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.


Fulcrum rare disease pipeline 200727

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

October 1, 2020

Development and Evaluation of a Whole-body MRI Protocol and Analysis Algorithms to Measure Changes in Skeletal Muscle in FSHD

Diego Cadavid, Michelle Mellion, Shane Raines, Per Widholm, and Olof Dahlqvist Leinhard

October 1, 2020

A Biomarker of Aberrant DUX4 Activity to Evaluate Losmapimod Treatment Effect in FSHD Phase 2 Trials

Lucienne Ronco, Kathryn Wagner, Diego Cadavid, Jayashree Chadchankar, Aaron Chang, Michelle Mellion, Adefowope Odueyungbo, Shane Raines, Anugraha Raman, Alan Robertson, Alejandro Rojas, Ning Shen, Perry Shieh, Jeffrey Statland, Rabi Tawil, Stephen Tapscott, Baziel van Engelen, Leo Wang, Nick Johnson, Owen Wallace

October 1, 2020

Open-Label Pilot Study of Losmapimod in FSHD1 (NCT04004000)

Joost Kools, Nicol Voermans, Karlien Mul, Lucienne Ronco, Adefowope Odueyungbo, Venkateswarlu Kondragunta, Kelly Marshall, Mary Bylo, Michelle Mellion, Baziel van Engelen, Diego Cadavid