Pipeline and Publications

Each of our product candidates within our FSHD, SCD, COVID-19 and beta-thalassemia programs is a small-molecule therapeutic that aims to treat the root cause of a genetically defined rare disease. We plan to utilize our product engine to complete four new drug target identification screens in Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1), Friedreich’s ataxia (FA) and alpha-synucleinopathies. We expect to advance the most compelling programs identified for development.

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

July 1, 2020

The Global Phosphorylation Landscape of SARS-CoV-2 Infection

Mehdi Bouhaddou, Danish Memon, Bjoern Meyer, Kris M. White, Veronica V. Rezelj, Miguel C. Marrero, Benjamin J. Polacco, James E. Melnyk, Svenja Ulferts, Robyn M. Kaake, Jyoti Batra, Alicia L. Richards, Erica Stevenson, David E. Gordon, Ajda Rojc, Kirsten Obernier, Jacqueline M. Fabius, Margaret Soucheray, Lisa Miorin, Elena Moreno, Cassandra Koh, Quang Dinh Tran, Alexandra Hardy, Rémy Robinot, Thomas Vallet, Benjamin E. Nilsson-Payant, Claudia Hernandez-Armenta, Alistair Dunham, Sebastian Weigang, Julian Knerr, Maya Modak, Diego Quintero, Yuan Zhou, Aurelien Dugourd, Alberto Valdeolivas, Trupti Patil, Qiongyu Li, Ruth Hüttenhain, Merve Cakir, Monita Muralidharan, Minkyu Kim, Gwendolyn Jang, Beril Tutuncuoglu, Joseph Hiatt, Jeffery Z. Guo, Jiewei Xu, Sophia Bouhaddou, Christopher J.P. Mathy, Anna Gaulton, Emma J. Manners, Eloy Félix, Ying Shi, Marissa Goff, Jean K. Lim, Timothy McBride, Michael C. O’Neal, Yiming Cai, Jason C.J. Chang, David J. Broadhurst, Saker Klippsten, Emmie De wit, Andrew R. Leach, Tanja Kortemme, Brian Shoichet, Melanie Ott, Julio Saez-Rodriguez, Benjamin R. tenOever, Dyche Mullins, Elizabeth R. Fischer, Georg Kochs, Robert Grosse, Adolfo García-Sastre, Marco Vignuzzi, Jeffery R. Johnson, Kevan M. Shokat, Danielle L. Swaney, Pedro Beltrao, Nevan J. Krogan

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).

Fulcrum Publications

December 7, 2020

In vitro characterization of FTX-6058, a novel small molecule fetal hemoglobin inducer for sickle cell disease

Billy Stuart, Pete Rahl, Kingsley Appiah, Ivan Efremov, Lorin A Thompson, Owen Wallace, Christopher Moxham

December 7, 2020

FTX-6058, a novel HbF-inducing agent for the treatment of Sickle Cell Disease and b-Thalassemia

Billy Stuart, Keqiang Xie, Mark Roth, Steven Kazmirski, Kinglsey Appiah, Richard Barnes, Qingyi Li, David Peters, Lucienne Ronco, Kim Stickland, Paul Bruno, Diego Cadavid, Lorin Thompson, Owen Wallace, Ivan Efremov, and Peter Rahl

December 7, 2020

In vivo characterization of FTX-6058, a novel small molecule fetal hemoglobin inducer for sickle cell disease

Keqiang Xie, Mark Roth, Ivan Efremov, Serena Silver, Lucienne Ronco, Lorin A Thompson, Kim Stickland, Christopher Moxham and Owen Wallace