10 gene therapy companies you should know about

By addressing the root cause of disease, rather than simply treating symptoms, the field of gene therapy has transformed modern medicine. From rare inherited disorders to cancer and cardiovascular conditions, there are numerous gene therapy companies harnessing cutting-edge tools like CRISPR and in vivo delivery systems to develop breakthrough treatments for many different diseases. In this article, we have listed 10 companies hoping to develop novel gene therapy candidates that will make a difference to people’s lives.  

4D Molecular Therapeutics  

• Headquarters: U.S. 
• Lead gene therapy candidate: 4D-150 for wet AMD and diabetic macular edema 
• Recent news: Announced the pricing of a $100 million underwritten offering 

Driven by an innovative vector platform and efficient product design and development engine, 4D Molecular Therapeutics, or 4DMT, is developing genetic medicines with customized vectors that are optimized for specific diseases. The company’s pipeline programs are currently focused on ophthalmology and pulmonology. 

4DMT has two clinical candidates in its pipeline. Its lead candidate, 4D-150, is being developed to treat wet age-related macular degeneration (AMD) and diabetic macular edema (DME). Just recently, the company announced positive interim 1.5- to 3.5-year data from its phase 1/2 clinical trial evaluating 4D-150 in patients with wet AMD, supporting the gene therapy’s potential as a backbone therapy with consistent and durable benefit over several years. The candidate is now in phase 3 trials, with enrollment expected to be completed in H2 2026.  

Meanwhile, the company’s other clinical candidate, 4D-710, is being developed to treat cystic fibrosis, and is currently in phase 2 of development. 4DMT announced last month that the Cystic Fibrosis Foundation (CF Foundation) would provide up to $11 million in additional funding and technical support to accelerate the development of 4D-710.  

Over the last month, 4DMT has announced an exclusive license agreement with Otsuka to develop and commercialize 4D-150 for the treatment of wet AMD and DME in the greater Asia-Pacific region, and announced the pricing of a $100 million underwritten offering.  

AAVantgarde 

• Headquarters: Italy 
• Lead gene therapy candidates: AAVB-039 for Stargardt disease and AAVB-081 for retinitis pigmentosa 
• Recent news: Raised $141 million in a series B round 

AAVantgarde is advancing gene therapy programs for inherited retinal diseases. Its lead programs target Stargardt disease and retinitis pigmentosa due to Usher syndrome type 1B, two inherited retinal diseases caused by mutations in unusually large genes that conventional adeno-associated virus (AAV) vectors cannot accommodate. However, AAVantgarde’s platform is designed to bypass this limitation, potentially broadening the reach of gene therapy to patients with diseases that have previously been considered untreatable. 

The company’s Stargardt disease candidate, AAVB-039, is currently undergoing a phase 1/2 trial. It works by delivering the full-length ABCA4 protein, and could address the root cause of the eye condition. Meanwhile, AAVantgarde’s candidate for retinitis pigmentosa associated with Usher syndrome type 1B, called AAVB-081, is also undergoing a phase 1/2 study. As the 1B variety of Usher syndrome is caused by mutations in the MYO7A gene, AAVB-081 is designed to deliver the MYO7A protein by gene augmentation. 

Earlier this month, the ophthalmology company closed a $141 million series B round, which will go toward supporting the completion of clinical proof-of-concept of the company’s two clinical studies.  

Beam Therapeutics 

• Headquarters: U.S. 
• Lead gene therapy candidate: BEAM-101 for sickle cell disease 
• Recent news: The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to BEAM-101

Beam Therapeutics is known in the industry for its commitment to base editing, a variant of CRISPR that enables the replacement of one DNA base with another without cutting both strands of the DNA. The aim of this approach is to reduce the risk of unwanted insertions or deletions and off-target damage associated with traditional CRISPR-Cas9 editing.  

The company’s lead clinical candidate is BEAM-101, a genetically modified cell therapy being developed for the treatment of sickle cell disease. The candidate is a one-time therapy designed to inhibit the transcriptional repressor BCL11A from binding to the promoter without disrupting BCL11A expression, leading to increased production of non-sickling and anti-sickling fetal hemoglobin, therefore mimicking the effects of naturally occurring variants seen in hereditary persistence of fetal hemoglobin. 

