Cambridge Healthtech Institute’s 2nd Annual

RNA as a Drug Target

Expanding the Boundaries of Druggable Targets

September 16-17, 2020


In the past year, the idea of targeting RNA directly with either a small molecule-like drug or with oligonucleotides has gone from an interesting idea to a viable business plan. It is expected that the FDA plans to make a decision on the first oral small molecule targeting splicing. Join us at the 2nd Annual RNA as a Drug Target conference, part of Discovery on Target, as we discuss not only the identification of specific and potent novel binders of RNA, but also the unique challenges that come with a novel drug target class.

Preliminary Agenda


Targeting Structurally and Functionally Diverse RNAs with Drug-Like Small Molecules

Jay Schneekloth, PhD, Senior Investigator, Chemical Biology Laboratory; Head, Chemical Genetics Section, Center for Cancer Research, National Cancer Institute, NIH

Targeting Pre-mRNA Splicing with Small Molecules

Marla Weetall, PhD, Vice President, Pharmacology, PTC Therapeutics

Translation Control Therapeutics

Iris Alroy, VP R&D, Anima Biotech, Inc.

TANGO (Targeted Augmentation of Nuclear Gene Output) for the Treatment of Genetic Diseases

Isabel Aznarez, PhD, Co-Founder & Vice President, Head of Biology, Stoke Therapeutics


PANEL DISCUSSION: What Can Each Modality Learn from One Another?

Moderator: Iris Alroy, VP R&D, Anima Biotech, Inc.
Panelists: Speakers of the Day


FEATURED PRESENTATION: Drugging RNA with Small Molecules — A Drug Discovery Perspective

Jennifer Petter, PhD, Founder & CSO, Arrakis Therapeutics


Discovery of Risdiplam, a Selective Survival of Motor Neuron-2 (SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA)

Hasane Ratni, PhD, Expert Scientist, Medicinal Chemistry, F. Hoffmann-La Roche, Basel, Switzerland

Drugging RNA

Natalie Dales, PhD, Director, Global Discovery Chemistry, Novartis



Development of A Novel miRNA-Based Platform Technology for Cancer

Jingfang Ju, PhD, Professor, Program Director of Oncogenic Drivers and Mechanisms of Carcinogenesis, Stony Brook



Oligonucleotide Therapeutics Now on Target: Advances in Antibody Oligonucleotide Conjugates (AOCs)

Arthur A. Levin, PhD, Executive Vice President, R&D, Avidity Biosciences

For more details on the conference, please contact:

Tanuja Koppal, PhD

Senior Conference Director
Cambridge Healthtech Institute


For partnering and sponsorship information, please contact:

Rod Eymael

Manager, Business Development

Cambridge Healthtech Institute

Phone: (+1) 781-247-6286