Downstream processing of oligonucleotides: meeting regulatory and commercial demands

Piotr Putaj obtained his PhD from the University of Lyon, France (Laboratory of Organometallic Chemistry LCOMS) in 2012. After two post-doctoral stays at the Charles University in Prague, Czechia and RWTH Aachen, Germany he transitioned to the biotech industry in 2017. He held various scientific and managerial positions at Future Synthesis (Poznan, Poland), MultiplexDX (Bratislava, Slovakia) and AIRNA Bio (Tübingen, Germany).

He is currently a Director of Oligonucleotide Chemistry at VICO Therapeutics in Leiden, the Netherlands. His research interests include optimization of potency and safety profile of oligonucleotides, new methodologies of bioconjugation and targeted delivery to tissues and organs.At VICO Therapeutics we are pioneering RNA-based science to target the root cause of rare genetic neurological diseases. Our lead molecule VO659 is the first clinical candidate directly targeting the CAG repeat expansion mutation that causes all polyglutamine diseases (e.g. Huntington disease).

Jared Jackson has worked in the oligonucleotide field since 2013. He attended Sonoma State University in Rohnert Park, CA, where he studied Biochemistry. His undergraduate research was conducted under Professor Jon M. Fukuto and involved performing organic synthesis centered around Nitric Oxide. 

In 2013, he began working at LGC Biosearch Technologies, where he performed Final QC and analytical method development using LC/MS and HPLC systems. He later moved into a process development role, focusing on synthesis method development as well as troubleshooting Cleavage and Deprotection protocols. 

After 8 years at LGC, he joined Ultragenyx Pharmaceuticals in Novato, CA, where he led internal synthesis, purification, and analytical development of all antisense oligonucleotides. He synthesized multiple modalities, including LNA gapmer, MOE steric blocking ASOs, and siRNA. Multiple conjugated modalities were also incorporated into these oligonucleotides to improve delivery. In addition to synthesis, he was responsible for purification of all oligonucleotides, using various RP and AX methods to achieve in vivo grade material. He also conducted analytical characterization of the materials he synthesized and purified using LC/MS methods.

Maria del Carme Pons Royo, PhD, is a postdoctoral researcher at the Massachusetts Institute of Technology (MIT), specializing in continuous and non-chromatographic purification strategies for biotherapeutics. She holds an MSc in Life Science & Technology from TU Delft and a PhD from the University of Natural Resources and Life Sciences, Vienna (BOKU). Her research focuses on the development and optimization of innovative bioprocesses, with a strong emphasis on integrated and continuous downstream processing.