The hematological disease gene therapy industry comprises biotechnological and pharmaceutical companies working to develop innovative treatments for blood disorders. These firms specialize in advanced genetic technologies, including gene therapy and CRISPR/Cas9 gene editing, to address severe genetic conditions such as sickle cell disease and β-thalassemia. With a patient-centered approach, these enterprises strive to transform lives through ground-breaking pharmaceutical research and development. Looking ahead, as genetic technologies continue to evolve, the industry is poised for significant growth and advancement, paving the way for more effective and personalized disease management solutions.


Top 26 Hematological Disease Gene Therapy Companies

1. bluebird bio

  • Website: bluebirdbio.com
  • Headquarters: Cambridge, Massachusetts, United States
  • Founded: 1993
  • Headcount: 1001-5000
  • Latest funding type: Post Ipo Equity
  • LinkedIn

Bluebird is a biotechnology company founded in 2010, specializing in ex-vivo gene therapy. With a focus on severe genetic diseases, they have industry-leading programs for sickle cell disease, β-thalassemia, and cerebral adrenoleukodystrophy.


2. Intellia Therapeutics, Inc.

  • Website: intelliatx.com
  • Headquarters: Cambridge, Massachusetts, United States
  • Founded: 2014
  • Headcount: 201-500
  • Latest funding type: Post Ipo Equity
  • LinkedIn

Intellia Therapeutics is a biotechnology company focused on developing CRISPR/Cas9 gene editing therapies. They are dedicated to transforming the lives of patients suffering from genetic diseases. Intellia Therapeutics is committed to using their expertise in genome editing to create innovative treatments.


3. Oxford Biomedica

  • Website: oxb.com
  • Headquarters: Oxford, Oxfordshire, United Kingdom
  • Founded: 1995
  • Headcount: 501-1000
  • Latest funding type: Post Ipo Equity
  • LinkedIn

Oxford Biomedica (oxb.com) is an innovative leading viral vector specialist - Lenti, AAV, Adeno and beyond - focused on delivering life changing therapies to patients. Their viral vector platform enables customers to bring next generation treatments for serious diseases to market.


4. REGENXBIO Inc.

  • Website: regenxbio.com
  • Headquarters: Rockville, Maryland, United States
  • Founded: 2008
  • Headcount: 201-500
  • Latest funding type: Post Ipo Equity
  • LinkedIn

REGENXBIO is a company dedicated to discovering and developing innovative gene therapies to address severe and debilitating diseases with unmet needs. They aim to improve treatment options and provide curative potential through their gene therapy solutions. With their NAV® Technology Platform, they are driving progress towards making gene therapy a reality for patients.


5. Sangamo Therapeutics, Inc.

  • Website: sangamo.com
  • Headquarters: Brisbane, California, United States
  • Founded: 1995
  • Headcount: 201-500
  • Latest funding type: Post Ipo Equity
  • LinkedIn

Sangamo is a biotechnology company that specializes in using zinc finger technology to develop genomic cures for severe diseases. Their expertise allows them to create treatments for patients who currently only have access to symptom management. They have a strong commitment to patient care and offer resources and support for both patients and healthcare professionals.


6. Precision BioSciences, Inc.

Precision BioSciences is a company dedicated to in vivo gene editing therapies. They specialize in permanently correcting genetic diseases through their ARCUS platform and CAR T immunotherapy. Precision BioSciences collaborates with healthcare companies and researchers to develop potentially curative treatments. They offer expertise in precision medicine and have a strong skill set within the genome editing industry.


7. Medpace

  • Website: medpace.com
  • Headquarters: Cincinnati, Ohio, United States
  • Founded: 1992
  • Headcount: 1001-5000
  • Latest funding type: Ipo
  • LinkedIn

Medpace is a full-service Contract Research Organization (CRO) that accelerates the advancement of safe and effective medical therapeutics. With a global reach, Medpace offers a wide range of services including clinical trial management, patient recruitment, and drug safety monitoring.


8. Spark Therapeutics, Inc.

  • Website: sparktx.com
  • Headquarters: Philadelphia, Pennsylvania, United States
  • Founded: 2013
  • Headcount: 501-1000
  • Latest funding type: Acquired
  • LinkedIn

Spark is a gene therapy company that strives to challenge the inevitability of genetic disease. They aim to discover, develop, and deliver potential treatments in ways unimaginable, combining their proprietary gene therapy platform with excellence in research and manufacturing.


