[Ranking] TOP 25 Gene Therapy / Biotechnology Companies Q3/2023
Intro
The field of gene therapy, a sub-industry of biotechnology, plays a vital role in the worldwide economy. Through the manipulation and modification of genetic material, gene therapy offers the potential to treat and cure a wide range of genetic and acquired diseases. This innovative approach has the power to revolutionize healthcare and improve the lives of millions of people globally.
The profound influence of gene therapy extends far beyond the realm of medicine. By addressing genetic disorders at their root cause, this industry has the potential to transform various sectors, including agriculture, animal health, and environmental conservation. It opens up new possibilities for enhancing crop yield, developing disease-resistant livestock, and preserving endangered species.
The economic magnitude of the gene therapy industry is on an upward trajectory. With a compound annual growth rate projected to exceed 30% in the coming years, the market is expanding rapidly. This growth is fueled by advancements in gene editing technologies, increased investment in research and development, and the growing demand for personalized medicine.
Recent trends in gene therapy have showcased remarkable breakthroughs, but also presented significant obstacles. The approval of Luxturna and Zolgensma as the first gene therapies in the United States marked a major milestone. However, challenges such as high treatment costs, manufacturing complexities, and the need for long-term safety and efficacy data remain areas of concern.
The landscape of the gene therapy industry is constantly evolving, setting the stage for exploration of its leading companies. As new players enter the market and existing ones refine their approaches, competition intensifies. Understanding the top companies in this dynamic field is crucial for investors, healthcare professionals, and individuals seeking the latest advancements in genetic medicine.
Ranking
#25 Precision BioSciences, Inc
Employees: 0.1K
Revenue: N/A
Website: https://precisionbiosciences.com
Description: Precision BioSciences, Inc is a biotechnology company headquartered in Durham, United States. Founded in 2006, the company specializes in gene therapy within the biotechnology industry. With a team of approximately 100 employees, Precision BioSciences is dedicated to developing innovative gene editing technologies to address genetic diseases. The company's website provides detailed information about their gene therapy solutions, research projects, and collaborations. Precision BioSciences is committed to advancing the field of gene therapy and making a positive impact on patients' lives through their cutting-edge biotechnological advancements.
#24 Intellia Therapeutics, Inc
Employees: 0.2K
Revenue: N/A
Website: https://www.intelliatx.com
Description: Intellia Therapeutics, Inc is a biotechnology company headquartered in Cambridge, United States. The company was founded in 2014 and operates in the gene therapy industry. With approximately 200 employees, Intellia Therapeutics focuses on developing therapeutic products using CRISPR/Cas9 gene editing technology. The company aims to leverage this technology to address genetic diseases by providing potentially curative treatments. Intellia Therapeutics collaborates with various partners and utilizes its proprietary platform to advance its pipeline of programs. Through its innovative approach, the company strives to improve patient outcomes and revolutionize the field of gene therapy.
#23 LogicBio Therapeutics, Inc
Employees: 0.1K
Revenue: N/A
Website: https://www.logicbio.com
Description: LogicBio Therapeutics, Inc is a gene therapy company based in Cambridge, United States. Founded in 2014, the company specializes in developing genetic medicines for rare diseases. With a focus on precision genome editing, LogicBio Therapeutics aims to address the underlying cause of diseases by integrating therapeutic genes into the patient's own genome. Their proprietary platform, GeneRide™, enables site-specific integration of therapeutic genes into the genome without the need for exogenous nucleases. By harnessing the power of gene therapy, LogicBio Therapeutics is dedicated to transforming the lives of patients with devastating genetic diseases.
#22 Crispr Therapeutics AG
Employees: 0.1K
Revenue: N/A
Website: https://www.crisprtx.com
Description: Crispr Therapeutics AG, headquartered in Zug, Switzerland, is a leading company in the gene therapy industry. Founded in 2014, the company focuses on developing transformative gene-based medicines using its proprietary CRISPR/Cas9 gene-editing platform. With a team of approximately 100 employees, Crispr Therapeutics is dedicated to harnessing the power of CRISPR technology to treat genetic diseases and improve patient outcomes. The company's innovative approach holds great promise in revolutionizing the field of biotechnology and has attracted significant attention from the scientific and medical communities. Through its cutting-edge research and development efforts, Crispr Therapeutics aims to bring life-changing therapies to patients in need.
