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REGENXBIO
Rockville, United States
B
251-500 Employees
2009
Key takeaway
REGENXBIO is focused on advancing in vivo gene therapy through its innovative AAV gene therapies, aiming to provide one-time treatments for various diseases. Their commitment to research and development in this field highlights their role as pioneers in gene therapy.
Highlighted product
Core business
Pioneers in Gene Therapy
SIRION BIOTECH
Planegg, Germany
A
11-50 Employees
2007
Key takeaway
SIRION Biotech specializes in optimizing adeno-associated vectors (AAV) and lentiviral vectors, which are crucial for in vivo gene therapy applications. With over 15 years of experience in viral vector development and a focus on therapeutic expression cassette optimization, the company supports the entire gene therapy development process from R&D to GMP production.
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Product
Cell Therapy (LV Technologies)
Lentiviral vectors integrate stably into the genome and are thus a reliable tool for ex vivo gene therapy or cell therapy.
Gene Therapy Review
City of Edinburgh, United Kingdom
A
1-10 Employees
2008
Key takeaway
The Gene Therapy Review is a leading resource for information on in vivo gene therapy, detailing the process of transferring genetic material to dysfunctional cells to correct deficiencies in a patient's DNA. This site, established in 2007, serves as a comprehensive hub for the life sciences research community.
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Core business
Home | Gene Therapy Review
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GeneWerk
Heidelberg, Germany
A
11-50 Employees
2014
Key takeaway
GeneWerk, now known as ProtaGene, specializes in gene therapy safety solutions, particularly addressing insertional mutagenesis and genotoxicity. Their expertise is crucial for developing safer gene therapies, making them a valuable partner in the field of cell and gene therapy development.
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Core business
Gene Therapy | Insertional Mutagenesis | Genotoxicity
GeneWerk are providers of gene therapy safety solutions focusing on insertional mutagenesis and genotoxicity to bring breakthrough therapies to clinics.
ViGeneron
Starnberg, Germany
A
1-10 Employees
2017
Key takeaway
ViGeneron is focused on delivering innovative gene therapies, particularly for ophthalmic diseases with high unmet medical needs. Their pipeline includes programs targeting inherited retinal diseases, utilizing proprietary adeno-associated virus (AAV) technology platforms.
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Core business
ViGeneron - The next generation gene therapy companyViGeneron
Nanocell Therapeutics
Utrecht, Netherlands
A
1-10 Employees
-
Key takeaway
NanoCell Therapeutics is focused on revolutionizing gene therapy by developing in vivo approaches that eliminate the need for ex-vivo cell manipulations. Their non-viral, DNA-based technology platform aims to transform adoptive cell therapies into more effective gene therapies, ensuring patients have access to innovative treatment options.
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Core business
NanoCell Therapeutics – Transforming cell & gene therapy
WhiteLab Genomics
Paris, France
A
11-50 Employees
2019
Key takeaway
WhiteLab Genomics focuses on accelerating gene therapy developments through advanced AI software solutions that optimize the design of payloads and vectors. This innovative approach aims to enhance the effectiveness of treatments that introduce functional genes to correct genetic disorders.
Highlighted product
Core business
Gene and Cell Therapy | AI Software Solutions | WhiteLab Genomics
At WhiteLab Genomics we develop state of the art software solutions using AI in order to accelerate and de-risk gene and cell therapy developments.
Be The Match BioTherapies
Minneapolis, United States
B
1001-5000 Employees
2016
Key takeaway
Be The Match BioTherapies is dedicated to providing comprehensive support for both allogeneic and autologous cell and gene therapy development. With over 35 years of experience, they empower the discovery, development, and delivery of next-generation therapies, ensuring that patients receive the critical treatments they need.
Highlighted product
Product
Cell and Gene Therapy Solutions | Be The Match BioTherapies
Be The Match BioTherapies helps you mitigate risk with allogeneic or autologous cell and gene therapy solutions that scale with you at every step.
Innovation Hubs for Gene Therapies
London, United Kingdom
A
11-50 Employees
2021
Key takeaway
The company focuses on manufacturing essential vectors for gene therapy trials, including lentivirus and adeno-associated virus (AAV). With the potential of gene therapies to treat various conditions and a strong research base in the UK, advancements in this field are rapidly progressing towards making these therapies a standard in healthcare.
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Core business
Capabilities :: Innovation Hubs for Gene Therapies
Vivet Therapeutics
Paris, France
A
11-50 Employees
2016
Key takeaway
VTX-801 is an innovative investigational gene therapy developed by Vivet Therapeutics for Wilson Disease, utilizing a rAAV-based vector to deliver a miniaturized ATP7B transgene that helps restore copper homeostasis and reduce copper accumulation. This therapy has received Orphan Drug Designation and Fast Track designation, highlighting its potential in gene therapy advancements.
