The cardiovascular disease gene therapy industry is an emerging sector in biomedical science, dedicated to the use of genetic techniques in curing cardiac issues. Companies in this field leverage specialized methods, like gene editing and viral vector therapies, to treat genetically defined diseases. These firms are frequently biotech units or pharmaceutical companies focused on innovative treatment approaches. There is a growing trend towards customized, targeted treatments, heralding a new era in health care. The future of this industry hints at a shift from conventional treatment to genetic therapies, promising improvements in patient outcomes and treatment efficiencies.


Top 27 Cardiovascular Disease Gene Therapy Companies

1. 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.


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. 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.


5. 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.


6. LEXEO Therapeutics

  • Website: lexeotx.com
  • Headquarters: New York, New York, United States
  • Founded: 2018
  • Headcount: 11-50
  • Latest funding type: Series B
  • LinkedIn

LEXEO is a gene therapy company that develops investigational therapies for rare monogenic diseases and diseases affecting prevalent patient populations. They utilize engineered adeno-associated viruses to transfer therapeutic genes to patients, targeting genetically defined cardiovascular and central nervous system diseases. LEXEO's therapies have potential in treating Friedreich's Ataxia, Alzheimer's, and other similar conditions.


7. Renova Therapeutics

Renova Therapeutics develops gene therapies to treat chronic conditions like congestive heart failure and Type 2 diabetes.


8. Nuevocor

  • Website: nuevocor.com
  • Headquarters: Singapore, Central Region, Singapore
  • Founded: 2020
  • Headcount: 1-10
  • Latest funding type: Series Unknown
  • LinkedIn

Nuevocor is a new biotech start-up in Singapore striving to develop gene therapies for genetic cardiomyopathies. It will leverage a deep understanding of cardiac cell biology, as well as a technology platform encompassing novel gene therapy vectors and proteomics-based target discovery


9. 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.


10. Rocket Pharmaceuticals

  • Website: rocketpharma.com
  • Headquarters: Cranbury, New Jersey, United States
  • Founded: 2015
  • Headcount: 51-200
  • Latest funding type: Post Ipo Equity
  • LinkedIn

Rocket Pharmaceuticals is a biotech company that specializes in developing gene therapies for rare and complex inherited childhood disorders. Their mission is to provide curative treatments for patients with devastating diseases through first-in-class and best-in-class therapies. They prioritize patient support and community building throughout the treatment process.


11. MedCity

MedCity HQ is a company that provides innovative solutions for the healthcare industry.


12. Ysios Capital

  • Website: ysioscapital.com
  • Headquarters: San Sebastián, País Vasco / Euskadi, Spain
  • Founded: 2008
  • Headcount: 11-50
  • LinkedIn

Ysios Capital is a leading life sciences investor financing highly innovative companies worldwide.


13. Neural Stem Cell Institute

  • Website: neuralsci.org
  • Headquarters: Rensselaer, New York, United States
  • Founded: 2007
  • Headcount: 11-50
  • LinkedIn

Neuralsci.org is a company focused on neurological research and cancer treatment. They specialize in the study of neural stem cells and the development of innovative therapies for various types of cancer, including lung cancer, thyroid cancer, and neuroblastoma.


14. Help Therapeutics

  • Website: helpsci.com.cn
  • Headquarters: Nanjing, Jiangsu, China
  • Founded: 2016
  • Headcount: 51-200
  • Latest funding type: Series C
  • LinkedIn

南京艾尔普再生医学科技有限公司 is a leader in the field of pluripotent stem cell technology in China, with a focus on developing stem cell therapy applications for better health. The company accelerates the development of stem cell industry and products by building a large biological sample information database, adhering to national and international standards to promote healthy development for human beings.


15. Hibiscus BioVentures

Hibiscus BioVentures is a venture studio that focuses on building patient-focused companies around innovative biotechnologies. They provide the technology, capital, and expertise needed to create transformative biotechnology companies.


16. Sofinnova Investments

  • Website: sofinnova.com
  • Headquarters: Menlo Park, California, United States
  • Founded: 1974
  • Headcount: 11-50
  • LinkedIn

Sofinnova is an investment banking firm that offers a wide range of financial services to its clients. They provide expertise and guidance in mergers and acquisitions, capital raising, and strategic advisory services. With a focus on the healthcare and life sciences industries, Sofinnova helps companies navigate complex financial transactions and achieve their growth and investment objectives.


17. Clora

  • Website: clora.com
  • Headquarters: Boston, Massachusetts, United States
  • Founded: 2016
  • Headcount: 11-50
  • Latest funding type: Pre Seed
  • LinkedIn

Clora is an intelligent platform that efficiently matches life science companies with flexible, on-demand expertise. We provide a more effective and efficient means to engage with the right talent when you need it, from drug discovery to commercialization. Our consultant network covers 300+ skillsets required during the product lifecycle, including Clinical Project Managers, Medical Writers, QA Auditors, Regulatory Experts, Biostatisticians, Data Managers, Toxicologists, and more.


