Our Science

Our Science

enGene has developed a robust non-viral gene-therapy platform to transport DNA sequences across mucosal barriers. The delivered sequences can encode proteins, peptides, antibodies, and the like. Importantly, we have formulated our proprietary DNA delivery system into a Gene Pill™ to provide oral delivery of therapeutic genes to the gut – a game-changing technology that enables oral delivery of protein therapeutics.

The problem

Currently, protein drugs are administered by needle injections to the periphery, which makes it very difficult to provide sufficient therapeutic concentrations of proteins to the intended target site without reaching the tipping point of systemic toxicities. Oral delivery of protein drugs is challenging, as proteins are fragile and prone to degradation. Furthermore, the large size of protein drugs also makes absorption into the body through the gut wall very challenging.

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The solution

enGene has built a safe and robust vehicle to transport DNA to the gut mucosa.

Gene Pill™ technology provides a breakthrough solution by delivering genes that encode various protein drugs to the gut non-invasively via the oral route. Once delivered into cells that line the gut, the desired therapeutic proteins are expressed and secreted into the blood. The Gene Pill™ platform promises to change the way we deliver protein-based medicines.

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enGene has pioneered the oral delivery of DNA to gut mucosal cells using its Gene Pill™ platform. The technology at the core of Gene Pill™ is based on an internally developed DNA carrier made of a synthetic polymer named Dually Derivitized Oligochitosan (DDX®) that is optimized for packaging and transporting DNA into epithelial cells situated behind a mucous barrier. The DDX® carrier platform is also amenable to carrying RNA and Oligo-NA, thereby facilitating the delivery of siRNA, miRNA, aptamers, and the like.

A highly differentiated approach to gene therapy

Gene therapies are starting to gain regulatory approvals for certain diseases, but these current therapies typically use viruses as vehicles or vectors to carry ‘healthy’ genes into human cells. Although viruses can be very efficient in transferring genes (DNA) into human cells, they have many drawbacks as carriers. For example, the virus may cause an immune response in patients and lead to diminishing effectiveness of subsequent doses.
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Scalable and proprietary manufacturing of gene therapies

Viral vectors are also very costly to manufacture, store and distribute. In designing the Gene Pill™, we chose to move away from the conventional gene delivery paradigm that uses viruses as carriers of genetic payloads. Our proprietary non-viral DDX® vehicle is a synthetic polymer that is highly efficient in packaging DNA. As a result, the cost of goods for our gene therapies is markedly lower. Additionally, our formulation can readily interconvert between a nanoparticle suspension and a dry powder formulation, while maintaining stability and functionality.

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The powder is filled into capsules which are subsequently enteric coated to protect the contents in the stomach. The enteric coating dissolves in a defined section of the intestine, releasing the DDX® particles to carry the therapeutic DNA into mucosal cells lining the gut.

The powder can also be reconstituted to a nanoparticle suspension for administration as an enema or as an intravesicular injection (to the bladder, for example).

The nanoparticle suspension unlocks all mucosal tissues

As a result of formulation flexibility, enGene can very effectively target mucosal tissues. These tissues are notoriously difficult for viral vectors and lipid nanoparticles to successfully treat, due to the challenges associated with mucosal barrier penetration and epithelial cell turnover.

Image

The Gene Pill™ enables oral delivery of gene therapies

There is a high unmet medical need for effective delivery of therapeutic proteins to the intestine. In many cases, localized or topical drug delivery is the preferred route of administration, as conventional delivery by needle injection does not provide sufficient drug levels in the intestine and often leads to unwanted systemic toxicities. The Gene Pill™ turns intestinal cells into ‘bioreactors’ to produce protein drugs for local or systemic effects - a form of gene therapy that concentrates expression of protein drugs in the gut, while limiting the broad distribution to the rest of the body. Our gene therapies are therefore designed around high standards for both safety and efficacy.
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The gut is the gateway to the periphery

Although the Gene Pill™ delivers genes to specific regions of the intestine for local effects, the gut is the gateway to the periphery. Over 70% of the liver’s blood flow comes directly from the intestine. As such, the DDX® platform can be leveraged to deliver specific proteins for the intention of secretion into the circulation from the intestine. This in turn enables the targeting of the liver and many other organs.

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enGene has developed a robust non-viral gene-therapy platform to transport DNA sequences across mucosal barriers. The delivered sequences can encode proteins, peptides, antibodies, and the like. Importantly, we have formulated our proprietary DNA delivery system into a Gene Pill™ to provide oral delivery of therapeutic genes to the gut – a game-changing technology that enables oral delivery of protein therapeutics.