#21 Rocket Pharmaceuticals, Inc
Employees: 0.1K
Revenue: N/A
Website: https://www.rocketpharma.com/
Description: Rocket Pharmaceuticals, Inc is a biotechnology company based in New York City, United States. Founded in 2015, Rocket Pharmaceuticals focuses on the field of gene therapy. With a team of approximately 100 employees, the company is dedicated to developing advanced genetic medicines for patients suffering from rare and devastating diseases. Rocket Pharmaceuticals aims to provide innovative solutions by leveraging its expertise in lentiviral vector and gene editing technologies. Through extensive research and clinical trials, the company strives to transform the lives of patients and address unmet medical needs in the gene therapy industry.
#20 REGENXBIO Inc
Employees: 0.1K
Revenue: 2M$
Website: https://www.regenxbio.com
Description: REGENXBIO Inc is a leading gene therapy company based in Rockville, United States. Founded in 2008, the company specializes in the development, manufacturing, and commercialization of gene therapy products for a wide range of diseases. With a team of approximately 100 dedicated employees, REGENXBIO is committed to advancing the field of gene therapy and improving patient outcomes. The company's revenue stands at an estimated 2 million dollars, reflecting its success in the biotechnology industry. Through innovative research and strategic partnerships, REGENXBIO aims to transform the treatment landscape by harnessing the power of gene therapy to address unmet medical needs.
#19 Adverum Biotechnologies, Inc
Employees: 0.1K
Revenue: 5M$
Website: https://www.adverum.com
Description: Adverum Biotechnologies, Inc is a leading gene therapy company based in Menlo Park, United States. Founded in 2006, the company specializes in developing novel gene therapies to treat serious ocular diseases. With a team of approximately 100 employees, Adverum Biotechnologies is dedicated to advancing its proprietary vector platform and innovative pipeline of gene therapy candidates. The company's mission is to transform the lives of patients by providing long-lasting and potentially curative treatments for retinal diseases. Adverum Biotechnologies has made significant advancements in the field of gene therapy and continues to explore new treatment options to address unmet medical needs in the biotechnology industry.
#18 Solid Biosciences Inc
Employees: 0.1K
Revenue: 5M$
Website: https://www.solidbio.com
Description: Solid Biosciences Inc, based in Cambridge, United States, is a leading company in the gene therapy sector of the biotechnology industry. The company was founded in 2013 and currently has approximately 100 employees. Solid Biosciences focuses on developing transformative treatments for Duchenne muscular dystrophy (DMD), a genetic disorder that results in progressive muscle degeneration. Leveraging its expertise in gene therapy, the company aims to address the underlying cause of DMD by delivering functional copies of the missing or mutated dystrophin gene to patients' muscles. With a revenue of 5 million dollars, Solid Biosciences continues to advance its innovative gene therapy approaches to potentially provide a new treatment option for individuals affected by DMD.
#17 Rubius Therapeutics, Inc
Employees: 0.5K
Revenue: 5M$
Website: https://www.rubiustx.com
Description: Rubius Therapeutics, Inc is a biotechnology company based in Cambridge, United States. Founded in 2013, the company specializes in gene therapy within the biotechnology industry. With a team of approximately 500 employees, Rubius Therapeutics is dedicated to developing innovative treatments using its proprietary Red Cell Therapeutics platform. The company focuses on engineering red blood cells to create cellular medicines that can potentially treat various diseases, including cancer, autoimmune disorders, and rare genetic diseases. Rubius Therapeutics aims to leverage the unique properties of red blood cells to deliver therapeutic proteins, enzymes, and antibodies directly to target cells in the body. With a revenue of 5 million dollars, the company continues to advance its research and development efforts to revolutionize the field of gene therapy.
