Gina Johnson
Precigen is a biopharmaceutical company that develops precision immunology treatments through gene and cell therapies. Their UltraCAR-T platform is a gene therapy that utilizes the non-viral Sleeping Beauty system to reprogram immune cells by inserting specific DNA sequences into the genome. The Sleeping Beauty system offers advantages such as ease of manufacturing, stability for storage, and lack of immunogenicity. Precigen has optimized the system to enhance gene elements, genetic payload capacity, and efficiency. The UltraCAR-T platform has the potential to revolutionize CAR-T treatment by increasing patient access through rapid and lower-cost manufacturing, and improved outcomes using advanced technologies for precise tumor targeting and immune system control.
| Characteristics | Values |
|---|---|
| Type | Non-viral transposon/transposase system |
| Function | Reprogram immune cells by inserting specific DNA sequences into the genome |
| Advantages | Ease and low cost of manufacturing, stability for longer-term storage, lack of immunogenicity once inside host cells |
| Use in UltraCAR-T platform | Simultaneously express antigen-specific CAR, kill switch, and mbIL15 genes |
| UltraCAR-T platform advantages | Rapid manufacturing, lower manufacturing-related costs, improved outcomes using advanced technologies for precise tumor targeting and control of the immune system |
| AdenoVerse platform | Efficient gene delivery of vaccine antigens and cytokines to modulate the immune system |
| AdenoVerse platform advantages | High payload capacity, amenable to repeated patient administration, superior performance characteristics, high-yield manufacturing |
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What You'll Learn
- Precigen's Sleeping Beauty is a non-viral gene transfer system
- It modifies T cells to target neoantigens
- Sleeping Beauty is used in UltraCAR-T, a multigenic autologous CAR-T platform
- The system has been optimised using UltraVector DNA construction
- Sleeping Beauty has advantages such as ease of manufacturing and stability
Precigen's Sleeping Beauty is a non-viral gene transfer system
Precigen is a biopharma company that works on precision immunology through novel gene and cell therapies. Their UltraCAR-T platform is a multigenic autologous CAR-T platform that utilizes the company's advanced non-viral Sleeping Beauty system. This system is used to simultaneously express an antigen-specific CAR, kill switch, and mbIL15 genes. The Sleeping Beauty system is a non-viral gene transfer system that stably reprograms immune cells by inserting specific DNA sequences into the genome.
The non-viral Sleeping Beauty system has been optimized using Precigen's UltraVector® DNA construction platform to deliver a large multigenic payload at high efficiency. This results in UltraCAR-T cells that are precision-engineered to produce a homogeneous cell product. The UltraCAR-T platform is differentiated from the competition by increasing patient access through rapid manufacturing, lower manufacturing-related costs, and improved outcomes using advanced technologies for precise tumor targeting and control of the immune system.
The Sleeping Beauty system brings several advantages, including the ease and relatively low cost of manufacturing, stability for longer-term storage, and a lack of immunogenicity once inside host cells. Precigen has made improvements to the system by optimizing gene elements, genetic payload capacity, and efficiency. The system enables high-level and stable gene transfer and contains the cell membrane-bound form of interleukin-15, which has been shown to promote the survival of immune Natural Killer (NK) cells with superior anti-tumor properties.
The AdenoVerse platform, another one of Precigen's innovations, utilizes a library of proprietary adenovectors for efficient gene delivery of vaccine antigens and cytokines to modulate the immune system. Their proprietary gorilla adenovectors have high payload capacity and are amenable to repeated patient administration.
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It modifies T cells to target neoantigens
Precigen's Sleeping Beauty system is a non-viral gene transfer system that modifies T cells to target neoantigens. It is a novel gene and cell therapy that works in combination to achieve efficacy and safety. The system is designed to simultaneously express an antigen-specific CAR, a kill switch, and mbIL15 genes.
The Sleeping Beauty system is based on the use of transposons and transposases to stably reprogram immune cells by inserting specific DNA sequences into the genome. This non-viral approach offers several advantages, including ease and low cost of manufacturing, stability for longer-term storage, and lack of immunogenicity within host cells.
The UltraCAR-T platform, which utilizes the Sleeping Beauty system, has the potential to revolutionize CAR-T treatment. It achieves this by increasing patient access through rapid manufacturing, lower manufacturing costs, and improved outcomes using advanced technologies for precise tumor targeting and control of the immune system.
The mbIL15 gene, or membrane-bound interleukin-15, is a proprietary chimeric cytokine that is engineered to be tethered to the cell surface to avoid systemic circulation. Expression of the mbIL15 gene has been shown to improve the functional characteristics of certain immune cells, including T cells, resulting in a longer-lasting anti-tumor response.
Overall, Precigen's Sleeping Beauty system offers a promising approach to precision immunology by effectively modifying T cells to target neoantigens and improve patient outcomes in the treatment of tumors.