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Core business
Our DNA - Vivet Therapeutics
Technologies which have been searched by others and may be interesting for you:
In vivo gene therapy refers to the direct delivery of therapeutic genes into the cells of a living organism to treat genetic disorders or diseases. This approach involves using vectors, often viral, to transport the desired genetic material into target cells. The primary goal is to correct or replace defective genes, thereby addressing the root cause of the disease. One of the significant advantages of in vivo gene therapy is its ability to target specific tissues or organs, allowing for localized treatment. This method can lead to long-lasting effects, as the introduced genes can potentially remain active within the host cells, offering a more durable solution compared to conventional treatments.
In vivo gene therapy involves the direct delivery of genetic material into a patient's cells within their body. This process typically utilizes vectors, which can be modified viruses or non-viral systems, to transport therapeutic genes to specific tissues. Once inside the target cells, the introduced genes can either replace faulty genes, provide new functions, or produce proteins that can help combat diseases. The effectiveness of in vivo gene therapy relies on several factors, including the choice of vector, the method of delivery, and the specific genetic target. By precisely targeting affected tissues, this approach aims to correct the underlying genetic problems associated with various conditions, offering potential treatments for genetic disorders, certain types of cancer, and other diseases.
1. Targeted Treatment
In vivo gene therapy allows for the direct delivery of therapeutic genes into specific tissues or organs, enabling targeted treatment of diseases at their source. This approach minimizes the risk of systemic side effects compared to other methods.
2. Long-lasting Effects
By introducing genetic material directly into the body, in vivo gene therapy can potentially provide long-lasting effects. Once the therapeutic gene is integrated into the patient’s cells, it can continue to produce the necessary proteins, offering a sustained treatment solution.
3. Broad Applicability
This method can address a wide range of genetic disorders, including rare diseases, inherited conditions, and some forms of cancer. Its versatility makes it a promising option for various therapeutic areas.
4. Reduced Need for Frequent Treatments
In vivo gene therapy often reduces the frequency of treatments required. Unlike traditional therapies that may need regular dosing, a single or limited number of administrations can lead to significant, prolonged benefits.
In vivo gene therapy has shown promise in treating a variety of diseases, particularly genetic disorders, cancers, and certain viral infections.
1. Genetic Disorders
Conditions like cystic fibrosis, hemophilia, and muscular dystrophy can be addressed by delivering corrective genes directly into the patient's body, enabling the production of functional proteins.
2. Cancers
Some forms of cancer, including leukemia and solid tumors, are being targeted through in vivo approaches that aim to modify the patient's immune cells or directly introduce therapeutic genes to inhibit tumor growth.
3. Viral Infections
In vivo gene therapy has potential applications in treating viral infections such as HIV, where strategies involve editing the genes of infected cells to eliminate the virus or enhance immune responses.
The versatility of this therapy offers hope for developing effective treatments across a wide range of medical conditions.
In Vivo Gene Therapy carries several risks that must be carefully considered. One significant concern is the potential for an immune response. The introduction of new genetic material can trigger the immune system, leading to inflammation or other adverse reactions. Additionally, there is a risk of off-target effects, where the therapy unintentionally alters genes other than the intended target, potentially causing harmful consequences. Another risk involves the delivery method. Various vectors, such as viral vectors, are used to transport the genetic material into the patient's cells. These vectors can sometimes integrate into the wrong location in the genome, leading to disruptions in normal cellular function or even oncogenic transformations. Monitoring and managing these risks are crucial for the safe application of In Vivo Gene Therapy.
Some interesting numbers and facts about your company results for In Vivo Gene Therapy
| Country with most fitting companies | United States |
| Amount of fitting manufacturers | 6990 |
| Amount of suitable service providers | 4827 |
| Average amount of employees | 11-50 |
| Oldest suiting company | 2007 |
| Youngest suiting company | 2021 |
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Some interesting questions that has been asked about the results you have just received for In Vivo Gene Therapy
What are related technologies to In Vivo Gene Therapy?
Based on our calculations related technologies to In Vivo Gene Therapy are Biomedical (Red), Bioinformatics (Gold), Environmental Biotechnology (Grey), Agricultural Biotechnology (Green), Food Related Biotechnology (Yellow)
Who are Start-Ups in the field of In Vivo Gene Therapy?
Start-Ups who are working in In Vivo Gene Therapy are Innovation Hubs for Gene Therapies
Which industries are mostly working on In Vivo Gene Therapy?
The most represented industries which are working in In Vivo Gene Therapy are Biotechnology, Other, Medical, Healthcare, Pharmaceuticals
How does ensun find these In Vivo Gene Therapy Companies?
ensun uses an advanced search and ranking system capable of sifting through millions of companies and hundreds of millions of products and services to identify suitable matches. This is achieved by leveraging cutting-edge technologies, including Artificial Intelligence.