9. BioMarin Pharmaceutical Inc.

  • Website: biomarin.com
  • Headquarters: San Rafael, California, United States
  • Founded: 1997
  • Headcount: 1001-5000
  • Latest funding type: Post Ipo Debt
  • LinkedIn

BioMarin is a pharmaceutical company specializing in genetic therapies. They develop and provide transformative treatments for various genetic diseases, such as Morquio A Syndrome, PKU, and MPS. With a focus on gene therapy research, BioMarin aims to maximize the impact of genetic innovation and improve patients' lives.


10. Novartis Nederland

  • Website: novartis.nl
  • Headquarters: Amsterdam, North Holland, Netherlands
  • Founded: 1996
  • Headcount: 10001+
  • LinkedIn

Novartis is a pharmaceutical company that focuses on developing and producing medications. With a mission to improve people's health and well-being, Novartis offers a range of medicines for various therapeutic areas. They are committed to clinical research and innovation, particularly in the fields of cell and gene therapy.


11. CRISPR Therapeutics

  • Website: crisprtx.com
  • Headquarters: Cambridge, Massachusetts, United States
  • Founded: 2013
  • Headcount: 201-500
  • Latest funding type: Grant
  • LinkedIn

CRISPR Therapeutics is a company that specializes in gene editing and cell therapies. They use the CRISPR/Cas9 therapeutic approach to treat genetically-defined diseases by editing cells inside the body.


12. Beam Therapeutics

  • Website: beamtx.com
  • Headquarters: Cambridge, Massachusetts, United States
  • Founded: 2017
  • Headcount: 501-1000
  • Latest funding type: Post Ipo Equity
  • LinkedIn

Beam Therapeutics is a cutting-edge biotech company focused on base editing applications, delivering clinically validated technologies for gene-based editing. They offer a suite of innovative, nonviral and viral delivery modalities to advance the field of genetic medicine.


13. Veristat

  • Website: veristat.com
  • Headquarters: Southborough, Massachusetts, United States
  • Founded: 1994
  • Headcount: 501-1000
  • Latest funding type: Private Equity
  • LinkedIn

Veristat is a scientific consulting and service provider company that specializes in biometrics, including data management, biostatistics, and statistical programming. They offer a wide range of services in the field of medical devices, rare diseases, cell and gene therapies, and clinical trials for various therapeutic areas. Veristat is known for its strategic approach, expertise in complex therapies, and ability to tackle unique scientific challenges.


14. Synteract

  • Website: synteract.com
  • Headquarters: Morrisville, North Carolina, United States
  • Founded: 1986
  • Headcount: 501-1000
  • Latest funding type: Acquired
  • LinkedIn

Synteract®, a Syneos Health® company, is a leading full service CRO focused on the emerging biopharma segment. The Company’s multidisciplinary teams support biotech and pharmaceutical companies across all phases of drug development, providing deep expertise in oncology, dermatology, general medicine, infectious disease and vaccines, neuroscience, pediatrics, and rare and orphan diseases. Synteract has conducted nearly 4,000 studies on six continents in over 62 countries.


15. Codexis, Inc.

  • Website: codexis.com
  • Headquarters: Redwood City, California, United States
  • Founded: 2002
  • Headcount: 201-500
  • Latest funding type: Post Ipo Equity
  • LinkedIn

Codexis is a company that focuses on discovering and developing life-changing treatments for patients. They tailor biotherapeutic performance using novel protein and gene therapies to address unmet medical needs and find treatments for a wide range of diseases and disorders.


16. Shanghai Medicilon Inc.

Medicilon is a company focused on accelerating R&D through higher efficiency and lower costs. They offer a range of services including synthetic chemistry, in vitro pharmacokinetic assays, enzymology & molecular interactions, and impurity traceability & control.


17. QPS Holdings, LLC

  • Website: qps.com
  • Headquarters: Newark, Delaware, United States
  • Founded: 1995
  • Headcount: 1001-5000
  • Latest funding type: Private Equity
  • LinkedIn

QPS is a provider of toxicology and drug development services, delivering high-quality data and comprehensive interpretation for Toxicology studies. They specialize in moving drugs from the lab to the clinic with well-executed Investigational New Drug (IND) packages.