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Sleeping Beauty is used in UltraCAR-T, a multigenic autologous CAR-T platform
Precigen is a biopharma company that works on advancing precision immunology through novel gene and cell therapies. Their UltraCAR-T platform is a multigenic autologous CAR-T platform that utilizes Precigen's non-viral Sleeping Beauty system to simultaneously express an antigen-specific CAR, kill switch, and mbIL15 genes.
The Sleeping Beauty system is a non-viral gene transfer system that can modify T cells to target neoantigens. It is a transposon/transposase system that stably reprograms immune cells by inserting specific DNA sequences into the genome. The advantages of this system include the ease and relatively low cost of manufacturing, stability for longer-term storage, and a lack of immunogenicity once inside host cells.
UltraCAR-T cells are precision-engineered to produce a homogeneous cell product. The manufacturing process eliminates the need for ex vivo propagation, reducing wait times for patients from weeks to less than two days. This is achieved through rapid manufacturing, lower manufacturing-related costs, and improved outcomes using advanced technologies for precise tumor targeting and control of the immune system.
The mbIL15 gene, or membrane-bound interleukin-15, is a proprietary chimeric cytokine that is engineered to be tethered to the cell surface to avoid systemic circulation. Expression of the mbIL15 gene improves the functional characteristics of certain immune cells, including T cells, resulting in a longer-lasting anti-tumor response.
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The system has been optimised using UltraVector DNA construction
Precigen's UltraCAR-T platform is a significant advancement in the CAR-T treatment landscape, offering rapid manufacturing, lower costs, and improved patient outcomes. This platform utilizes Precigen's non-viral Sleeping Beauty system, which has been optimized using the UltraVector DNA construction platform.
The UltraVector technology, combined with Precigen's AdenoVerse vectors, enables the engineering of cutting-edge gene therapies for complex diseases. This system allows for a large multigenic payload to be delivered with high efficiency, resulting in UltraCAR-T cells that are precision-engineered to produce a homogeneous cell product.
The UltraVector DNA construction platform has been instrumental in optimizing the Sleeping Beauty system by enhancing gene elements, genetic payload capacity, and efficiency. This optimization has led to a clinically-validated system capable of enabling high-level and stable gene transfer.
By utilizing UltraVector, Precigen has improved the Sleeping Beauty system's ability to reprogram immune cells by inserting specific DNA sequences into the genome. This non-viral approach offers advantages such as ease of manufacturing, stability for storage, and a lack of immunogenicity within host cells.
The combination of UltraCAR-T and UltraVector technologies has resulted in a potent platform that can efficiently target and control the immune system, providing patients with improved treatment outcomes.
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Sleeping Beauty has advantages such as ease of manufacturing and stability
Precigen's Sleeping Beauty is a non-viral gene transfer system that modifies T cells so they target neoantigens. It is used in Ziopharm's R&D programs, specifically its CAR-T and TCR-T platforms.
The Sleeping Beauty system has advantages such as ease of manufacturing and stability. It is a non-viral transposon/transposase system that can stably reprogram immune cells by inserting specific DNA sequences into the genome. This system brings the advantages of non-viral vectors, including ease and low cost of manufacturing, stability for longer-term storage, and a lack of immunogenicity once inside host cells.
The UltraCAR-T platform uses the non-viral Sleeping Beauty system, which has been optimized using the UltraVector® DNA construction platform to deliver a large multigenic payload at high efficiency. As a result, UltraCAR-T cells are precision-engineered to produce a homogeneous cell product that simultaneously expresses an antigen-specific CAR, kill switch, and mbIL15 genes. The manufacturing process eliminates the need for ex vivo propagation, decreasing wait times for patients from weeks to less than two days.
The AdenoVerse platform, another one of Precigen's innovations, utilizes a library of proprietary adenovectors for efficient gene delivery of vaccine antigens and cytokines to modulate the immune system. Their gorilla adenovectors can deliver up to 12kb of genetic payload in vivo, which is larger than other viral vectors currently used in gene therapy.
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Frequently asked questions
Precigen's Sleeping Beauty is an advanced non-viral system that simultaneously expresses an antigen.
Sleeping Beauty is a non-viral transposon/transposase system that stably reprograms immune cells by inserting specific DNA sequences into the genome. It is a gene delivery system that offers advantages such as ease of manufacturing, stability for storage, and lack of immunogenicity.
Sleeping Beauty offers the benefits of non-viral vectors, including ease and lower costs of manufacturing, stability for long-term storage, and lack of immunogenicity once inside host cells.
The UltraCAR-T platform is a differentiated treatment method that increases patient access through rapid and low-cost manufacturing, and improved outcomes using advanced technologies for precise tumor targeting. It utilizes the non-viral Sleeping Beauty system to deliver a large multigenic payload with high efficiency.
