Monogenic disease gene therapy companies tackle genetic disorders at their core, aiming to provide long-lasting treatments for single-gene mutations. These firms pioneer innovative solutions using gene editing, AAV vectors, and other advanced technologies to target diseases like hemophilia, retinal disorders, and numerous rare genetic conditions. With growing investment and collaboration, the industry shows promising potential to expand treatment options for patients, offering more effective and personalized healthcare solutions. The accelerated advancements in gene therapy are set to revolutionize healthcare, once considered incurable, opening a pathway to hope for many afflicted by these debilitating conditions.

The companies in this listing vary significantly in size, with many employing between 51 to 1000 people. They are primarily based in the United States, with some emerging firms in China and Europe. Founded between 1999 and 2024, each company specializes in gene therapies targeting specific genetic disorders, ranging from ocular diseases to neurodegenerative and rare genetic conditions. Their innovative approaches reflect a focus on both research and clinical execution, indicating their commitment to patient care and advancing therapeutic frontiers.

Continue reading to learn about the top monogenic disease gene therapy companies.

Top 23 Monogenic Disease Gene Therapy Companies

1. Spark Therapeutics, Inc.

• Website: sparktx.com
• Ownership type: Corporate
• Headquarters: Philadelphia, Pennsylvania, United States (USA)
• Employee distribution: United States (USA) 100%
• Latest funding: $4.3B, February 2019
• Founded year: 2013
• Headcount: 501-1000
• LinkedIn: spark-therapeutics-inc

Spark Therapeutics, Inc., founded in 2013 and based in Philadelphia, Pennsylvania, is a biotechnology company dedicated to developing gene therapy treatments for genetic diseases. The company has a particular focus on conditions such as hemophilia and inherited retinal diseases. Spark aims to transform patient care by providing innovative therapies that offer long-lasting effects. Their operations span research, development, and manufacturing of gene therapies, ensuring that they can deliver effective treatment options to patients and healthcare professionals. Spark is notable for its development of Luxturna, a groundbreaking gene therapy that targets a specific genetic mutation causing vision loss. In 2019, the company secured $4.3 billion in funding, underscoring investor confidence in their mission and capabilities. Spark is also a member of the Roche Group, which enhances its resources and reach in the gene therapy market.

2. Agtc

• Website: agtc.com
• Ownership type: Venture Capital
• Headquarters: Alachua, Florida, United States (USA)
• Employee distribution: United States (USA) 100%
• Latest funding: $23.5M, October 2022
• Founded year: 1999
• Headcount: 51-200
• LinkedIn: agtc

Agtc, known as Beacon Therapeutics, is a biotechnology firm based in Alachua, Florida, specializing in gene therapy aimed at treating retinal diseases. Founded in 1999, the company has developed a pipeline that includes AGTC-501, a gene therapy for X-Linked Retinitis Pigmentosa (XLRP), a severe genetic eye disorder. Beacon Therapeutics is currently conducting clinical trials to evaluate the safety and efficacy of AGTC-501, with a focus on restoring vision for patients suffering from this debilitating condition. The company also explores treatments for other retinal diseases, such as Dry Age-related Macular Degeneration and Cone Rod Dystrophy. In 2022, they secured $23.5 million in funding, which underscores their active role in the biotechnology sector and their commitment to advancing gene therapy solutions.

3. AskBio Inc.

• Website: askbio.com
• Ownership type: Corporate
• Headquarters: Durham, North Carolina, United States (USA)
• Employee distribution: United States (USA) 75%, United Kingdom (UK) 14%, France 6%, Other 5%
• Latest funding: $2.0B, October 2020
• Founded year: 2001
• Headcount: 501-1000
• LinkedIn: askbio

AskBio Inc., also known as Asklepios BioPharmaceutical, Inc., is a biotechnology firm based in Durham, North Carolina. Founded in 2001, the company focuses on gene therapy, utilizing advanced AAV technology to develop innovative treatments for genetic diseases. AskBio collaborates with researchers and healthcare providers to create therapies that address significant unmet medical needs across various genetic conditions. The company has a robust clinical pipeline that includes programs for neuromuscular diseases, central nervous system disorders, and ultra-rare genetic conditions. AskBio has also made notable advancements in gene therapy technology, including the acquisition of Synpromics, a leader in synthetic promoter technology, which enhances their capabilities in gene expression control. In 2020, AskBio secured substantial funding, amounting to $2 billion, to support its research and development efforts.

