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 mRNA AND PRE-mRNA WITH ALL MODALITIES

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

OPTIMIZING SMALL MOLECULES FOR RNA TARGETS

 

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

 

nEW mODALITES

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

University

 

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:

Mana Chandhok

Conference Producer

Cambridge Healthtech Institute

Phone: (+1) 781-972-1350

Email: mchandhok@healthtech.com

 

For partnering and sponsorship information, please contact:

Rod Eymael

Manager, Business Development

Cambridge Healthtech Institute

Phone: (+1) 781-247-6286

Email: reymael@healthtech.com