#16 Iovance Biotherapeutics, Inc
Employees: 0.1K
Revenue: 10M$
Website: https://www.iovance.com
Description: Iovance Biotherapeutics, Inc is a biotechnology company based in San Carlos, United States. Founded in 2007, the company specializes in gene therapy within the biotechnology industry. With approximately 100 employees, Iovance Biotherapeutics focuses on developing and commercializing novel cancer immunotherapies based on tumor-infiltrating lymphocyte (TIL) technology. Their gene therapy treatments aim to harness the power of a patient's own immune system to target and destroy cancer cells. Iovance Biotherapeutics is committed to advancing innovative gene therapy solutions to improve the lives of cancer patients worldwide.
#15 Editas Medicine, Inc
Employees: 0.1K
Revenue: 10M$
Website: https://www.editasmedicine.com
Description: Editas Medicine, Inc is a biotechnology company based in Cambridge, United States. Founded in 2013, the company operates in the gene therapy industry. With a team of approximately 100 employees, Editas Medicine focuses on developing transformative genomic medicines to treat a range of genetic diseases. The company utilizes CRISPR technology to edit genes and aims to provide innovative therapies for patients in need. Editas Medicine is dedicated to advancing the field of gene editing and has made significant progress in its research and development efforts. With a revenue of approximately 10 million dollars, Editas Medicine continues to explore new possibilities in gene therapy and strives to improve the lives of individuals affected by genetic disorders.
#14 Regenxbio Inc
Employees: 0.2K
Revenue: 10M$
Website: https://www.regenxbio.com
Description: Regenxbio Inc is a biotechnology company headquartered in Rockville, United States. Founded in 2008, the company specializes in gene therapy within the biotechnology industry. With approximately 200 employees, Regenxbio focuses on developing and commercializing gene therapy treatments for various diseases. The company's website, https://www.regenxbio.com, provides further information on their research and development efforts. Regenxbio reported a revenue of 10 million dollars in the most recent financial year.
#13 Orchard Therapeutics Plc
Employees: 0.2K
Revenue: 10M$
Website: https://www.orchard-tx.com/
Description: Orchard Therapeutics Plc is a biotechnology company based in London, United Kingdom. Founded in 2015, the company specializes in gene therapy within the biotechnology industry. With a team of approximately 200 employees, Orchard Therapeutics focuses on developing and commercializing innovative treatments for rare diseases. The company's mission is to transform the lives of patients by providing long-term and potentially curative therapies. Orchard Therapeutics has established a strong presence in the field of gene therapy and continues to advance its pipeline of product candidates. Through its research and development efforts, Orchard Therapeutics aims to address unmet medical needs and improve the lives of patients worldwide.
#12 Audentes Therapeutics, Inc
Employees: 0.1K
Revenue: 10M$
Website: https://www.audentestx.com
Description: Audentes Therapeutics, Inc is a leading biotechnology company specializing in gene therapy. Headquartered in San Francisco, United States, Audentes Therapeutics focuses on developing and commercializing innovative treatments for rare genetic diseases. Founded in 2012, the company has a dedicated team of approximately 100 employees who are committed to advancing gene therapy research and development. Audentes Therapeutics has achieved significant milestones in the field of gene therapy and has generated a revenue of 10 million dollars. With its cutting-edge technology and expertise, the company aims to transform the lives of patients with rare genetic disorders through the development of gene-based medicines.
#11 Sangamo Therapeutics, Inc
Employees: 0.1K
Revenue: 15M$
Website: https://www.sangamo.com
Description: Sangamo Therapeutics, Inc is a biotechnology company based in Richmond, United States. Founded in 1995, the company operates in the gene therapy industry. With approximately 100 employees, Sangamo Therapeutics focuses on developing genomic medicines to treat various genetic diseases. The company utilizes its proprietary zinc finger protein (ZFP) genome editing technology to modify genes and correct genetic mutations. Through their research and development efforts, Sangamo Therapeutics aims to provide innovative therapies to improve the lives of patients with genetic disorders. For more information about their activities and projects, visit their website at https://www.sangamo.com.
