What Fulcrum Therapeutics does

Fulcrum Therapeutics, Inc. is a clinical-stage biopharmaceutical company founded in 2016 and headquartered at 26 Landsdowne Street, Cambridge, Massachusetts. The company develops small molecule therapeutics designed to modulate gene expression and treat the root cause of genetically defined rare diseases. Its lead clinical candidate was pociredir (FTX-6058), an oral EED inhibitor intended to induce fetal hemoglobin (HbF) expression for sickle cell disease (SCD); secondary programs included losmapimod, a p38α/β MAPK inhibitor for facioscapulohumeral muscular dystrophy (FSHD), a preclinical EED inhibitor FTX-6274 for castration-resistant prostate cancer, and discovery-stage programs in beta-thalassemia and Bone Marrow Failure Syndromes including Diamond Blackfan Anemia.

The company's discovery platform is built on three proprietary components: the FulcrumSeek database for computational target identification, chemogenomic and CRISPR-Cas9 screening methodologies, and patient-derived cell models from genetically defined disease populations. This platform produced five candidates across hematology, neuromuscular, and oncology indications, and attracted scientific founders from Harvard/MGH, MIT, and NYU, including Bradley Bernstein, Michael Green, Rudolf Jaenisch, Jeannie T. Lee, and Danny Reinberg. Fulcrum is publicly listed on NASDAQ under ticker FULC and has raised capital through multiple public stock offerings totaling approximately $494.2 million across 2021–2025.

Fulcrum's business model is biopharmaceutical R&D: it is pre-revenue with no approved products and no commercial pricing. Revenue is intended to come from product sales upon regulatory approval of approved candidates, with current cash reserves of approximately $333.3 million as of Q1 2026 providing stated runway into 2029. The company's June 2026 announcement that FDA had flagged malignancy risk for any PRC2-targeting intervention triggered discontinuation of the pociredir program and a formal strategic review exploring mergers, acquisitions, or other transactions; the company subsequently reduced its workforce from 57 to 9 employees and is now operating as a substantially diminished entity facing multiple securities law investigations related to the program discontinuation.

Name

Fulcrum Therapeutics

Legal name

Fulcrum Therapeutics, Inc.

Website

http://www.fulcrumtx.com

Company type

Public

Founded year

2016

Operating status

Operating

Headcount range

51–100 employees

Short description

Fulcrum Therapeutics is a clinical-stage biopharmaceutical company developing small molecule gene expression modulators to treat genetically defined rare diseases, including sickle cell disease, FSHD, beta-thalassemia, and bone marrow failure syndromes.

Ownership category

akta.pro rank

Product category

Rare Disease Biopharmaceuticals

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Services-Commercial Physical & Biological Research (8731), Medicinal Chemicals & Botanical Products (2833)

akta.pro primary industry

Target Identification & Validation Platforms (omics-driven, chemoproteomics, genetic validation) (HLAAAIAI)

akta.pro secondary industry

Blood Factors & Hemostasis Biologics (recombinant clotting factors, thrombolytics) (HLAAAAAL)

Keywords

• Rare disease therapeutics
• Small molecule drugs
• Gene expression modulation
• Sickle cell disease
• Clinical-stage biopharmaceutical

Headquarters

HQ city

Cambridge

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Marketing or Sales, Operations, Infrastructure

Revenue model

1. Drug Development and Commercialization: Pre-revenue clinical-stage biopharmaceutical company developing small molecule therapeutics for genetically defined rare diseases. Revenue will come from product sales upon regulatory approval. Current programs include pociredir for sickle cell disease, losmapimod for FSHD, and discovery programs for beta-thalassemia and other conditions.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels6 records

Core offering

Fulcrum Therapeutics is a clinical-stage biopharmaceutical company developing oral small molecule drugs that modulate gene expression to treat the root cause of genetically defined rare diseases. Its pipeline centers on pociredir (FTX-6058) for sickle cell disease and losmapimod for facioscapulohumeral muscular dystrophy (FSHD), with preclinical and discovery-stage programs in beta-thalassemia, bone marrow failure syndromes, and oncology. The company uses a proprietary product engine combining the FulcrumSeek database, patient-derived cell models, and chemogenomic/CRISPR-Cas9 screening to identify therapeutic targets.

Product overview

Fulcrum Therapeutics is a clinical-stage biopharmaceutical company developing small molecule therapeutics for genetically defined rare diseases. The company's lead product is pociredir (FTX-6058), an oral fetal hemoglobin inducer in Phase 1b trials for sickle cell disease. Additional clinical candidates include losmapimod for facioscapulohumeral muscular dystrophy (FSHD). The pipeline also includes preclinical and discovery-stage programs for beta-thalassemia, bone marrow failure syndromes (including Diamond Blackfan Anemia), and FTX-6274 for oncology. The company uses a proprietary product engine combining patient-derived cell models, CRISPR-Cas9 screening, and a proprietary database (FulcrumSeek) for drug target identification.