18. SmartPharm Therapeutics, Inc.

  • Website: smartpharmtx.com
  • Headquarters: Cambridge, Massachusetts, United States
  • Founded: 2017
  • Headcount: 11-50
  • Latest funding type: Acquired
  • LinkedIn

SmartPharm Therapeutics is a biotech company offering gene-encoded therapeutics (GET) for a broad range of therapeutic opportunities.


19. Vault Bioventures

  • Website: vaultbio.com
  • Headquarters: San Diego, California, United States
  • Founded: 2011
  • Headcount: 11-50
  • LinkedIn

Vault Bioventures is a San Diego-based biopharmaceutical consultancy offering commercialization, business development, and product development services. Our team of executive biopharma experts maximizes your assets' and portfolios' value by interlocking market needs with clinical development.


20. AavantiBio, Inc.

  • Website: aavantibio.com
  • Headquarters: Cambridge, Massachusetts, United States
  • Founded: 2020
  • Headcount: 51-200
  • Latest funding type: Acquired
  • LinkedIn

AavantiBio is a genetic therapy company focused on developing innovative treatments for serious genetic disorders. Through groundbreaking research and strategic partnerships, they aim to provide life-changing medicines to patients in need.


21. Renovacor

  • Website: renovacor.com
  • Headquarters: Philadelphia, Pennsylvania, United States
  • Founded: 2019
  • Headcount: 11-50
  • Latest funding type: Acquired
  • LinkedIn

Renovacor is a company that aims to deliver innovative precision therapies to improve the lives of patients and families battling genetically-driven cardiovascular disease and mechanistically-related diseases.


22. Viralgen Vector Core

Viralgen is a company specializing in AAV manufacturing, development, and validation. With extensive expertise in AAV and its regulations, they offer comprehensive services throughout the entire process. Their operations include in-house QC lab for critical release assays, regulatory support, and program management. The company's customers could include pharmaceutical companies, biotech startups, and research institutions. Viralgen can be categorized as a biotech manufacturing company. Their offering keywords include AAV manufacturing, development, validation, and QC lab. Their business model keywords include Manufacturing, Consulting, and Biotech. Relevant categories for the industry could be Gene Therapy, Pharmaceutical, and Biotechnology. The company's customers likely include pharmaceutical companies, biotech startups, and research institutions. Other relevant keywords could be Gene Therapy, Cell-based Therapy, and Genetic Diseases.


23. Forge Biologics

  • Website: forgebiologics.com
  • Headquarters: Columbus, Ohio, United States
  • Founded: 2020
  • Headcount: 51-200
  • Latest funding type: Series C
  • LinkedIn

Forge Biologics is a company focused on developing innovative gene therapies to treat patients with devastating rare diseases. They offer end-to-end manufacturing services, from preclinical to commercial, to accelerate gene therapy programs. Their team of diverse and passionate individuals trailblazes a new model for making medicines. They specialize in AAV gene therapies and operate a state-of-the-art GMP facility called 'the Hearth.'


24. Avrobio

  • Website: avrobio.com
  • Headquarters: Cambridge, Massachusetts, United States
  • Founded: 2015
  • Headcount: 51-200
  • Latest funding type: Ipo
  • LinkedIn

AVROBIO is a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease. They are focused on targeting lysosomal disorders and developing personalized therapies for patients. Their goal is to enable individuals with genetic diseases to live longer and free from disease progression.


25. Jaguar Gene Therapy

  • Website: jaguargenetherapy.com
  • Headquarters: Lake Forest, Illinois, United States
  • Founded: 2019
  • Headcount: 51-200
  • Latest funding type: Series Unknown
  • LinkedIn

Jaguar Gene Therapy is a company that specializes in gene therapy and aims to accelerate breakthroughs in treating severe genetic diseases. They have a team of experts who develop and manufacture gene therapies, leveraging their expertise and cutting-edge technologies to deliver safe and effective treatments. Their mission is to provide hope and improved quality of life for patients and their families.


26. Mustang Bio

  • Website: mustangbio.com
  • Headquarters: Worcester, Massachusetts, United States
  • Founded: 2015
  • Headcount: 51-200
  • Latest funding type: Post Ipo Debt
  • LinkedIn

Mustang Bio is a clinical-stage biopharmaceutical company focused on translating medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors, and rare genetic diseases. They aim to acquire rights to these technologies, fund research and development, and bring the therapies to market. Mustang has collaborations with top medical institutions for developing CAR T therapies and lentiviral gene therapy. They are registered under the Securities Exchange Act of 1934 and founded by Fortress Biotech.


27. Stoke Therapeutics

  • Website: stoketherapeutics.com
  • Headquarters: Bedford, Massachusetts, United States
  • Founded: 2017
  • Headcount: 51-200
  • Latest funding type: Post Ipo Equity
  • LinkedIn

Stoke Therapeutics is a biotechnology company that is focused on addressing severe diseases by upregulating protein expression with RNA-based medicines. Their goal is to develop the first disease-modifying therapy for Dravet syndrome, a severe and progressive genetic epilepsy. With their proprietary research platform called TANGO, Stoke aims to restore missing proteins by increasing protein output from healthy genes.


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