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

muscles

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

November 17, 2020

Motion sensor-acquired reachable workspace correlates with patient-reported upper extremity activities of daily living (ADL) function in facioscapulohumeral dystrophy

Maya N. Hatch, Gregorij Kurillo, Vicky Chan, Jay J. Han. Muscle & Nerve (2020)

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

September 23, 2021

Revolutionizing drug discovery in genetically-defined muscle disease using single-cell and high-dimensional datasets

L. Alejandro Rojas, Ayla Ergun, Caroline Sartain, Anthony Accorsi, Richard Lieberman, Anugraha Raman, Joe Maglio, Erin Valentine, Mary Wertz, Elizabeth Townsend, Damon Polioudakis, Ivan Efremov, Lorin A. Thompson, Serena J. Silver, Christopher M. Moxham

September 23, 2021

An Open-Label Study of Losmapimod to Evaluate the Safety, Tolerability, and Biomarker and Clinical Outcome Assessment Changes in Subjects with FSHD1

Joost Kools, Nicol Voermans, KarlienMul, Lucienne Ronco, John Jiang, Jennifer Shoskes, Kelly Marshall, Diego Cadavid, Michelle L. Mellion, Baziel van Engelen

September 23, 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