The Global Drugs Based on Gene Therapy Market was valued at USD 9.06 Billion in 2023 and is projected to reach USD 47.47 Billion by 2032, expanding at an impressive Compound Annual Growth Rate (CAGR) of 20.2% during the forecast period (2023-2032). This exponential growth stems from advancements in vector technologies, increasing FDA approvals, and growing clinical evidence of long-term therapeutic benefits in treating genetic disorders.
As the biopharmaceutical industry shifts toward precision genetic medicine, investment in gene therapy development has reached unprecedented levels. This analysis profiles the Top 10 Companies Dominating the Gene Therapy Drugs Market, featuring pioneering biotechs and established pharma giants shaping the future of curative treatments.
🔟 1. Biogen Inc.
Headquarters: Cambridge, Massachusetts, USA
Key Products: Spinraza®, AAV-based neurological therapies
A neuroscience leader turning to gene therapy, Biogen strengthened its position through strategic acquisitions including Nightstar Therapeutics. The company’s adeno-associated virus (AAV) vector platform targets central nervous system disorders with high unmet needs.
Pipeline Highlights:
- Phase III AAV9 gene therapy for spinal muscular atrophy
- Clinical-stage ophthalmic gene therapies
- Novel capsid development for improved CNS targeting
9️⃣ 2. Novartis AG
Headquarters: Basel, Switzerland
Key Products: Zolgensma®, Kymriah®
The commercial leader in gene therapies, Novartis owns both the first FDA-approved gene therapy for spinal muscular atrophy and pioneering CAR-T cell therapy. Its 2018 acquisition of AveXis established a robust AAV manufacturing infrastructure.
Pipeline Highlights:
- 3 clinical-stage CNS gene therapies
- Next-gen CAR-T constructs in development
- Scalable gene therapy manufacturing platforms
8️⃣ 3. Gilead Sciences (Kite Pharma)
Headquarters: Foster City, California, USA
Key Products: Yescarta®, Tecartus®
Gilead’s Kite subsidiary dominates the gene-modified cell therapy space with two FDA-approved CAR-T treatments. The company continues innovating with allogeneic “off-the-shelf” therapies and improved manufacturing processes.
Pipeline Highlights:
- 4 clinical-stage CAR-T candidates
- Allogeneic cell therapy programs
- Automated cell processing technologies
Download FREE Sample Report: Drugs Based on Gene Therapy Market – View in Detailed Research Report
7️⃣ 4. Sarepta Therapeutics
Headquarters: Cambridge, Massachusetts, USA
Key Products: Elevidys®, DMD gene therapy pipeline
A pure-play gene therapy innovator, Sarepta focuses exclusively on genetic medicines for rare diseases. The company received accelerated approval for its Duchenne muscular dystrophy gene therapy in 2023.
Pipeline Highlights:
- 6 clinical-stage DMD gene therapies
- Gene editing and RNA-targeting platforms
- Muscle-targeted delivery technologies
6️⃣ 5. Alnylam Pharmaceuticals
Headquarters: Cambridge, Massachusetts, USA
Key Products: Onpattro®, Amvuttra®
The RNA interference therapy leader has pioneered nucleic acid-based gene silencing. Alnylam’s proprietary delivery platform enables targeting of liver, CNS, and other tissues for genetic disorders.
Pipeline Highlights:
- 7 clinical-stage RNAi therapeutics
- Novel conjugate delivery technologies
- Cardiometabolic and CNS pipelines
5️⃣ 6. Spark Therapeutics (Roche)
Headquarters: Philadelphia, Pennsylvania, USA
Key Products: Luxturna®
A Roche subsidiary since 2019, Spark developed the first FDA-approved gene therapy for a genetic disease (retinal dystrophy). The company maintains a diverse pipeline of AAV-based therapies.
Pipeline Highlights:
- Phase III hemophilia A gene therapy
- CNS-targeted AAV programs
- Novel capsid engineering research
Download FREE Sample Report: Drugs Based on Gene Therapy Market – View in Detailed Research Report
4️⃣ 7. Bluebird bio
Headquarters: Somerville, Massachusetts, USA
Key Products: Zynteglo®, Skysona®
A pioneer in lentiviral gene therapies, bluebird bio specializes in ex-vivo gene-modified cell treatments for hematologic and CNS disorders, with two approved therapies for genetic blood diseases.
Pipeline Highlights:
- Gene therapy for cerebral adrenoleukodystrophy
- Sickle cell disease program
- Lentiviral manufacturing expertise
3️⃣ 8. Amgen
Headquarters: Thousand Oaks, California, USA
Key Products: Imlygic®, investigational gene therapies
Amgen is expanding beyond biologics into gene therapies, drawing on its manufacturing scale and oncology expertise. The company is developing both in vivo gene therapies and ex vivo cell therapies.
Pipeline Highlights:
- Oncolytic virus therapy platform
- Cardiovascular gene therapy programs
- Strategic partnerships with gene therapy innovators
2️⃣ 9. Orchard Therapeutics
Headquarters: London, United Kingdom
Key Products: Libmeldy®
Specializing in hematopoietic stem cell gene therapies, Orchard focuses on severe inherited metabolic and immune disorders. The company’s ex vivo approach genetically modifies a patient’s own stem cells.
Pipeline Highlights:
- Approved therapy for metachromatic leukodystrophy
- Clinical programs for Wiskott-Aldrich syndrome
- Next-generation stem cell engineering
1️⃣ 10. Human Stem Cells Institute (HSCI)
Headquarters: Moscow, Russia
Key Products: Neovasculgen®, cell/gene therapies
A leader in Eastern European gene therapy markets, HSCI develops and commercializes regenerative medicines with a focus on cardiovascular applications and rare diseases.
Pipeline Highlights:
- Commercial VEGF gene therapy product
- Personalized medicine approaches
- Expanding clinical trial network
Get Full Report Here: Drugs Based on Gene Therapy Market – View in Detailed Research Report
🧬 Future Outlook: The Next Frontier of Genetic Medicine
The gene therapy sector has evolved from experimental treatments to commercially viable precision medicines. With over 400 clinical trials underway, the industry is overcoming initial challenges around manufacturing, delivery, and long-term efficacy.
📊 Market Transformation Drivers:
- Accelerated regulatory pathways in US/EU (11 FDA approvals as of 2024)
- Improved viral vector production capacity (>30% annual increase)
- Value-based pricing models gaining payer acceptance
- Expansion beyond rare diseases to common conditions
🔄 Emerging Technology Trends:
- Next-generation viral vectors with enhanced targeting
- Gene editing platforms (CRISPR, base editing)
- Redosable and regulatable systems
- Automated cell therapy manufacturing
The profiled companies represent the vanguard of this therapeutic revolution, translating decades of research into life-changing treatments for patients with genetic disorders.
Get Full Report Here: Drugs Based on Gene Therapy Market – View in Detailed Research Report