April 11, 2025
Gene therapy is advancing rapidly, with the industry projected to grow from $3.61 billion in 2019 to $35.6 billion by 2027, driven by rising demand for treatments targeting cancer, neurological conditions, and muscular atrophy.
This growth stems from improved viral vector technology (the modified virus that delivers genes to cells) and targeted therapies (focus on specific genes, proteins, or cellular processes) like T-cell treatments for B-cell lymphoma. While current treatments remain costly, increasing FDA approvals and ongoing research are accelerating progress, particularly in the U.S. and Europe.
In this blog, we'll highlight key trends in gene therapy and the top companies driving innovation. We'll also explain how Biostate AI supports researchers with affordable total RNA sequencing services, helping them advance genetic research efficiently.
Gene therapy is a medical approach that modifies genes to treat or prevent disease. It can replace faulty genes, disable malfunctioning ones, or introduce new genes to help the body fight illness. Researchers are exploring its potential for conditions like cancer, cystic fibrosis, and sickle cell disease, with some therapies already FDA-approved.
Notable treatments include gene therapy for Leber congenital amaurosis, a rare blindness-causing condition, and CAR T-cell therapy for blood cancers like acute lymphoblastic leukemia (ALL). As research advances, the growing demand for gene therapy drives rapid market expansion.
The global gene therapy market is projected to reach $2.4 billion by 2035, with a compound annual growth rate (CAGR) of 19.25%. This growth reflects rising demand for treatments targeting genetic disorders, which affect approximately 70 million people worldwide and contribute to over 40% of infant mortality cases.
Here are some key factors driving the growth of the industry.
As the gene therapy market expands, identifying the companies driving innovation becomes crucial. These industry leaders are often at the forefront of developing groundbreaking therapies, attracting investment, and shaping future treatment options. Knowing what sets them apart offers valuable insights into key advancements in the field.
The gene therapy sector includes approximately 345 companies worldwide, ranging from emerging biotech firms to established pharmaceutical leaders. As competition grows, innovation is advancing treatment options for genetic and acquired conditions.
Companies in this field are often ranked based on three key factors.
With these factors in mind, several companies have emerged as key players in the gene therapy industry, driving innovation and delivering promising treatments.
Top gene therapy companies excel in developing treatments using advanced genetic techniques. They often lead the industry with strong research capabilities, successful clinical programs, and approved therapies that address conditions with limited treatment options. These companies also tend to have solid financial backing, supporting ongoing innovation and development.
Below are seven leading companies that are shaping the future of gene therapy with their promising treatments and research efforts.
Krystal Biotech is a gene therapy company focused on treating rare skin conditions using its proprietary STAR-D platform. This platform employs a modified HSV-1 vector to deliver genes directly to skin cells, allowing repeat dosing without strong immune responses.
Their lead product, Vyjuvek™, is the first FDA-approved gene therapy for dystrophic epidermolysis bullosa (DEB), a severe genetic disorder. Vyjuvek is a topical treatment that delivers the COL7A1 gene to promote collagen production and improve wound healing.
Krystal Biotech is also developing KB105 for TGM1 deficiency and KB104 for Netherton syndrome, with additional preclinical programs underway. The company's financial position has improved after Vyjuvek’s FDA approval and commercial launch.
CRISPR Therapeutics develops gene-editing therapies using its CRISPR-Cas9 platform. Its lead product, exagamglogene autotemcel (exa-cel), is approved in the US, EU, and UK for sickle cell disease and beta-thalassemia. Exa-cel edits stem cells to boost fetal hemoglobin production.
The company is also advancing allogeneic CAR-T therapies for cancers and exploring in vivo gene editing for liver, muscle, and other conditions. With substantial financial backing, CRISPR Therapeutics continues to expand its CRISPR-Cas9 applications to address new disease targets.
Beam Therapeutics specializes in base editing, a precise gene editing technology that enables single-base changes in DNA and RNA without causing double-strand breaks. This method reduces off-target effects and supports targeted therapies for genetic diseases, cancer, and infections.
Key programs include BEAM-101 for sickle cell disease, BEAM-201 for T-cell acute lymphoblastic leukemia, and BEAM-301 for Glycogen Storage Disease Ia (GSDIa). With substantial financial backing and significant pharmaceutical partnerships, Beam Therapeutics continues to expand its platform and advance its lead candidates.