In June 2025, Beam announced new safety and efficacy data from its phase 1/2 trial of BEAM-101, in which 17 treated patients showed durable increases in fetal hemoglobin, reductions in sickle hemoglobin, fast engraftment, normalized hemolysis markers, and no vaso-occlusive crises after engraftment. Moreover, in August, the U.S. Food and Drug Administration (FDA) also granted Regenerative Medicine Advanced Therapy designation to BEAM-101 – the designation is designed to support the development and evaluation of regenerative medicines, including genetic therapies, with the intention of addressing serious or life-threatening diseases that have unmet medical needs. 

CRISPR Therapeutics 

• Headquarters: U.S. and Switzerland 
• Latest gene therapy approval: Casgevy for sickle cell disease and beta-thalassemia 
• Recent news: Announced positive phase 1 results for in vivo CRISPR/Cas9 gene therapy CTX310 for cardiovascular disease 

A big name within the gene therapy sector, CRISPR Therapeutics is focused on developing transformative gene therapies for serious diseases using its proprietary CRISPR/Cas9 platform. The company has established a portfolio of therapeutic programs across a broad range of disease areas, including hemoglobinopathies, oncology, regenerative medicine, cardiovascular diseases, autoimmune diseases, and rare diseases.  

The company is applying gene editing to treat disease in two primary ways: targeting specific genes that cause or influence the course of a disease and engineering the next generation of cell therapies that can address an array of diseases. In either case, the cells can be edited ex vivo or in vivo using the CRISPR/Cas9 delivery system. 

It was CRISPR Therapeutics that, in partnership with Vertex Pharmaceuticals, received FDA approval in December 2023 for the first-ever CRISPR-based gene therapy, CASGEVY. The gene therapy essentially works by turning on fetal hemoglobin, which makes it so that sickle cell disease or beta-thalassemia patients have no symptoms. More specifically, CASGEVY targets the BCL11A gene, as this is what usually prevents the production of fetal hemoglobin in adults.  

According to its website, the gene therapy company currently has five other candidates in clinical trials, listed under the areas of autoimmune disease and immuno-oncology, in vivo approaches, regenerative medicine, and other disclosed partnered programs. It recently announced positive phase 1 results from its ongoing trial of CTX310, an in vivo CRISPR/Cas9 gene-editing therapy targeting ANGPTL3 for the treatment of cardiovascular disease.  

Editas Medicine 

• Headquarters: U.S. 
• Lead gene therapy candidate: EDIT-401 for LDL cholesterol 
• Recent news: Pivoted toward focus on in vivo gene editing medicines and nominated EDIT-401 as lead development candidate 

Editas Medicine is focused on translating the power and potential of the CRISPR/Cas12a and CRISPR/Cas9 genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. While Cas9 is able to locate, bind to, and edit targeted genes, Cas12a greatly expands the range of ways that CRISPR gene editing can be used to produce genomic medicines. It provides researchers with more options for where and how to edit DNA and makes use of a shorter guide RNA than Cas9.  

The gene therapy company has recently pivoted toward focusing on in vivo approaches; in September 2025, it nominated its lead in vivo development candidate, called EDIT-401, which is a one-time therapy designed to significantly reduce LDL cholesterol (LDL-C) levels. It has shown an approximately 90% mean reduction of LDL-C in preclinical studies, and is on track for human proof-of-concept data by the end of 2026.  

Editas has also made several significant collaborations in recent years. In December 2023, Vertex Pharmaceuticals entered into a non-exclusive license agreement with Editas for its Cas9 gene editing technology. And, last year, Editas announced a two–year extension to its collaboration with Bristol Myers Squibb to research, develop, and commercialize autologous and allogeneic alpha-beta T cell medicines for the treatment of cancer and autoimmune diseases, as well as a collaboration with Genevant that combines Editas’ CRISPR Cas12a genome editing systems with Genevant’s lipid nanoparticle (LNP) technology in the development of in vivo gene editing medicines.  