4. Prevail Therapeutics

• Website: prevailtherapeutics.com
• Ownership type: Corporate
• Headquarters: New York, New York, United States (USA)
• Employee distribution: United States (USA) 100%
• Latest funding: $880.0M, December 2020
• Founded year: 2017
• Headcount: 51-200
• LinkedIn: prevail-therapeutics

Prevail Therapeutics, founded in 2017 and based in New York City, is a biotechnology company dedicated to developing gene therapies aimed at treating neurodegenerative diseases. The company specializes in conditions such as Parkinson's disease, Gaucher disease, and frontotemporal dementia, focusing on disease-modifying treatments that target the genetic underpinnings of these disorders. Prevail employs a precision medicine approach, leveraging advancements in human genetics and AAV-based gene therapy to create therapies that could potentially slow or halt disease progression. The company has initiated clinical trials for its lead programs, PR001 and PR006, which are designed to address the needs of patients with specific genetic mutations. In December 2020, Prevail Therapeutics secured significant funding, amounting to $880 million, which underscores investor confidence in their innovative approach and pipeline. In 2021, Prevail was acquired by Eli Lilly and Company, becoming a wholly owned subsidiary, which further enhances its capabilities and resources in the gene therapy space.

5. EdiGene Inc.

• Website: edigene.com
• Ownership type: Private Equity
• Headquarters: Beijing, Beijing, China
• Employee distribution: China 100%
• Latest funding: Series B, $61.6M, April 2021
• Founded year: 2015
• Headcount: 201-500
• LinkedIn: edigene

EdiGene Inc., founded in 2015 and based in Beijing, China, is a biotechnology firm dedicated to advancing gene editing and therapy solutions. The company specializes in addressing critical unmet medical needs, particularly in the areas of cancer and genetic diseases. EdiGene's innovative platforms, including LEAPER for RNA editing and MAGIC for cell therapy, are designed to transform treatment options for patients. Their product pipeline includes therapies targeting monogenic diseases such as Duchenne Muscular Dystrophy (DMD), Usher Syndrome Type II, and Alpha-1 Antitrypsin Deficiency (AATD). EdiGene has successfully secured funding, with their last round amounting to approximately $61.6 million in April 2021, which supports their ongoing research and development initiatives.

6. SalioGen Therapeutics

• Website: saliogen.com
• Ownership type: Venture Capital
• Headquarters: Lexington, Massachusetts, United States (USA)
• Employee distribution: United States (USA) 100%
• Latest funding: Series B, $115.0M, January 2022
• Founded year: 2020
• Headcount: 51-200
• LinkedIn: saliogen

SalioGen Therapeutics, founded in 2020 and based in Lexington, Massachusetts, is a biotechnology firm dedicated to advancing genetic medicine. The company specializes in developing innovative therapies using its proprietary Gene Coding technology, which enables the integration of large DNA sequences into the genome at specific locations. This technology is particularly relevant for treating genetic disorders such as cystic fibrosis and Stargardt disease. SalioGen's approach avoids the complications associated with viral vectors and double-stranded breaks, making it a promising option for patients and healthcare providers seeking effective treatments. The company has attracted significant investment, securing $115 million in Series B funding in January 2022, which underscores its potential in the genetic medicine space. SalioGen is actively pursuing strategic partnerships to further its gene therapy programs, indicating a commitment to collaboration and growth in the field.

7. American Gene Technologies International Inc.

• Website: americangene.com
• Ownership type: Venture Capital
• Headquarters: Rockville, Maryland, United States (USA)
• Employee distribution: United States (USA) 100%
• Latest funding: Series D, April 2022
• Founded year: 2007
• Headcount: 11-50
• LinkedIn: american-gene-technologies-international-inc

American Gene Technologies International Inc. (AGT) is a biotechnology firm based in Rockville, Maryland, founded in 2007. The company specializes in gene therapy and innovative treatments for infectious diseases, cancers, and genetic disorders. AGT is particularly focused on developing clinical solutions that aim for long-term cures, with notable programs targeting HIV and Phenylketonuria (PKU). Their pipeline includes a one-time treatment for PKU, which is designed to provide a lasting solution for this genetic disorder. AGT conducts clinical trials and collaborates with medical researchers to advance its therapies. The company operates a 27,000 square foot facility that includes specialized laboratories for vector production and translational science. AGT has also received funding through a Series D round, indicating investor confidence in their approach and potential.