Intellia Therapeutics is a gene editing company developing CRISPR-based therapies for genetic diseases, autoimmune conditions, and cancers. The company is advancing both in vivo (editing genes inside the body) and ex vivo (editing cells outside the body) approaches.
Key programs include NTLA-2001 for transthyretin amyloidosis (Phase 3), NTLA-2002 for hereditary angioedema (Phase 3), and NTLA-3001 for Alpha-1 Antitrypsin Deficiency (Phase 1).
Intellia has secured strong financial backing through partnerships, notably with Regeneron Pharmaceuticals. In October 2023, NTLA-2002 received PRIME designation from the EMA, which supports faster development and review. The company continues to progress its clinical trials, aiming to demonstrate the safety and efficacy of its therapies.
uniQure is a gene therapy company developing treatments for rare and severe diseases, focusing on neurological disorders, liver conditions, and hemophilia. Their technology platform uses adeno-associated virus (AAV) vectors to improve gene delivery efficiency.
Key programs include EtranaDez (AMT-061) for hemophilia B, which aims to enable Factor IX production, and AMT-130 for Huntington's disease, designed to reduce mutant huntingtin protein levels.
uniQure has secured funding through partnerships and public offerings. The company continues to share promising clinical data for EtranaDez and is actively enrolling patients in AMT-130 trials. Their pipeline includes four candidates targeting various conditions.
Verve Therapeutics is a gene editing company developing single-course treatments for cardiovascular disease. They focus on using base editing to modify DNA in the liver, reducing proteins linked to lipid metabolism. Their lead candidate, VERVE-101, targets the PCSK9 gene to lower LDL cholesterol levels permanently. They are also working on therapies targeting ANGPTL3 and LPA genes.
Verve has secured funding through venture capital and partnerships, supporting its research efforts. The company recently began clinical trials for VERVE-101 in patients with heterozygous familial hypercholesterolemia (HeFH) and has presented encouraging preclinical data on its lipid-lowering potential. Their pipeline currently includes four candidates.
MeiraGTx is a clinical-stage gene therapy company focused on developing treatments for inherited retinal diseases, neurodegenerative disorders, and salivary gland conditions. They use adeno-associated virus (AAV) vectors for gene delivery.
Their key programs include:
MeiraGTx has secured funding through venture investments and partnerships. The company is advancing therapies for conditions with significant unmet needs, and six products are in developing. They are recognized as one of the top gene therapy companies based on their robust pipeline.
These seven companies are advancing gene therapy with innovative approaches like CRISPR, base editing, and viral vector delivery. While these technologies focus on precise gene modifications, understanding their impact at a molecular level is equally important.
This is where RNA sequencing (RNA-Seq) plays a vital role. By analyzing gene expression, RNA-Seq helps researchers track how cells respond to gene therapies, ensuring treatments target the intended genes effectively. It also helps identify potential side effects, monitor therapeutic progress, and refine dosing strategies. RNA-Seq remains key to improving both safety and long-term outcomes as gene therapy develops.
Gene therapy's rapid growth highlights its potential to transform treatments for cancer, neurological conditions, and other complex diseases. As research expands, innovations in viral vectors and targeted therapies push the industry forward. However, with high development costs and ongoing clinical trials, researchers face challenges in delivering effective solutions.
Biostate AI supports gene therapy research with affordable total RNA sequencing starting at $80 per sample. Our services help researchers study gene expression and treatment responses with ease. By simplifying data collection and analysis, we enable scientists to focus on breakthroughs. Biostate AI makes RNA sequencing accessible for projects of all sizes.
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1. How is gene therapy delivered to patients?
A: Gene therapy is typically delivered using modified viruses or non-viral methods like nanoparticles or direct injections to transfer genetic material into target cells.
2. Is gene therapy a one-time treatment?
A: Some gene therapies are designed as single-dose treatments, especially for genetic disorders, while others may require multiple doses depending on the condition.
3. What are the risks of gene therapy?
A: Potential risks include immune reactions, unintended effects on other genes, or the risk of the modified virus causing harm. Clinical trials aim to minimize these risks.
4. Who is eligible for gene therapy?
A: Eligibility depends on the condition being treated, the patient's health status, and available treatment options. Gene therapy is often considered for severe or rare conditions.
5. How long does it take for gene therapy to show results?
A: The timeline varies; some patients may see improvements within weeks, while others may require longer monitoring to assess outcomes.
