Top 28 Firms in Pulmonary Gene Therapy Sector
Top 28 Firms in Pulmonary Gene Therapy Sector
The biotechnology industry that these companies operate in is chiefly engaged with gene therapy applications for pulmonary diseases. Comprised largely by therapeutic and biotechnological firms, like CRISPR Therapeutics, Intellia Therapeutics, and Sangamo Therapeutics, this industry pioneers the development of innovative treatments and cures. Stitching together the threads of biology and technology, these companies utilize cutting-edge techniques such as CRISPR/Cas9 gene editing and zinc finger technology. By intervening at the genetic level they offer the potential to not only manage but eventually cure a range of serious lung diseases. Given the rapidly evolving scientific landscape, this industry promises significant advancements in the near future.
Top 28 Pulmonary Disease Gene Therapy Companies
1. CRISPR Therapeutics
- Website: crisprtx.com
- Headquarters: Cambridge, Massachusetts, United States
- Founded: 2013
- Headcount: 201-500
- Latest funding type: Grant
CRISPR Therapeutics is a company that specializes in gene editing and cell therapies. They use the CRISPR/Cas9 therapeutic approach to treat genetically-defined diseases by editing cells inside the body.
2. Intellia Therapeutics, Inc.
- Website: intelliatx.com
- Headquarters: Cambridge, Massachusetts, United States
- Founded: 2014
- Headcount: 201-500
- Latest funding type: Post Ipo Equity
Intellia Therapeutics is a biotechnology company focused on developing CRISPR/Cas9 gene editing therapies. They are dedicated to transforming the lives of patients suffering from genetic diseases. Intellia Therapeutics is committed to using their expertise in genome editing to create innovative treatments.
3. Sangamo Therapeutics, Inc.
- Website: sangamo.com
- Headquarters: Brisbane, California, United States
- Founded: 1995
- Headcount: 201-500
- Latest funding type: Post Ipo Equity
Sangamo is a biotechnology company that specializes in using zinc finger technology to develop genomic cures for severe diseases. Their expertise allows them to create treatments for patients who currently only have access to symptom management. They have a strong commitment to patient care and offer resources and support for both patients and healthcare professionals.
4. Forge Biologics
- Website: forgebiologics.com
- Headquarters: Columbus, Ohio, United States
- Founded: 2020
- Headcount: 51-200
- Latest funding type: Series C
Forge Biologics is a company focused on developing innovative gene therapies to treat patients with devastating rare diseases. They offer end-to-end manufacturing services, from preclinical to commercial, to accelerate gene therapy programs. Their team of diverse and passionate individuals trailblazes a new model for making medicines. They specialize in AAV gene therapies and operate a state-of-the-art GMP facility called 'the Hearth.'
5. Jaguar Gene Therapy
- Website: jaguargenetherapy.com
- Headquarters: Lake Forest, Illinois, United States
- Founded: 2019
- Headcount: 51-200
- Latest funding type: Series Unknown
Jaguar Gene Therapy is a company that specializes in gene therapy and aims to accelerate breakthroughs in treating severe genetic diseases. They have a team of experts who develop and manufacture gene therapies, leveraging their expertise and cutting-edge technologies to deliver safe and effective treatments. Their mission is to provide hope and improved quality of life for patients and their families.
6. Stoke Therapeutics
- Website: stoketherapeutics.com
- Headquarters: Bedford, Massachusetts, United States
- Founded: 2017
- Headcount: 51-200
- Latest funding type: Post Ipo Equity
Stoke Therapeutics is a biotechnology company that is focused on addressing severe diseases by upregulating protein expression with RNA-based medicines. Their goal is to develop the first disease-modifying therapy for Dravet syndrome, a severe and progressive genetic epilepsy. With their proprietary research platform called TANGO, Stoke aims to restore missing proteins by increasing protein output from healthy genes.
7. Tevard Biosciences
- Website: tevard.com
- Headquarters: Cambridge, Massachusetts, United States
- Headcount: 11-50
Tevard Biosciences is a biotech company that specializes in developing tRNA-based therapies to cure rare genetic diseases. They focus on creating innovative gene therapy approaches to treat conditions like Dravet syndrome and other genetic epilepsies. Their unique platforms, developed by scientific co-founders Dr. Jeff Coller and Dr. Chris Ahern, have the potential to provide durable cures for many rare diseases caused by haploinsufficiency and nonsense mutations.
8. Expression Therapeutics, Inc.
- Website: expressiontherapeutics.com
- Headquarters: Tucker, Georgia, United States
- Headcount: 11-50
Expression Therapeutics is a clinical stage gene and cell therapy company focused on developing potentially curative therapeutics for deadly childhood and adult genetic diseases. They utilize a multi-platform approach and proprietary non-signaling chimeric antigen receptor technology to enhance targeting capability and alleviate concerns of long-term T cell ablation and tonic signaling. Their clinical programs use lentiviral and adeno-associated viral vectors.