The problem

Currently, protein drugs are administered by needle injections to the periphery, which makes it very difficult to provide sufficient therapeutic concentrations of proteins to the intended target site without reaching the tipping point of systemic toxicities. Oral delivery of protein drugs is challenging, as proteins are fragile and prone to degradation. Furthermore, the large size of protein drugs also makes absorption into the body through the gut wall very challenging.

Image

The solution

enGene has built a safe and robust vehicle to transport DNA to the gut mucosa.

Gene Pill™ technology provides a breakthrough solution by delivering genes that encode various protein drugs to the gut non-invasively via the oral route. Once delivered into cells that line the gut, the desired therapeutic proteins are expressed and secreted into the blood. The Gene Pill™ platform promises to change the way we deliver protein-based medicines.

Image

enGene has pioneered the oral delivery of DNA to gut mucosal cells using its Gene Pill™ platform. The technology at the core of Gene Pill™ is based on an internally developed DNA carrier made of a synthetic polymer named Dually Derivitized Oligochitosan (DDX®) that is optimized for packaging and transporting DNA into epithelial cells situated behind a mucous barrier. The DDX® carrier platform is also amenable to carrying RNA and Oligo-NA, thereby facilitating the delivery of siRNA, miRNA, aptamers, and the like.

A highly differentiated approach to gene therapy

Gene therapies are starting to gain regulatory approvals for certain diseases, but these current therapies typically use viruses as vehicles or vectors to carry ‘healthy’ genes into human cells. Although viruses can be very efficient in transferring genes (DNA) into human cells, they have many drawbacks as carriers. For example, the virus may cause an immune response in patients and lead to diminishing effectiveness of subsequent doses.
Image

Scalable and proprietary manufacturing of gene therapies

Viral vectors are also very costly to manufacture, store and distribute. In designing the Gene Pill™, we chose to move away from the conventional gene delivery paradigm that uses viruses as carriers of genetic payloads. Our proprietary non-viral DDX® vehicle is a synthetic polymer that is highly efficient in packaging DNA. As a result, the cost of goods for our gene therapies is markedly lower. Additionally, our formulation can readily interconvert between a nanoparticle suspension and a dry powder formulation, while maintaining stability and functionality.

Image
The powder is filled into capsules which are subsequently enteric coated to protect the contents in the stomach. The enteric coating dissolves in a defined section of the intestine, releasing the DDX® particles to carry the therapeutic DNA into mucosal cells lining the gut.

The powder can also be reconstituted to a nanoparticle suspension for administration as an enema or as an intravesicular injection (to the bladder, for example).

The nanoparticle suspension unlocks all mucosal tissues

As a result of formulation flexibility, enGene can very effectively target mucosal tissues. These tissues are notoriously difficult for viral vectors and lipid nanoparticles to successfully treat, due to the challenges associated with mucosal barrier penetration and epithelial cell turnover.

Image

The Gene Pill™ enables oral delivery of gene therapies

There is a high unmet medical need for effective delivery of therapeutic proteins to the intestine. In many cases, localized or topical drug delivery is the preferred route of administration, as conventional delivery by needle injection does not provide sufficient drug levels in the intestine and often leads to unwanted systemic toxicities. The Gene Pill™ turns intestinal cells into ‘bioreactors’ to produce protein drugs for local or systemic effects - a form of gene therapy that concentrates expression of protein drugs in the gut, while limiting the broad distribution to the rest of the body. Our gene therapies are therefore designed around high standards for both safety and efficacy.
Image

The gut is the gateway to the periphery

Although the Gene Pill™ delivers genes to specific regions of the intestine for local effects, the gut is the gateway to the periphery. Over 70% of the liver’s blood flow comes directly from the intestine. As such, the DDX® platform can be leveraged to deliver specific proteins for the intention of secretion into the circulation from the intestine. This in turn enables the targeting of the liver and many other organs.

Image

enGene has established an extensive patent portfolio protecting our proprietary gene delivery technology and novel product candidates

We are working independently and with pharma partners to develop our optimized gene delivery technology to treat inflammatory bowel disease (IBD), non-muscle invasive bladder cancer (NMIBC), short bowel syndrome (SBS), and ulcerative colitis (UC).

Visit Our Pipeline for more information.

enGene has established an extensive patent portfolio protecting our proprietary gene delivery technology and novel product candidates

We are working independently and with pharma partners to develop our optimized gene delivery technology to treat inflammatory bowel disease (IBD), non-muscle invasive bladder cancer (NMIBC), short bowel syndrome (SBS), and ulcerative colitis (UC).

Visit Our Pipeline for more information.