Free List Of Top 10 Ex Vivo Gene Therapy Companies

The field of gene therapy is undergoing a revolution, with ex vivo gene therapy offering immense potential for treating a wide range of genetic disorders. In the United States, several visionary companies are at the forefront of this revolutionary technology, advancing the development of ex vivo gene therapies and paving the way for transformative medical treatments.

Introduction

Ex vivo gene therapy involves the modification of a patient’s cells outside the body to correct genetic defects, followed by reintroduction of these modified cells into the patient. This innovative approach holds great promise for treating a variety of genetic conditions, including rare diseases and certain types of cancer, by addressing the underlying genetic abnormalities.

As the demand for ex vivo gene therapy continues to grow, several companies in the US have emerged as leaders in this burgeoning field. These companies are spearheading groundbreaking research and development efforts to bring potentially life-changing therapies to individuals in need. Below is an overview of the top 10 ex vivo gene therapy companies in the US, highlighting their sector, year founded, annual revenue, and a brief description of their contributions to the field.

Bluebird Bio, Inc

– Sector: Biotechnology

– Year Founded: 1992

– Annual Revenue: Approximately $100 million

– Short Description: Bluebird Bio focuses on developing gene therapies for severe genetic diseases and cancer. With a strong focus on ex vivo gene therapy, the company’s innovative treatments aim to address genetic abnormalities at the cellular level.

– Website: [Bluebird Bio, Inc](https://www.bluebirdbio.com/)

Sangamo Therapeutics, Inc

– Sector: Biotechnology

– Year Founded: 1995

– Annual Revenue: Approximately $90 million

– Short Description: Sangamo Therapeutics is at the forefront of developing genomic medicines, including ex vivo gene therapies, to potentially cure genetic diseases. The company’s platform enables precise gene editing and gene regulation for therapeutic applications.

– Website: [Sangamo Therapeutics, Inc](https://www.sangamo.com/)

Novartis Gene Therapies

– Sector: Pharmaceutical

– Year Founded: 2018 (as an independent company)

– Annual Revenue: Approximately $2 billion

– Short Description: Novartis Gene Therapies, formerly known as AveXis, is dedicated to developing and commercializing novel gene therapies for rare and lifesaving genetic diseases. The company’s ex vivo gene therapy research aims to bring transformative treatments to patients with unmet medical needs.

– Website: [Novartis Gene Therapies](https://www.novartis.com/)

Orchard Therapeutics

– Sector: Biotechnology

– Year Founded: 2015

– Annual Revenue: Approximately $60 million

– Short Description: Orchard Therapeutics is committed to developing ex vivo gene therapies for rare genetic diseases, with a focus on inherited metabolic disorders and immune deficiencies. The company’s innovative approach involves genetically modifying a patient’s own cells to restore normal gene function.

– Website: [Orchard Therapeutics](https://orchard-tx.com/)

Regenxbio Inc

– Sector: Biotechnology

– Year Founded: 2008

– Annual Revenue: Approximately $30 million

– Short Description: Regenxbio Inc is a leading clinical-stage biotechnology company focused on developing ex vivo gene therapies using its proprietary V Technology Platform. The company’s research aims to address a wide range of genetic diseases by delivering functional genes into patients’ cells.

– Website: [Regenxbio Inc](https://www.regenxbio.com/)

Abeona Therapeutics Inc

– Sector: Biotechnology

– Year Founded: 1974

– Annual Revenue: Approximately $25 million

– Short Description: Abeona Therapeutics is dedicated to developing gene and cell therapies, including ex vivo gene therapies, for patients with life-threatening rare genetic diseases. The company’s innovative approach involves harnessing the potential of gene-corrected cell therapies to address unmet medical needs.

– Website: [Abeona Therapeutics Inc](https://www.abeonatherapeutics.com/)

Rocket Pharmaceuticals, Inc

– Sector: Biotechnology

– Year Founded: 2011

– Annual Revenue: Approximately $20 million

– Short Description: Rocket Pharmaceuticals is advancing the development of ex vivo gene therapies for rare childhood genetic disorders, with a focus on hematologic and neurologic conditions. The company’s innovative research aims to deliver potentially curative treatments to patients worldwide.

– Website: [Rocket Pharmaceuticals, Inc](https://www.rocketpharma.com/)

Precision Biosciences, Inc

– Sector: Biotechnology

– Year Founded: 2006

– Annual Revenue: Approximately $45 million

– Short Description: Precision Biosciences is leveraging its proprietary ARCUS genome editing platform to develop ex vivo gene therapies for a range of genetic disorders and oncology indications. The company’s innovative approach enables precise and efficient gene editing for therapeutic applications.

– Website: [Precision Biosciences, Inc](https://www.precisionbiosciences.com/)

Avrobio, Inc

– Sector: Biotechnology

– Year Founded: 2015

– Annual Revenue: Approximately $15 million

– Short Description: Avrobio, Inc is focused on developing ex vivo gene therapies for rare and ultra-rare genetic diseases, with a particular emphasis on lysosomal storage disorders. The company’s innovative approach involves genetically modifying a patient’s own stem cells to deliver therapeutic benefits.

– Website: [Avrobio, Inc](https://www.avrobio.com/)

Logicbio Therapeutics, Inc

– Sector: Biotechnology

– Year Founded: 2014

– Annual Revenue: Approximately $10 million

– Short Description: Logicbio Therapeutics is dedicated to developing genome editing technologies and ex vivo gene therapies to address rare genetic diseases. The company’s platform enables precise genetic modifications to potentially restore normal gene function in patients.

– Website: [Logicbio Therapeutics, Inc](https://www.logicbio.com/)

These top 10 ex vivo gene therapy companies in the US are driving innovation in the field of gene therapy, with a focus on developing transformative treatments for individuals affected by genetic diseases. As these companies continue to push the boundaries of medical science, their pioneering research and development efforts hold the promise of bringing much-needed relief to patients and families grappling with the impact of genetic disorders.