Gene therapy is rapidly transforming the landscape of modern medicine, offering promising solutions for previously incurable diseases. In 2024, several companies are at the forefront of this groundbreaking field, driving innovation and expanding the possibilities of genetic treatments. These pioneers are not only developing cutting-edge therapies but also setting new standards for patient care and medical advancements. In this article, we highlight the top 10 companies leading the charge in gene therapy, showcasing their most significant contributions and breakthroughs. Discover how these trailblazers are shaping the future of healthcare and improving lives worldwide.
The global gene therapy market, valued at USD 7.4 billion in 2022, is poised for significant growth, projected to reach an impressive USD 29.2 billion by 2031. This market is expected to exhibit a robust compound annual growth rate (CAGR) of 19.4% during the forecast period from 2024 to 2031. A key market trend driving this growth is the accelerated development of gene therapies for rare diseases, highlighting the sector's focus on innovative treatments for previously unmet medical needs.
Top 10 Gene Therapy Companies Worldwide
1) Gilead Sciences, Inc:
Gilead Sciences, Inc. is a leading biopharmaceutical company with over three decades of pioneering breakthroughs in medicine, dedicated to creating a healthier world for everyone. Committed to advancing innovative treatments, Gilead focuses on preventing and treating life-threatening diseases such as HIV, viral hepatitis, and cancer. With operations in more than 35 countries worldwide, Gilead has a global impact. Recently, Gilead Sciences, Inc. and Kite Pharma, Inc. announced a definitive agreement under which Gilead will acquire Kite. Gilead specializes in discovering, developing, manufacturing, and commercializing proprietary therapeutics for challenging infectious diseases, including viral, fungal, and bacterial infections, as well as cancer.
2) Novartis AG:
Novartis is a leading global medicines company that leverages innovative science and digital technologies to create transformative treatments for critical medical needs. The company is dedicated to developing, manufacturing, and marketing healthcare products. Its innovative medicines segment focuses on researching, developing, manufacturing, distributing, and selling patented pharmaceuticals, and comprises two business units: Novartis Oncology and Novartis Pharmaceuticals. Novartis AG has assembled a team with exceptional expertise, unified by a shared vision to develop gene therapies that have the potential to positively impact the lives of patients and families affected by rare and life-threatening neurological genetic diseases.
3) Amgen Inc:
Amgen Inc. (Applied Molecular Genetics Inc.) is an American multinational biopharmaceutical company specializing in therapeutic areas such as cardiovascular disease, oncology, bone health, neuroscience, nephrology, and inflammation. Amgen boasts a diverse biosimilar portfolio that spans multiple therapeutic areas, including oncology, immunology, and supportive care. The company's commitment to biosimilars is driven by its belief in expanding patient access to high-quality, cost-effective biologic therapies. Additionally, Amgen has a wide range of biosimilar products in various stages of development, focusing on biosimilars for well-established reference products, while anticipating the expiration of patents and exclusivity rights for these biologics.
4) Bluebird Bio:
Bluebird bio is at the forefront of the gene therapy revolution, boasting nearly 15 years of scientific and clinical expertise in rare diseases and immuno-oncology. With a proven scientific approach and a lentiviral-based manufacturing platform, Bluebird bio leverages a complementary set of tools, including gene editing, to advance its innovative therapies. The company actively supports programs and activities designed to educate healthcare professionals and the scientific community about diseases and their treatment options, often providing sponsorship funding to third-party institutions or organizations. Committed to transparency and maintaining the independence of patient organizations, Bluebird bio ensures ethical and clear interactions with its customers.
5) Biogen:
Biogen is a leading global biotechnology company that pioneers scientific advancements and drives innovations for complex and devastating diseases. Biogen is advancing a robust pipeline of potential therapies across neurology, neuropsychiatry, specialized immunology, and rare diseases, all while remaining focused on its mission to serve humanity through science and foster a healthier, more sustainable, and equitable world. The company has several product candidates targeting a range of indications, including Parkinson's disease, Lupus, Depression, CNS and neuromuscular disorders, Amyotrophic Lateral Sclerosis, and Alzheimer’s disease. Biogen markets its products through a direct sales force, marketing groups, and distributors across the Americas, Europe, Asia, and other regions.
6) BioMarin:
BioMarin focuses on developing first-in-class and best-in-class therapeutics that provide life-changing advances to patients with rare genetic diseases. The company creates and markets innovative drugs for the treatment of uncommon genetic conditions such as Duchenne muscular dystrophy (DMD), achondroplasia, phenylketonuria (PKU), late infantile neuronal ceroid lipofuscinosis (CLN2), and hemophilia. BioMarin's discoveries have led to many industry 'firsts' and best-in-class therapies, driving significant advancements in the field. The company is proud of its role in improving the lives of individuals affected by hereditary illnesses, consistently pushing the boundaries of medical science.
7) Ferring Pharmaceuticals:
Ferring Pharmaceuticals is a research-driven, specialty biopharmaceutical group with a strong global presence. The company identifies, develops, and markets innovative products in reproductive medicine and maternal health, urology, and gastroenterology. With over 50 years of experience in developing treatments for mothers and babies, Ferring Pharmaceuticals has established itself as a leader in these fields. The company operates its own manufacturing facilities in several European countries, South America, China, India, and the U.S., and has subsidiaries in nearly 60 countries. Ferring markets its treatments in 110 countries, ensuring its innovative solutions reach patients worldwide.
8) F. Hoffmann-La Roche Ltd:
Roche is a leader in research-focused healthcare, leveraging its combined strengths in pharmaceuticals and diagnostics. As one of the largest biotech companies, Roche offers truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology, and neuroscience. The company is also a global leader in in vitro diagnostics and tissue-based cancer diagnostics, as well as a frontrunner in diabetes management. Roche's personalized healthcare strategy aims to provide medicines and diagnostics that enable tangible improvements in patients' health, quality of life, and survival. The company offers its products and services to hospitals, commercial laboratories, healthcare professionals, researchers, and pharmacists. Alongside its subsidiaries and partners, Roche operates in various countries worldwide, extending its impact globally.
9) Spark Therapeutics, Inc:
Spark Therapeutics, Inc. is a leading developer of gene therapy products, dedicated to unlocking the power of gene therapy to accelerate healthcare transformation. The company focuses on discovering, developing, and delivering one-time gene therapies, with specializations in retina-directed, liver-directed, and central nervous system therapies. These therapies are designed for patients with viable retinal cells and confirmed biallelic RPE65 mutation-associated retinal dystrophy. In 2019, Spark Therapeutics was acquired by Hoffmann-La Roche, further enhancing its capabilities and reach in the gene therapy field.
10) Bristol-Myers Squibb Company:
Bristol-Myers Squibb Co. (BMS) is a specialty biopharmaceutical company dedicated to the discovery, development, licensing, manufacturing, marketing, distribution, and sale of medicines and related medical products for patients with serious diseases. The company's primary focus areas include cancer, cardiovascular, immunology, and fibrotic therapeutic projects. BMS distributes its products worldwide to wholesalers, retail pharmacies, medical professionals, hospitals, and government entities, with a strong presence in the US, Europe, and Japan. The company's research efforts are centered on discovering and developing novel medicines that address serious diseases in areas of significant unmet medical need.