9. Charcot-Marie-Tooth Association
- Website: cmtausa.org
- Headquarters: Glenolden, Pennsylvania, United States
- Founded: 1983
- Headcount: 11-50
The Charcot-Marie-Tooth Association (CMTA) is a nonprofit organization dedicated to funding research for a cure for Charcot-Marie-Tooth disease (CMT). They support clinical trials, gene therapy, and other research initiatives in order to find treatments and improve the lives of individuals with CMT.
10. Neural Stem Cell Institute
- Website: neuralsci.org
- Headquarters: Rensselaer, New York, United States
- Founded: 2007
- Headcount: 11-50
Neuralsci.org is a company focused on neurological research and cancer treatment. They specialize in the study of neural stem cells and the development of innovative therapies for various types of cancer, including lung cancer, thyroid cancer, and neuroblastoma.
11. ATP (Apple Tree Partners)
- Website: appletreepartners.com
- Headquarters: New York, New York, United States
- Founded: 1999
- Headcount: 11-50
- Latest funding type: Undisclosed
Apple Tree Partners is a pioneer in life sciences venture capital and invests in transformative biotechnology companies. They provide flexible capital, strategic insight, and operational resources to build sustainable, research-driven enterprises.
12. Epistemic AI
- Website: epistemic.ai
- Headquarters: New York, Connecticut, United States
- Founded: 2018
- Headcount: 11-50
- Latest funding type: Seed
Epistemic AI (EAI) is a company that offers a Research Intelligence platform to help researchers access relevant documents and data related to diseases, their diagnosis, treatments, and biological functions. The platform utilizes artificial intelligence to provide insights into the underlying relationships among diseases and facilitate research processes.
13. Invivo Capital
- Website: invivo.capital
- Headquarters: Barcelona, Barcelona, Spain
- Founded: 2019
- Headcount: 1-10
Invivo Capital Partners promoted by Albert Ferrer and Lluís Pareras, is a management company investing in early stages companies in the healthcare sector. Invivo Capital Partners has a team of highly qualified professionals, with the scientific, technical and financial know-how required to analyse and make investments to provide value to the invested company. It also has a network of contacts and key strategic relationships in the health ecosystem. With the experience gained through the first fund Healthequity SCR, the management team has raised a second fund, called Invivo Ventures FCR. Invivo Ventures FCR has been registered to the CNMV in March 2019, it has achieved a first closing of 40M €, including European Investment Fund (EIF), Instituto de Crédito Oficial (ICO), Institut Català de Finances (ICF) and other private investors.
14. Viralgen Vector Core
- Website: viralgenvc.com
- Headquarters: San Sebastián, Gipuzkoa, Spain
- Founded: 2017
- Headcount: 51-200
Viralgen is a company specializing in AAV manufacturing, development, and validation. With extensive expertise in AAV and its regulations, they offer comprehensive services throughout the entire process. Their operations include in-house QC lab for critical release assays, regulatory support, and program management. The company's customers could include pharmaceutical companies, biotech startups, and research institutions. Viralgen can be categorized as a biotech manufacturing company. Their offering keywords include AAV manufacturing, development, validation, and QC lab. Their business model keywords include Manufacturing, Consulting, and Biotech. Relevant categories for the industry could be Gene Therapy, Pharmaceutical, and Biotechnology. The company's customers likely include pharmaceutical companies, biotech startups, and research institutions. Other relevant keywords could be Gene Therapy, Cell-based Therapy, and Genetic Diseases.
15. Avrobio
- Website: avrobio.com
- Headquarters: Cambridge, Massachusetts, United States
- Founded: 2015
- Headcount: 51-200
- Latest funding type: Ipo
AVROBIO is a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease. They are focused on targeting lysosomal disorders and developing personalized therapies for patients. Their goal is to enable individuals with genetic diseases to live longer and free from disease progression.
16. Mustang Bio
- Website: mustangbio.com
- Headquarters: Worcester, Massachusetts, United States
- Founded: 2015
- Headcount: 51-200
- Latest funding type: Post Ipo Debt
Mustang Bio is a clinical-stage biopharmaceutical company focused on translating medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors, and rare genetic diseases. They aim to acquire rights to these technologies, fund research and development, and bring the therapies to market. Mustang has collaborations with top medical institutions for developing CAR T therapies and lentiviral gene therapy. They are registered under the Securities Exchange Act of 1934 and founded by Fortress Biotech.
17. Chroma Medicine
- Website: chromamedicine.com
- Headquarters: Cambridge, Massachusetts, United States
- Founded: 2021
- Headcount: 51-200
Chroma Medicine is a genomic medicine company that is developing a new class of therapeutics for precise control of gene expression. They aim to revolutionize treatment of diseases by harnessing epigenetics, nature's mechanism for gene regulation.