Genetix Biotherapeutics 

• Headquarters: U.S. 
• Latest gene therapy approval: Lyfgenia for sickle cell disease 
• Recent news: Changed its name from bluebird bio to Genetix Biotherapeutics 

Previously known as bluebird bio, Genetix Biotherapeutics has recently “returned to its roots” with a name that is almost identical to the one it had when it was first founded in 1992 as Genetix Pharmaceuticals. After being bought by Carlyle and SK Capital this summer, the gene therapy company has also put in place a newly appointed leadership team, transitioned into a privately held company, and has a newly sharpened focus on “commercial execution to deliver life-changing genetic therapies.” 

In 2022, Genetix received two back-to-back approvals – the first for ZYNTEGLO to treat beta-thalassemia, and the second for SKYSONA to treat early, active cerebral adrenoleukodystrophy (CALD), a rare, progressive, neurodegenerative disease that primarily affects young boys. 

Meanwhile, the company’s latest approval came in December 2023, after the FDA approved its treatment for sickle cell disease. The treatment, called Lyfgenia (also known as lovo-cel), is a one-time gene therapy that has the potential to resolve vaso-occlusive events and is custom-designed to treat the underlying cause of sickle cell disease, which is a complex and progressive genetic disease associated with unpredictable and debilitating vaso-occlusive events. LYFGENIA works by permanently adding a functional β-globin gene to patients’ own hematopoietic (blood) stem cells (HSCs). 

Kriya Therapeutics  

• Headquarters: U.S. 
• Lead gene therapy candidate: KRIYA-825 for geographic atrophy 
• Recent news: Raised $320 million in series D funding 

Kriya Therapeutics wants to revolutionize how gene therapies are designed, developed, and manufactured, with a goal of improving speed to market and reducing cost so that these treatments can be accessible “not just for the few, but for the many”. The company’s pipeline currently includes nine candidates for chronic diseases, including geographic atrophy, thyroid eye disease, type 1 diabetes, metabolic associated steatohepatitis (MASH) and trigeminal neuralgia. 

According to Kriya’s website, it now has three candidates in clinical trials: KRIYA-825 for geographic atrophy, KRIYA-586 for thyroid eye disease, and KRIYA-748 for trigeminal neuralgia. Although early preclinical data has been presented for some of its candidates, the company has tended to stay relatively secretive and, therefore, not much is known about the progress of these drugs. 

In September 2025, Kriya closed an eye-catching $320 million series D round that curiously opposed the current downward trend in gene therapy funding. It said that the proceeds from this would be used to support clinical trials for its five lead gene therapy programs, as well as the continued utilization of the company’s research and manufacturing engine for new product development. This round follows a number of big fundraisings for the gene therapy company; in 2020, it raised $80 million in series A funding, before bringing home $100 million in series B funding in 2021, and $270 million in series C funding in 2022.  

MeiraGTx 

• Headquarters: U.S. and U.K. 
• Lead gene therapy candidates: AAV-GAD for Parkinson’s disease and AAV-AQP1 for radiation-induced xerostomia 
• Recent news: Entered into a collaboration with Eli Lilly to develop and commercialize genetic medicines in ophthalmology 

With a gene regulation platform technology that allows precise, dose-responsive control of gene expression by oral small molecules with a dynamic range that can exceed 5000-fold, MeiraGTx is looking to develop treatments for patients suffering from a range of serious diseases. It has an initial focus on three distinct areas of unmet medical need, which include ocular diseases, neurodegenerative diseases, and severe forms of xerostomia – otherwise known as radiation-induced dry mouth.  

The gene therapy company currently has several programs in late-stage clinical development: a phase 3-ready program called for the treatment of Parkinson’s disease that recently received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA, a potentially pivotal phase 2 program for the treatment of radiation-induced xerostomia, and a program for the treatment of AIPL1-associated congenital blindness. 

Earlier this year, MeiraGTx entered into a collaboration with Hologen AI to expedite the phase 3 development of its Parkinson’s disease candidate, AAV-GAD, and industrialize the company’s proprietary manufacturing process. Moreover, earlier this week, MeiraGTx also entered into a strategic collaboration with pharma giant Eli Lilly to develop and commercialize genetic medicines in ophthalmology.  