#10 Fate Therapeutics, Inc
Employees: 0.5K
Revenue: 50M$
Website: https://fatetherapeutics.com
Description: Fate Therapeutics, Inc is a biotechnology company based in San Diego, United States. Founded in 2013, the company specializes in gene therapy within the biotechnology industry. With approximately 500 employees, Fate Therapeutics focuses on the development and commercialization of programmed cellular immunotherapies for the treatment of cancer and immune disorders. The company utilizes its proprietary induced pluripotent stem cell (iPSC) product platform to engineer and manufacture cell-based therapies. Fate Therapeutics has achieved significant advancements in the field of gene therapy and continues to explore innovative solutions to address unmet medical needs.
#9 UniQure N.V.
Employees: 0.5K
Revenue: 100M$
Website: https://www.uniqure.com
Description: UniQure N.V. is a biotechnology company based in Amsterdam, Netherlands. Founded in 1998, the company specializes in gene therapy, a field that aims to treat genetic diseases by modifying or replacing faulty genes. UniQure develops and commercializes gene therapies for various diseases, including hemophilia, Huntington's disease, and cardiovascular disorders. With a team of approximately 500 employees, the company is at the forefront of advancing gene therapy treatments. UniQure has generated a revenue of around 100 million dollars, leveraging its expertise in the biotechnology industry and its commitment to improving patients' lives through innovative gene therapies.
#8 Spark Therapeutics, Inc
Employees: 0.5K
Revenue: 100M$
Website: https://www.sparktx.com
Description: Spark Therapeutics, Inc, based in Philadelphia, United States, is a leading company in the gene therapy industry. Since its foundation in 2013, the company has been at the forefront of developing innovative gene therapies to treat a range of genetic diseases. With a team of approximately 500 employees, Spark Therapeutics is dedicated to transforming the lives of patients by providing potentially life-changing treatments. The company has achieved significant advancements in the field of gene therapy and has successfully commercialized products such as LUXTURNA®, a gene therapy for inherited retinal disease. Spark Therapeutics continues to expand its pipeline and collaborate with other organizations to accelerate the development of gene therapies for various genetic disorders.
#7 Bluebird Bio, Inc
Employees: 1.2K
Revenue: 150M$
Website: https://www.bluebirdbio.com
Description: Bluebird Bio, Inc is a leading biotechnology company in the gene therapy industry. Headquartered in Cambridge, United States, the company was founded in 2010. With a team of approximately 1.2K employees, Bluebird Bio is dedicated to developing innovative gene therapies to treat severe genetic diseases and cancer. The company focuses on using its proprietary gene editing and gene delivery platforms to develop transformative treatments. Bluebird Bio has made significant advancements in the field of gene therapy, with a strong emphasis on addressing unmet medical needs. Through its research and development efforts, the company aims to provide hope and improved outcomes for patients worldwide.
#6 Ultragenyx Pharmaceutical Inc
Employees: 1.2K
Revenue: 330M$
Website: https://www.ultragenyx.com
Description: Ultragenyx Pharmaceutical Inc is a biotechnology company headquartered in Novato, United States. Founded in 2010, the company operates in the gene therapy industry. With a workforce of approximately 1.2K employees, Ultragenyx focuses on developing and commercializing novel treatments for rare genetic diseases. The company's mission is to transform the lives of patients by bringing innovative therapies to market. Ultragenyx has achieved significant milestones in the field of gene therapy, leveraging its expertise to develop potential cures for various rare diseases. The company's dedication to research and development is reflected in its annual revenue of approximately 330 million dollars.
#5 Bluebird Bio, Inc
Employees: 1.2K
Revenue: 500M$
Website: https://www.bluebirdbio.com
Description: Bluebird Bio, Inc is a leading biotechnology company headquartered in Cambridge, United States. Specializing in gene therapy, Bluebird Bio is at the forefront of developing innovative treatments for genetic diseases. Since its founding in 2010, the company has focused on utilizing gene therapy to address severe genetic disorders by modifying a patient's own cells and reinserting them into their body. With a dedicated team of 1.2 thousand employees, Bluebird Bio has made significant advancements in the field and has achieved promising results in multiple clinical trials. The company's revenue stands at 500 million dollars, reflecting the growing demand for gene therapy solutions in the healthcare industry.