18. ToolGen Inc.
- Website: toolgen.com
- Headquarters: Seoul, Kyonggi-Do, Korea (the Republic of)
- Founded: 1999
- Headcount: 51-200
- Latest funding type: Corporate Round
ToolGen is a biotechnology company specializing in therapeutics development, with a focus on gene correction for the treatment of genetic diseases and the enhancement of traits in plants and animals. They possess a patented genome editing platform and offer cutting-edge genome editing technologies for a range of applications.
19. Arbor Biotechnologies
- Website: arbor.bio
- Headquarters: Cambridge, Massachusetts, United States
- Founded: 2016
- Headcount: 11-50
- Latest funding type: Series B
Arbor Biotechnologies is a biotechnology company focused on pioneering gene editing technology to develop functional cures for genetic diseases, with a specific emphasis on liver and CNS diseases.
20. Tune Therapeutics
- Website: tunetx.com
- Headquarters: Durham, North Carolina, United States
- Founded: 2020
- Headcount: 11-50
- Latest funding type: Series A
Tune Therapeutics is a biotech company that leverages its proprietary TEMPO platform to manipulate gene expression and reverse pathways of cancer, genetic diseases, and aging. By targeting multiple genes simultaneously, Tune Therapeutics can change cell fate and function without permanently altering DNA. With an accomplished team of scientists and biotech executives, Tune Therapeutics aims to revolutionize disease treatment and pave the way for personalized medicine.
21. Life Edit Therapeutics
- Website: lifeeditinc.com
- Headquarters: Research Triangle Park, North Carolina, United States
- Founded: 2017
- Headcount: 11-50
- Latest funding type: Acquired
Life Edit Inc. is a company that specializes in gene editing therapies for patients with genetic diseases. They are dedicated to making innovative and curative treatments accessible to those in need. With a focus on discovery and unlimited potential, they aim to rewrite the future of gene editing by providing versatile options for targeting genetic disorders.
22. Excision BioTherapeutics Inc.
- Website: excisionbio.com
- Headquarters: Philadelphia, Pennsylvania, United States
- Founded: 2015
- Headcount: 11-50
Excision BioTherapeutics is a biotech company specializing in gene editing therapies. They offer innovative solutions for treating genetic diseases and improving patient outcomes.
23. Juniper Biologics
- Website: juniperbiologics.com
- Headquarters: Singapore, Central Region, Singapore
- Headcount: 11-50
Juniper Biologics is a pharmaceutical company specializing in gene therapy and oncology. They offer innovative treatments for rare diseases and provide supportive care for patients. With a focus on accessibility and ESG commitments, their mission is to improve the health and quality of life of patients everywhere.
24. Ascidian Therapeutics
- Website: ascidian-tx.com
- Headquarters: Boston, Massachusetts, United States
- Founded: 2020
- Headcount: 11-50
- Latest funding type: Series A
Ascidian Therapeutics is a biotechnology company revolutionizing the treatment of genetic diseases through RNA exon editing. Their innovative platform enables the replacement of multiple contiguous exons, providing a powerful and versatile RNA therapeutic approach. By leveraging designer RNA, Ascidian's technology offers the durability of gene therapy without the risks of direct DNA editing. They are redefining the treatment landscape by editing genes at the RNA level, resulting in full-length functional proteins.
25. Code Biotherapeutics
- Website: codebiotx.com
- Headquarters: Hatfield, Pennsylvania, United States
- Founded: 2020
- Headcount: 11-50
- Latest funding type: Series A
Code Biotherapeutics is a company actively engaged in establishing partnerships for the development of genetic medicines to treat rare and prevalent diseases. They are focused on leveraging their proprietary DNA delivery platform, 3DNA®, to overcome challenges in delivering genetic medicines and potentially cure life-threatening diseases.
26. Modalis Therapeutics
- Website: modalistx.com
- Headquarters: Cambridge, Massachusetts, United States
- Founded: 2016
- Headcount: 11-50
- Latest funding type: Ipo
Modalistx.com is a company that provides business solutions and services. They specialize in offering a range of products and services to help businesses optimize their operations and achieve their goals. With their expertise in various industries, they provide customized solutions tailored to meet the specific needs of each client. Their focus is on delivering high-quality products and services that drive growth, improve efficiency, and enhance customer satisfaction.
27. Mosaic TX
- Website: mosaic-tx.com
- Headquarters: Hinxton, Cambridgeshire, United Kingdom
- Founded: 2020
- Headcount: 11-50
Mosaic is a biotechnology company that develops and delivers transformative therapies for genetic diseases.
28. Rznomics Inc
- Website: rznomics.com
- Headquarters: 성남시, 경기도, Korea (the Republic of)
- Founded: 2017
- Headcount: 11-50
- Latest funding type: Series C
Rznomics is a biotech company that aims to develop gene therapy drugs using RNA-based platforms to address various unmet medical needs, particularly for cancer, degenerative, and genetic diseases.
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