8. Castle Creek Biosciences, Inc.

• Website: castlecreekbio.com
• Ownership type: Venture Capital
• Headquarters: Exton, Pennsylvania, United States (USA)
• Employee distribution: United States (USA) 100%
• Latest funding: $75.0M, February 2025
• Founded year: 2020
• Headcount: 11-50
• LinkedIn: castlecreekbio

Castle Creek Biosciences, Inc., founded in 2020 and based in Exton, Pennsylvania, is a biotechnology firm dedicated to developing innovative gene therapies for genetic diseases. The company is particularly focused on dystrophic epidermolysis bullosa (DEB), a severe genetic skin disorder that results from mutations in the COL7A1 gene. Castle Creek operates through in-house manufacturing and is preparing to launch pivotal clinical trials for its lead candidate, D-Fi, which aims to provide functional type VII collagen to affected areas. The company has received $75 million in funding, underscoring its potential and commitment to addressing the needs of patients with limited treatment options. With a robust pipeline and a focus on transformative therapies, Castle Creek is positioned to make significant contributions to the field of gene therapy for genetic diseases.

9. Regel Therapeutics

• Website: regeltherapeutics.com
• Ownership type: Private
• Headquarters: Boston, Massachusetts, United States (USA)
• Employee distribution: United States (USA) 100%
• Latest funding: Other (Grant), $1.0M, September 2023
• Founded year: 2020
• Headcount: 11-50
• LinkedIn: regel-therapeutics

Regel Therapeutics, founded in 2020 and based in Boston, Massachusetts, is a private biotechnology company dedicated to advancing targeted gene therapy for severe genetic diseases. The company leverages its proprietary T3 platform, which combines AAV delivery with innovative technologies to modulate gene expression specifically in affected cells. This targeted approach aims to address the underlying causes of genetic disorders, offering hope for patients with conditions that currently lack effective treatments. Regel's pipeline includes therapies for Dravet syndrome, SCN2A haploinsufficiency, and SYNGAP1 haploinsufficiency, all of which are significant monogenic diseases. In September 2023, Regel Therapeutics secured $1 million in grant funding, underscoring its commitment to research and development in the biotechnology sector.

10. Affinia Therapeutics

• Website: affiniatx.com
• Ownership type: Private Equity
• Headquarters: Waltham, Massachusetts, United States (USA)
• Employee distribution: United States (USA) 100%
• Latest funding: Series B, $110.0M, May 2021
• Founded year: 2019
• Headcount: 51-200
• LinkedIn: touchdown-therapeutics

Affinia Therapeutics, founded in 2019 and based in Waltham, Massachusetts, is a biotechnology firm dedicated to advancing gene therapies. The company specializes in creating targeted treatments for genetic diseases, with a particular focus on conditions affecting the heart and nervous system. Utilizing their proprietary ART (Affinia Rationally-designed Therapies) platform, Affinia employs advanced techniques such as artificial intelligence and structural modeling to design therapies that improve the targeting of genetic payloads. This innovative approach aims to enhance the safety and effectiveness of gene therapies. Affinia has successfully raised $110 million in Series B funding as of May 2021, indicating strong investor confidence in their mission and potential. Their pipeline includes promising candidates for genetic cardiomyopathy and sporadic ALS, showcasing their commitment to addressing significant unmet medical needs in the field of genetic diseases.

11. Astellas Gene Therapies

• Website: astellasgenetherapies.com
• Ownership type: Private
• Headquarters: San Francisco, California, United States (USA)
• Employee distribution: United States (USA) 100%
• Founded year: 2012
• Headcount: 201-500
• LinkedIn: audentes-therapeutics

Astellas Gene Therapies, based in San Francisco, California, is a biotechnology company that specializes in developing gene therapies for rare genetic diseases. Founded in 2012, the company operates as a center of excellence within Astellas Pharma Inc. Their mission is to transform the lives of patients suffering from conditions like X-Linked Myotubular Myopathy and Pompe disease. Astellas Gene Therapies employs a proprietary adeno-associated virus (AAV) platform for drug discovery and has established internal manufacturing capabilities to support their innovative approaches. The company is committed to understanding the needs of patients and families, which informs their research and clinical development strategies. They are currently advancing several gene therapy programs through clinical trials, demonstrating their active role in the gene therapy sector.