#4 Kite Pharma, Inc
Employees: 1.2K
Revenue: 950M$
Website: https://www.kitepharma.com
Description: Kite Pharma, Inc, headquartered in Santa Monica, United States, is a leading biotechnology company specializing in gene therapy. Since its establishment in 2009, Kite Pharma has made significant advancements in the field of biotechnology, particularly in the area of gene therapy. With a dedicated team of approximately 1.2 thousand employees, the company focuses on developing innovative treatments for cancer and other serious diseases. Kite Pharma has achieved notable success in the industry, generating an impressive revenue of 950 million dollars. Through its cutting-edge research and development efforts, Kite Pharma strives to revolutionize the field of gene therapy and provide life-saving solutions to patients worldwide.
#3 Sarepta Therapeutics, Inc
Employees: 1.2K
Revenue: 950M$
Website: https://www.sarepta.com
Description: Sarepta Therapeutics, Inc is a biotechnology company headquartered in Cambridge, United States. Since its founding in 1980, the company has been a key player in the gene therapy industry. With a workforce of approximately 1.2 thousand employees, Sarepta Therapeutics focuses on developing innovative genetic medicines to treat rare diseases. The company's primary focus is on the development of RNA-based therapeutics, utilizing its proprietary platform technologies. Sarepta Therapeutics has made significant advancements in the treatment of Duchenne muscular dystrophy (DMD), a rare genetic disorder, and is known for its groundbreaking gene therapy products. With a revenue of approximately 950 million dollars, the company continues to attract attention and investment in the biotechnology sector.
#2 BioMarin Pharmaceutical Inc
Employees: 3.2K
Revenue: 1.7B$
Website: https://www.biomarin.com
Description: BioMarin Pharmaceutical Inc, headquartered in San Rafael, United States, is a leading company in the gene therapy industry. Since its establishment in 1997, the company has been dedicated to developing innovative therapies for rare genetic diseases. With a workforce of approximately 3,200 employees, BioMarin is committed to transforming the lives of patients through cutting-edge research and development. The company's revenue reached 1.7 billion dollars, a testament to its success in the biotechnology sector. BioMarin's focus on gene therapy has positioned it as a key player in the advancement of personalized medicine, aiming to address unmet medical needs and improve patient outcomes.
#1 Novartis International AG
Employees: 110K
Revenue: 47.5B$
Website: https://www.novartis.com/
Description: Novartis International AG, headquartered in Basel, Switzerland, is a leading player in the gene therapy industry within the biotechnology sector. Founded in 1996, the company has grown to employ approximately 110,000 individuals worldwide. With a revenue of 47.5 billion USD, Novartis focuses on developing innovative gene-based treatments that aim to address a wide range of diseases and conditions. Through their extensive research and development efforts, Novartis strives to revolutionize healthcare by harnessing the potential of gene therapy to provide targeted and personalized treatment options. Their commitment to advancing gene therapy has positioned Novartis as a key player in the biotechnology industry.
Summary
Gene therapy is a rapidly growing sub-industry within the field of biotechnology, with several companies making significant advancements in this field. These companies have been ranked based on their market capitalization and their contributions to gene therapy research and development. The top companies in this sub-industry have achieved remarkable success and have made substantial progress in the treatment of genetic diseases.
One unexpected finding during the investigation is the increasing interest and investment in gene therapy by major pharmaceutical companies. This indicates the potential of gene therapy to revolutionize the healthcare industry and offers new hope for patients with genetic disorders.
The premier companies in gene therapy include industry giants like Novartis, Bluebird Bio, and Spark Therapeutics. These companies have dedicated significant resources to gene therapy research and have successfully developed and commercialized gene therapies for various genetic diseases.
One common element driving the success of these leading firms is their focus on innovative gene delivery methods. They have developed sophisticated delivery systems that can effectively deliver therapeutic genes to target cells in the body. This ensures the efficient and safe delivery of gene therapies.
What sets these eminent companies apart from their industry rivals is their ability to overcome the challenges associated with gene therapy, such as immune responses, manufacturing scalability, and pricing. They have developed cutting-edge technologies to address these challenges and have demonstrated promising results in clinical trials.