Fulcrum Therapeutics is a member of SCDAA’s SCD C.A.R.E.S. Consortium.
We launched Fulcrum Therapeutics with a vision to develop small molecules to improve the lives of patients with genetically defined rare diseases in areas of high unmet medical need. Fulcrum uses proprietary technology to identify drug targets that can modulate gene expression to treat the known root cause of gene mis-expression.
At Fulcrum, we are scientific leaders focused on creating therapies that transform the lives of patients and caregivers. Our patient-centered culture empowers us to attentively address the needs of the rare disease community, guiding our scientific endeavors. This community is the foundation of everything we do and the reason we consistently move forward together every day, no exceptions.
Featured Clinical Trials
Fulcrum is developing pociredir (FTX-6058) as an oral therapeutic designed to induce expression of fetal hemoglobin (HbF) to compensate for the mutated adult hemoglobin in SCD, resulting in reduction or elimination of symptoms. Pociredir is currently being evaluated in a Phase 1b clinical trial for the potential treatment of SCD, which we refer to as the PIONEER study (NCT: NCT05169580). To learn more about the study, visit pioneerscdstudy.com.
- https://www.pioneerscdstudy.com/
- https://clinicaltrials.gov/study/NCT05169580?cond=Sickle%20Cell%20Disease&term=fulcrum&rank=1
Additional resources for warriors and the SCD community can be found here.
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The SCD C.A.R.E.S. Consortium aims to change the perception of clinical trials and increase the participation of sickle cell patients in clinical trials to ultimately provide more therapies to the SCD community. Together, the consortium works together to educate and encourage more trial participation while providing opportunities and better options for the treatment of sickle cell disease.