12. AnLong Bio

• Website: anlongbio.com
• Ownership type: Venture Capital
• Headquarters: Beijing, Beijing, China
• Employee distribution: China 100%
• Latest funding: Series A, January 2024
• Founded year: 2019
• Headcount: 51-200
• LinkedIn: anlongbio

AnLong Bio, founded in 2019 and based in Beijing, China, is a biotechnology firm dedicated to advancing gene therapy solutions for rare diseases. The company aims to provide accessible and affordable treatments by leveraging a robust network of clinical and manufacturing experts. AnLong Bio has developed platforms for adeno-associated virus (AAV) and RNA interference (RNAi), and they have initiated multiple preclinical projects. Notably, their gene therapy product, AL-001, received approval for clinical trials in early 2023, targeting age-related macular degeneration. With a focus on innovation and a commitment to addressing unmet medical needs, AnLong Bio is positioned to make significant contributions to the field of gene therapy.

13. iECURE, inc.

• Website: iecure.com
• Ownership type: Venture Capital
• Headquarters: Philadelphia, Pennsylvania, United States (USA)
• Employee distribution: United States (USA) 100%
• Latest funding: Series A, $65.0M, November 2022
• Founded year: 2012
• Headcount: 11-50
• LinkedIn: iecure

iECURE, inc. is a biotechnology firm based in Philadelphia, Pennsylvania, founded in 2012. The company specializes in gene therapy and genetic medicine, with a particular emphasis on developing innovative treatments for genetic disorders, especially those related to liver diseases. iECURE employs advanced gene editing techniques to create therapeutics aimed at correcting 'loss of function' diseases. Their primary focus is on rare, monogenic liver disorders, which affect thousands of patients globally, particularly children. iECURE is currently advancing its clinical programs for conditions such as ornithine transcarbamylase (OTC) deficiency, citrullinemia type 1, and phenylketonuria (PKU). The company has established a foundational partnership with the University of Pennsylvania’s Gene Therapy Program, leveraging their expertise in vector engineering and gene therapy development. In November 2022, iECURE secured $65 million in Series A funding, underscoring investor confidence in their mission to provide long-lasting, curative therapies for patients with genetic disorders.

14. HuidaGene Therapeutics

• Website: huidagene.com
• Ownership type: Venture Capital
• Headquarters: Shanghai, Shanghai, China
• Employee distribution: China 88%, United States (USA) 12%
• Latest funding: Series C, May 2022
• Founded year: 2018
• Headcount: 51-200
• LinkedIn: %e8%be%89%e5%a4%a7%ef%bc%88%e4%b8%8a%e6%b5%b7%ef%bc%89%e7%94%9f%e7%89%a9%e7%a7%91%e6%8a%80%e6%9c%89%e9%99%90%e5%85%ac%e5%8f%b8

HuidaGene Therapeutics, founded in 2018 and based in Shanghai, China, is a biotechnology firm dedicated to advancing gene editing and gene therapies. The company specializes in developing innovative treatments for genetic disorders, with a particular emphasis on neurology and ophthalmology. HuidaGene's research and development efforts are aimed at transforming genomic medicine, providing precise and effective therapies for patients with life-threatening conditions. Their pipeline includes several promising candidates targeting monogenic diseases, such as MECP2 Duplication Syndrome and inherited retinal dystrophies caused by RPE65 mutations. The company has made significant strides in its clinical programs, including receiving orphan drug designations from the FDA for some of its therapies. HuidaGene's commitment to addressing the needs of patients globally is evident in its strategic partnerships and ongoing clinical trials.

15. AAVantgarde Bio

• Website: aavantgarde.com
• Ownership type: Venture Capital
• Headquarters: Milan, Lombardy, Italy
• Employee distribution: United Kingdom (UK) 49%, Italy 44%, Switzerland 7%
• Latest funding: Series A, $65.3M, June 2023
• Founded year: 2021
• Headcount: 11-50
• LinkedIn: aavantgarde-bio

AAVantgarde Bio is a clinical-stage biotechnology company based in Milan, Italy, founded in 2021. The company specializes in gene therapy, focusing on innovative treatments for inherited retinal diseases. AAVantgarde has developed two proprietary adeno-associated viral (AAV) vector platforms aimed at addressing the challenges associated with gene delivery, particularly for larger genes that exceed the typical capacity of AAV vectors. Their current pipeline includes programs targeting Usher syndrome type 1B and Stargardt disease, both of which have significant unmet medical needs. AAVantgarde is actively involved in clinical trials to validate their therapies, showcasing their commitment to advancing treatment options for patients with genetic disorders. The company has successfully raised €61 million in Series A funding, which will support the development of their therapeutic programs and further their mission to provide effective solutions for underserved patient populations.

