Pipeline and Publications

Our goal: To employ our proprietary product engine to systematically identify and validate cellular drug targets that can modulate gene expression to treat the known root cause of genetically defined diseases.


Broad potential to change lives

Our approach to drug discovery and development is applicable across a large number of genetically defined diseases. We have developed a rigorous assessment and selection process to determine which of the approximately 7,000 rare, genetically defined diseases we intend to evaluate in drug target identification activities. We are applying our product engine to discover drug targets to modulate gene expression and develop product candidates for the potential treatment of the root cause of these diseases.

Muscle Disorders


CNS Disorders

central nervous system

Blood Disorders

red blood cells

We create value through our pipeline

By staying true to our passion and always prioritizing patients first, we plan to progress our programs and create significant value for all of our stakeholders.


Supporting Literature

February 1, 2019

MRI-informed muscle biopsies correlate MRI with pathology and DUX4 target gene expression in FSHD

Leo H Wang, Seth D Friedman, Dennis Shaw, Lauren Snider, Chao-Jen Wong, Chris B Budech Sandra L Poliachik, Nancy E Gove, Leann M Lewis, Amy E Campbell, Richard J F L Lemmers, Silvère M Maarel, Stephen J Tapscott, Rabi N Tawil. Human Molecular Genetics (2019).

August 1, 2018

Facioscapulohumeral dystrophy: activating an early embryonic transcriptional program in human skeletal muscle

Amy E. Campbell, Andrea E. Belleville, Rebecca Resnick, Sean C. Shadle, Stephen J. Tapscott. Human Molecular Genetics (2018).

November 14, 2017

Adding quantitative muscle MRI to the FSHD clinical trial toolbox

Karlien Mul, Sanne C.C. Vincenten, Nicol C. Voermans, Richard J.L.F. Lemmers, Patrick J. van der Vliet, Silvère M. van der Maarel, George W. Padberg, Corinne G.C. Horlings, and Baziel G.M. van Engelen. Neurology (2017).

Fulcrum Publications

April 9, 2021

Discovery of Clinical Candidate FTX 6058: A Potent, Orally Bioavailable Upregulator of Fetal Hemoglobin for Treatment of Sickle Cell Disease

Ivan V. Efremov, Kingsley Kofi Appiah, Angela Cacace, Yanfei Dong, Shawn D. Johnstone, Steven Kazmirski, Qingyi Li, Christopher Moxham, Peter Rahl, Mark Roth, Billy Stuart, Lorin A. Thompson, III, Owen B. Wallace, Keqiang Xie, Feng Zhou

March 18, 2021

Quantitative Muscle Analysis in FSHD Using Whole Body MRI: Composite Muscle Measurements for Cross Sectional Analysis

Per Widholm, Markus Karlsson, André Ahlgren, Olof Dahlqvist Leinhard, Rabi Tawil, Kathryn Wagner, Jeffrey Statland, Leo Wang, Perry Shieh, Baziel Van Engelen, Diego Cadavid, Lucienne Ronco, Adefowope Odueyungbo, Jay Han, Maya Hatch, Michelle L. Mellion

March 18, 2021

In-Home Passive Measurements of Mobility and Sleep in FSHD Patients and their Relationship with Clinical Disease Severity Metrics

Rumen Hristov, Hariharan Rahul, Zachary Kabelac, Vicky Chan, Maya Hatch, Jay Han, Diego Cadavid, Michelle Mellion, Dina Katabi