Interview with Christiane Niederlaender, at AMBR Consulting released ahead of RNA Therapeutics Conference

SMi’s 11th annual RNA Therapeutics conference, taking place on 19th – 20th February in London, will bring together industry experts from leading RNA therapeutics companies to discuss the challenges for clinical translation of RNA-based therapeutics. The two-day event will present an overview of the applications of RNA-based drugs for modulation of gene and protein expression, and genome editing, with a focus on recent developments in delivery technologies.

SMi recently interviewed industry expert Christiane Niederlaender, Director at AMBR Consulting Ltd, who will be speaking at the RNA Therapeutics conference. Here is a snapshot of what was discussed:

The Oligonucleotide Therapeutics market has matured greatly over recent years, what key differences have you noticed in the last year regarding significant developments?

“The Oligonucleotide sector has seen an increase in regulatory approval, in particular, for the RNAi based therapies. My personal focus, however, is on RNAs that are biological medicines, and in this sector I see a lot of promise in the new mRNA-based vaccines, some of which are biologicals, where new developments mean that these products are now more stable, and have better translation. At the same time, CRISPR Cas approaches are now entering the clinic and the very first clinical results seem to show promise.”

What are your thoughts on the developing regulatory expectations around RNA Therapeutics and how is this currently impacting your role?
“From a regulatory perspective CRISPR Cas approaches are perhaps the most challenging. Due to the specifics of the legislation in Europe, these products can fall into a variety of medicine categories each with slightly differing regulatory implications. I think it will be important for regulators to come to an understanding of how to standardize their regulation bearing the risk profile in mind. Some of these considerations also apply to mRNA therapies.”

The full version of the interview and event brochure are available to download online at

Christiane Niederlaender will be presenting on “When RNA Therapeutics are Biological Medicines: Regulatory Considerations for the EU”:
• Classifications of RNA therapeutics within the medicines framework
• RNAs in CRISPR approaches and the CMC considerations
• Key regulatory issues as highlighted by case studies

RNA Therapeutics
Focus Day: 18th February 2020
Conference: 19th – 20th February 2020
Copthorne Tara Hotel, London, UK