16. Forge Biologics

• Website: forgebiologics.com
• Ownership type: Corporate
• Headquarters: Columbus, Ohio, United States (USA)
• Employee distribution: United States (USA) 99%, Other 1%
• Latest funding: $620.0M, November 2023
• Founded year: 2020
• Headcount: 201-500
• LinkedIn: forge-biologics

Forge Biologics, founded in 2020 and based in Columbus, Ohio, is a biotechnology firm focused on the development and manufacturing of gene therapies. The company provides comprehensive manufacturing services for AAV gene therapies and plasmid DNA, catering to pharmaceutical and biotech companies aiming to bring innovative treatments for rare diseases to market. Their operations are conducted in a state-of-the-art cGMP compliant facility, ensuring adherence to high production standards. Notably, Forge is developing FBX-101, a gene therapy targeting Krabbe Disease, which highlights their commitment to addressing monogenic diseases. With a recent funding round of $620 million completed in November 2023, Forge Biologics is positioned to accelerate its efforts in the gene therapy space.

17. Amarna Therapeutics

• Website: amarnatherapeutics.com
• Ownership type: Venture Capital
• Headquarters: Leiden, South Holland, Netherlands
• Employee distribution: Netherlands 88%, Spain 12%
• Latest funding: Other (Grant), January 2022
• Founded year: 2008
• Headcount: 11-50
• LinkedIn: amarna-therapeutics-b.v.

Amarna Therapeutics, founded in 2008 and based in Leiden, South Holland, is a biotechnology firm specializing in gene therapy. The company is dedicated to developing innovative treatments for a range of diseases, including Type 1 Diabetes and Hemophilia, utilizing its proprietary Nimvec™ platform. This platform is designed to deliver transgenes effectively and non-immunogenically, which is crucial for the success of gene therapies. Amarna Therapeutics is actively working on a pipeline that includes therapies for monogenic diseases, autoimmune disorders, and chronic inflammation. The company has a small team of around 17 employees and has received grants for its research initiatives, indicating ongoing support for its innovative projects. Their recent interactions with regulatory bodies, such as the FDA, highlight their commitment to advancing their gene therapy candidates toward clinical application.

18. Eidos Therapeutics

• Website: eidostx.com
• Ownership type: Corporate
• Headquarters: San Francisco, California, United States (USA)
• Employee distribution: United States (USA) 100%
• Latest funding: $175.0M, October 2020
• Founded year: 2016
• Headcount: 11-50
• LinkedIn: eidos-therapeutics

Eidos Therapeutics, based in San Francisco, California, is a biotechnology firm founded in 2016. The company is dedicated to developing transformative medicines for genetic diseases. Their primary focus is on transthyretin amyloidosis (ATTR) and autosomal dominant hypocalcemia type 1 (ADH1). Eidos is advancing Acoramidis, an investigational small molecule aimed at stabilizing the TTR protein, which is crucial for patients suffering from ATTR. This condition is often underdiagnosed and can lead to severe health complications. Eidos is also involved in the development of BBP-812, a gene therapy targeting Canavan disease, which highlights their commitment to addressing rare genetic disorders. The company has received significant funding, with a reported amount of $175 million in October 2020, which supports their ongoing research and development efforts.

19. Arbor Biotechnologies

• Website: arbor.bio
• Ownership type: Private Equity
• Headquarters: Cambridge, Massachusetts, United States (USA)
• Employee distribution: United States (USA) 100%
• Latest funding: Series C, $73.9M, March 2025
• Founded year: 2016
• Headcount: 51-200
• LinkedIn: arborbio

Arbor Biotechnologies, founded in 2016 and based in Cambridge, Massachusetts, is a biotechnology company dedicated to advancing genetic medicines. The firm specializes in innovative gene editing technologies, particularly programmable DNA editors, aimed at providing tailored solutions for genetic diseases. Arbor serves healthcare providers and research organizations within the biotechnology sector, focusing on the development of curative therapies. The company has made significant strides in its research and development efforts, evidenced by its recent Series C funding round, which raised $73.9 million in March 2025. This funding supports their mission to create effective treatments for genetic disorders, particularly in the areas of liver and central nervous system diseases. Arbor's pipeline reflects a commitment to addressing the complexities of genetic diseases, making them a notable entity in the gene therapy landscape.

