the existing nearby flexible volume manufacturing facility that, up until this point, has been used for manufacturing Biogen’s protein-based parenterals. The ready-to-fill drug substance was then further processed into drug product vials. This was our first test case to demonstrate that Biogen can manufacture ASOs safely, at scale, and achieve high purity and yield. Results will be presented on the scalability of the ASO process from bench to GMP scale, highlighting successes and challenges faced with scale-up of the downstream ASO process.

Antisense oligonucleotide purification process: Successes and challenges during scale-up

255th National Meeting (2018)

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