18. Lovelace Biomedical Research Institute

Lovelace Biomedical is a not-for-profit preclinical contract research organization that offers unparalleled understanding of the biology of disease. With a wide array of services, our team of experts provides every step of the preclinical research process, from analytical chemists developing precise formulations and assays to veterinarians determining the best animal models. We are dedicated to serving humanity through research on the causes and cures of disease.


19. uniQure

  • Website: uniqure.com
  • Headquarters: Amsterdam, 1105Bp, Netherlands
  • Founded: 1998
  • Headcount: 201-500
  • Latest funding type: Post Ipo Equity
  • LinkedIn

uniQure is a global leader in gene therapy, specializing in delivering breakthrough treatments that transform patients' lives through the power of genomic medicine.


20. Editas Medicine

  • Website: editasmedicine.com
  • Headquarters: Cambridge, Massachusetts, United States
  • Founded: 2013
  • Headcount: 51-200
  • Latest funding type: Post Ipo Equity
  • LinkedIn

Editas Medicine is a biotechnology company specializing in CRISPR gene editing. They offer innovative solutions for patients and focus on developing cutting-edge therapies. With a strong research pipeline and a dedicated team, Editas Medicine aims to revolutionize the field of genetic medicine.


21. Imagine Institute of Genetic Diseases

Institut Imagine is a company that focuses on genomic research and provides AI solutions for genomics. They offer services and technologies that help in the analysis and interpretation of genomic data.


22. Discovery Life Sciences

  • Website: dls.com
  • Headquarters: Huntsville, Alabama, United States
  • Founded: 2018
  • Headcount: 501-1000
  • LinkedIn

Discovery Life Sciences is a globally recognized and trusted provider of biospecimen analysis and distribution services for the pharmaceutical, biotechnology, and diagnostics industries. With a focus on science and a client-centric approach, we offer customizable proteomic and genomic services, enabling comprehensive understanding of the proteome and accelerating drug and diagnostic development programs. Our expertise in tissue biomarker analysis, assay development, and clinical trial testing ensures accurate classification of biomarkers for successful drug and companion diagnostic development. We are committed to advancing science and providing the highest quality research-quality samples.


23. Genetic Engineering & Biotechnology News

  • Website: genengnews.com
  • Headquarters: New Rochelle, New York, United States
  • Founded: 1981
  • Headcount: 51-200
  • LinkedIn

GEN is a leading source of information in the biotechnology industry. They provide in-depth reporting, analysis, and multimedia content that captivates and informs a large audience in the life sciences. With their brand dominance and deep audience engagement, GEN helps companies achieve their marketing goals faster.


24. Orchard Therapeutics

  • Website: orchard-tx.com
  • Headquarters: London, England, United Kingdom
  • Founded: 2015
  • Headcount: 201-500
  • Latest funding type: Post Ipo Equity
  • LinkedIn

Orchard Therapeutics is a healthcare company specializing in genetic and severe disease treatments. They focus on discovering, developing, and commercializing new treatments using hematopoietic stem cell (HSC) gene therapy. By genetically modifying a patient's own blood stem cells, they aim to correct the underlying cause of disease in a single treatment.


25. Astellas Gene Therapies

Astellas Gene Therapies is a company dedicated to developing transformative genetic medicines for patients suffering from life-threatening diseases. With a focus on rare diseases, their innovative therapies target specific genetic mutations through gene replacement, exon skipping gene therapy, and vectorized RNA knockdown. Leveraging Astellas' global resources and expertise in immune biology, they aim to expand their reach and develop valuable new genetic medicines. Join their team for exciting job opportunities.


26. IsoPlexis

  • Website: isoplexis.com
  • Headquarters: Branford, Connecticut, United States
  • Founded: 2013
  • Headcount: 201-500
  • Latest funding type: Acquired
  • LinkedIn

IsoPlexis is a company that offers functional proteomics solutions for various research fields. Their innovative technologies enable the analysis of cellular and regenerative medicine, infectious diseases and vaccines, cancer immunology, inflammation and neurology. They provide cutting-edge instrumentation, software, and services to accelerate the understanding of immune response mechanisms and the development of potent therapies. With a focus on single-cell analysis, IsoPlexis aids researchers in uncovering new insights and advancing precision medicine.


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