Rocket Pharmaceuticals 

• Headquarters: U.S. 
• Lead gene therapy candidate: RP-A501 for Danon disease 
• Recent news: The FDA accepted the resubmission of a Biologics License Application for KRESLADI for severe LAD-I 

Rocket Pharmaceuticals is advancing a sustainable pipeline of genetic therapies designed to correct the root cause of complex and rare disorders. The company uses a multi-platform approach that allows it to design the optimal gene therapy for each indication. Its pipeline is comprised of first-in-class gene therapies that incorporate both adeno-associated viral vector (AAV) and lentiviral vector (LVV) approaches to gene therapy. 

Rocket’s adeno-associated virus (AAV)-based portfolio targets cardiovascular indications, and includes a late-stage program for Danon disease, a devastating heart failure condition resulting in thickening of the heart, an early-stage program in clinical trials for PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death, and a preclinical program targeting BAG3-associated dilated cardiomyopathy (DCM), a heart failure condition that causes enlarged ventricles. 

Meanwhile, its LVV-based gene therapies target hematologic diseases and consist of late-stage programs for Fanconi anemia (FA), a difficult-to-treat genetic disease that leads to bone marrow failure and potentially cancer, leukocyte adhesion deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections that are frequently fatal, and pyruvate kinase deficiency (PKD), a monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. 

The company has had several significant announcements in recent months: In July, it received Regenerative Medicine Advanced Therapy designation for its gene therapy for PKP2-arrhythmogenic cardiomyopathy; in August, the FDA lifted a clinical hold on its phase 2 trial of its candidate for Danon disease; and in October, the FDA accepted the resubmission of a Biologics License Application for KRESLADI (marnetegragene autotemcel; marne-cel), an LVV-based gene therapy for severe LAD-I.  

Vertex Pharmaceuticals 

• Headquarters: U.S. 
• Latest gene therapy approval: Casgevy for sickle cell disease and beta-thalassemia 
• Recent news: Established a collaboration with Orna Therapeutics to develop gene editing therapies for sickle cell disease and beta-thalassemia 

Vertex is a global biotech company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has several approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases, including cystic fibrosis, sickle cell disease, and transfusion-dependent beta thalassemia. 

Back in 2019, the FDA approved Vertex’s Trikafta for the treatment of cystic fibrosis in people aged 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which is the most common cystic fibrosis-causing mutation. 

Additionally, as mentioned previously, Vertex Pharmaceuticals co-developed the first-ever CRISPR-based gene therapy, Casgevy, with CRISPR Therapeutics for the treatment of sickle cell disease and beta-thalassemia. As part of the deal that was made between the two companies, Vertex leads global development, manufacturing, and commercialization of Casgevy with support from CRISPR Therapeutics.  

Moreover, furthering its gene therapy research for sickle cell disease and beta-thalassemia, Vertex established a collaboration with Orna Therapeutics in January 2025 to utilize Orna’s proprietary LNP delivery solutions to enhance its efforts in developing next-generation gene editing therapies for patients with sickle cell disease and beta-thalassemia.  

Gene therapy: A once-booming field now seeing signs of struggle

Once seen as the next great leap in biotech, the gene therapy field has been struggling of late, plagued by layoffs, restructurings, and company closures. This has even affected major players like Genetix Biotherapeutics, which cut around a quarter of its workforce amid doubts about financial viability, and Sarepta Therapeutics, which laid off hundreds of staff and decided to pivot toward siRNA medicines as its Duchenne muscular dystrophy gene therapy, Elevidys, faced safety issues. Moreover, contract manufacturers like Catalent, which built large facilities to serve the booming market, are also downsizing due to a sudden drop in demand from gene therapy clients. 

These struggles stem from a mix of scientific, commercial, and macroeconomic pressures. Many approved gene therapies carry multimillion-dollar price tags but have seen slow patient uptake, delaying the revenues needed to offset years of high research and development (R&D) and manufacturing costs. At the same time, rising regulatory scrutiny and adverse safety events have cooled investor enthusiasm just as capital markets have tightened, leaving many firms starved of cash. It is ultimately factors like these that have created a challenging environment for a once-surging field that still holds vast long-term potential but faces a sobering short-term reckoning.