20. Rampart Bioscience

• Website: rampartbio.com
• Ownership type: Venture Capital
• Headquarters: Monrovia, California, United States (USA)
• Employee distribution: United States (USA) 100%
• Latest funding: Series A, $85.0M, October 2023
• Founded year: 2019
• Headcount: 1-10
• LinkedIn: rampart-bioscience

Rampart Bioscience, founded in 2019 and based in Monrovia, California, is a biotechnology firm dedicated to developing innovative DNA-based medicines aimed at treating genetic diseases. The company is particularly focused on creating safe and effective gene therapies that address significant unmet medical needs. With a team of experienced professionals in biotechnology and gene therapy, Rampart is committed to reshaping treatment options for patients with genetically driven diseases. Their proprietary HALO platform is designed to produce potent, durable, and redosable therapies. Currently, Rampart is advancing its lead program targeting hypophosphatasia, a rare genetic disorder that affects bone mineralization. In October 2023, Rampart announced an $85 million Series A financing round, which underscores the confidence investors have in their mission and technology. This funding will support their ongoing efforts to develop novel gene therapies that can significantly improve patient outcomes.

21. Capsida Biotherapeutics

• Website: capsida.com
• Ownership type: Venture Capital
• Headquarters: Thousand Oaks, California, United States (USA)
• Employee distribution: United States (USA) 100%
• Latest funding: February 2023
• Founded year: 2019
• Headcount: 51-200
• LinkedIn: capsida-biotherapeutics

Capsida Biotherapeutics, founded in 2019 and based in Thousand Oaks, California, is a biotechnology firm dedicated to advancing gene therapy. The company specializes in creating targeted, non-invasive therapies for conditions such as genetic epilepsy and Parkinson's disease. Utilizing their proprietary capsid engineering technology, Capsida aims to enhance the delivery and efficacy of gene therapies. Their pipeline includes programs specifically designed for genetic epilepsy linked to STXBP1 mutations and Parkinson's disease associated with GBA mutations. Capsida's integrated approach combines research and development with manufacturing capabilities, allowing them to streamline the process of bringing therapies to patients. They have established partnerships with notable companies like AbbVie and CRISPR Therapeutics, further solidifying their position in the industry. As of February 2023, they have not reported any recent funding, but their ongoing projects and partnerships indicate a robust operational framework.

22. Carbon Biosciences

• Website: carbonbio.com
• Ownership type: Venture Capital
• Headquarters: Boston, Massachusetts, United States (USA)
• Employee distribution: United States (USA) 100%
• Latest funding: Series A, $38.0M, June 2022
• Founded year: 2021
• Headcount: 11-50
• LinkedIn: c12bio

Carbon Biosciences, founded in 2021 and based in Boston, Massachusetts, is a biotechnology firm dedicated to advancing gene therapy and genetic medicines. The company aims to develop innovative treatments for genetic diseases by harnessing the power of natural viral evolution. Their proprietary CBN vector library is designed to offer advantages such as enhanced tissue specificity and the ability to deliver larger payloads, which are critical for effective gene therapy. Carbon Biosciences has attracted significant investment, securing $38 million in Series A funding in June 2022, which underscores investor confidence in their approach. The company is actively working on a gene therapy candidate for cystic fibrosis, highlighting their commitment to addressing monogenic diseases. Their primary customers include healthcare providers and pharmaceutical companies seeking advanced therapeutic solutions.

23. Entos Pharmaceuticals

• Website: entospharma.com
• Ownership type: Private
• Headquarters: Edmonton, Alberta, Canada
• Employee distribution: United States (USA) 51%, Canada 49%
• Latest funding: Other (Grant), $4.0M, January 2025
• Founded year: 2016
• Headcount: 11-50
• LinkedIn: entospharmaceuticals

Entos Pharmaceuticals, founded in 2016 and based in Edmonton, Alberta, Canada, is a private biotechnology firm specializing in genetic medicines. The company leverages its proprietary Fusogenix PLV™ technology to create innovative therapies aimed at treating genetic diseases. Their focus is on the research and development of nucleic acid therapies, which are essential for addressing various genetic disorders. Entos has a robust pipeline that includes programs for conditions like Stargardt disease and cystic fibrosis, showcasing their commitment to tackling significant health challenges. The company has received funding, including a recent grant of $4 million in January 2025, which supports their ongoing research efforts. With a team of approximately 45 employees, Entos operates in both Canada and the United States, collaborating with healthcare providers and researchers to advance patient care.

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