Differentiator

Problem solved

Functional benefit

Products and services

• Pociredir (FTX-6058) Oral small molecule EED inhibitor designed to induce fetal hemoglobin (HbF) expression to compensate for mutated adult hemoglobin in sickle cell disease patients; evaluated in the Phase 1b PIONEER clinical trial.
• Losmapimod p38α/β MAPK inhibitor being developed for facioscapulohumeral muscular dystrophy (FSHD), identified through Fulcrum's drug discovery platform to reduce aberrant DUX4 expression in skeletal muscle.
• FTX-6274 Preclinical EED (Embryonic Ectoderm Development) inhibitor with preclinical data suggesting potential for treating castration-resistant prostate cancer.
• Beta-thalassemia program Early-stage discovery program targeting beta-thalassemia, a rare genetic blood disorder characterized by chronic anemia and transfusion dependence.
• Bone Marrow Failure Syndromes program Discovery-stage program targeting bone marrow failure syndromes (BMFS) including Diamond Blackfan Anemia (DBA), a rare genetic disorder of impaired red blood cell production.

Quantifiable outcome

• 12.2% mean absolute HbF increase at Week 12 with 20mg pociredir dose
• +7 more outcomes

Named customers3 records

Segments4 records

Ideal customer profiles1 record

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Eight partnerships are on record, tiered minor and core.

• Cayenne Wellness CenterminorStrategic or Co-development Partner · 19 June 2026Co-host of the 7th Annual 24-Hour World Sickle Cell Day Marathon, a free virtual event focused on equity in sickle cell care. Fulcrum served as Community Sponsor, bringing together patients, caregivers, advocates, healthcare professionals, and researchers from multiple countries.
• Global Alliance of Sickle Cell Disease Organizations (GASCDO)minorStrategic or Co-development Partner · 19 June 2026Co-host of the 7th Annual 24-Hour World Sickle Cell Day Marathon alongside Cayenne Wellness Center. GASCDO is a global coalition working to improve outcomes for sickle cell disease patients.
• FSHD Industry CollaborativeminorStrategic or Co-development Partner · 1 January 2026Initiative by FSHD Society, SOLVE FSHD, and FSHD CTRN to improve clinical trials for facioscapulohumeral muscular dystrophy through data analysis, biomarker development, and collaborative efforts among industry and academic entities.
• LifeSci Advisors, LLCcoreGTM or Marketing PartnerInvestor and media relations contact: Kevin Gardner ([email protected], 617-283-2856). Provides IR and communications support for investor engagement and media inquiries.
• Kaplan Fox & Kilsheimer LLPminorOthersLaw firm investigating Fulcrum Therapeutics for potential securities law violations following pociredir program discontinuation and stock price decline.
• Levi & KorsinskyminorOthersLaw firm investigating Fulcrum Therapeutics officers and directors for potential securities law violations related to prior public statements about Phase III timelines.
• Holzer & Holzer, LLCminorOthersLaw firm investigating Fulcrum Therapeutics for potential federal securities law violations following June 2026 pociredir discontinuation announcement.
• SueWallStminorOthersLaw firm investigating Fulcrum Therapeutics for possible securities law violations regarding insider trading disclosures and financial commitments.

Scale indicators11 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• Beam Therapeutics: Developing base-editing therapies for sickle cell disease (BEAM-101) and beta-thalassemia. Direct peer in next-generation SCD therapeutics with similar genetically-defined disease focus.
• Global Blood Therapeutics: Developed voxelotor (Oxbryta), an oral small molecule for sickle cell disease, until acquisition by Pfizer in 2022. Direct comparable in SCD small-molecule modality and rare hematology focus.
• Disc Medicine: Clinical-stage rare hematology company developing bitopertin for erythropoietic protoporphyria and DISC-0974 for anemia. Comparable rare blood disorder focus and small-molecule strategy.
• Acceleron Pharma: Developed luspatercept (Reblozyl) for beta-thalassemia and MDS, acquired by Merck in 2021. Direct overlap with Fulcrum's hematology rare-disease pipeline and gene-expression-modulating therapeutics.
• bluebird bio: Developed gene therapies for sickle cell disease (Lyfgenia) and beta-thalassemia (Zynteglo). Direct peer in addressing SCD and beta-thalassemia through disease-modifying approaches.
• CRISPR Therapeutics: Co-developed Casgevy, the first FDA-approved CRISPR gene-editing therapy for sickle cell disease with Vertex. Direct competitor pursuing disease-modifying SCD treatment.
• Dyne Therapeutics: Developing antisense oligonucleotide therapies for FSHD (DYNE-101) and other rare muscle diseases. Closest direct competitor in FSHD therapeutic development.

Broad incumbents

• Vertex Pharmaceuticals: Co-developed Casgevy (exa-cel) for sickle cell disease with CRISPR Therapeutics and has commercial-stage rare disease infrastructure. Broad incumbent in SCD therapeutics with potential M&A interest in FSHD assets.
• Ionis Pharmaceuticals: Established antisense oligonucleotide leader with rare-disease franchises across hematology, neurology, and metabolic disorders. Broader incumbent in rare disease antisense therapeutics.

Emerging players

• Avidity Biosciences: Developing antibody-RNA conjugates for FSHD and other rare neuromuscular diseases. Emerging competitor in the FSHD space overlapping with Fulcrum's losmapimod program.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights6 records

Customer concentration

• Xx.com/fulcrumtx
• Facebookfacebook.com/FulcrumTx
• LinkedInlinkedin.com/company/fulcrum-therapeutics
• Instagraminstagram.com/fulcrumtx

Revenue estimate

Valuation estimate

Number of profiles

Profiles10 records

Funding overview

Funding rounds9 records

Investors16 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Fulcrum Therapeutics