It’s time to unlock the full potential of genetic medicine.
Imagine a world not limited by disease.
Envisioning a future where people everywhere live free from the devastation of genetic disease.
At Generation Bio, our platform, comprised of our ceDNA construct, our ctLNP delivery system and our scalable rapid enzymatic manufacturing process, is designed to overcome the limitations of current genetic medicine approaches. Our mission is to create an entirely new class of non-viral genetic medicine that leaves no patient behind, providing long-term efficacy, while offering the ability to redose if needed, treating a wide range of both rare and prevalent diseases.
Our mission isn’t easy, and we’re okay with that.
At Generation Bio, our platform, comprised of our ceDNA construct, our ctLNP delivery system and our scalable rapid enzymatic manufacturing process, is designed to overcome the limitations of current genetic medicine approaches. Our mission is to create an entirely new class of non-viral genetic medicine that leaves no patient behind, providing long-term efficacy, while offering the ability to redose if needed, treating a wide range of both rare and prevalent diseases.
Our proprietary non-viral genetic medicine platform
Three breakthroughs in construct, delivery and manufacturing empower our mission to create a new class of scalable, redosable, non-viral genetic medicine for hundreds of millions of patients living with rare and prevalent diseases.
BROAD THERAPEUTIC REACH.
Our closed-ended DNA construct has far-reaching possibilities.
Different genetic diseases involve different sized genes. Our closed-ended DNA construct (ceDNA, pronounced sed-na) has almost three times the capacity of AAV capsids, enabling it to carry large, and even multiple, genes to a cell. With this ability, ceDNA could allow us to treat a wider range of rare and prevalent diseases than what is currently possible with viral gene therapy. Further, ceDNA has the potential to offer durable expression, which means one treatment could last years.
Take a deeper dive into ceDNA.
BROAD THERAPEUTIC REACH.
Our closed-ended DNA construct has far-reaching possibilities.
Different genetic diseases involve different sized genes. Our closed-ended DNA construct (ceDNA, pronounced sed-na) has almost three times the capacity of AAV capsids, enabling it to carry large, and even multiple, genes to a cell. With this ability, ceDNA could allow us to treat a wider range of rare and prevalent diseases than what is currently possible. Further, ceDNA has the potential to offer durable expression, which means one treatment could last years.
TAKE A DEEPER DIVE INTO ceDNA.
Different genetic diseases involve different sized genes. Our closed-ended DNA construct (ceDNA, pronounced sed-na) has almost three times the capacity of AAV capsids, enabling it to carry large, and even multiple, genes to a cell. With this ability, ceDNA could allow us to treat a wider range of rare and prevalent diseases than what is currently possible with viral gene therapy. Further, ceDNA has the potential to offer durable expression, which means one treatment could last years.
Let's start at the beginning. Literally.
We are focused on opening a new frontier of genetic medicine that will allow treatment in infancy, before a genetic disease has a chance to progress. We see a future where people live long, full lives never realizing how their genetic conditions could have once affected them.
REDOSABLE. TITRATABLE.
By changing the way genetic medicine is delivered, we’re changing what’s possible.
In order to do its important work, our ceDNA has to carry a gene or genes to the right location in the body and access the cell’s nucleus. For this, we’ve engineered our cell-targeted lipid nanoparticle (ctLNP). Being non-viral, this delivery system can be available to everyone, and unlike viral gene therapy, does not exclude people with preexisting immunity. Further, since our ctLNP has been designed to avoid activation of the immune system upon initial dose, a person’s treatment can be titrated in steps to achieve the right individual outcome, without overshooting, and redosed if needed to extend the effect of treatment over a lifetime.
Take a deeper dive into ctLNP.
REDOSABLE. TITRATABLE.
By changing the way genetic medicine is delivered, we’re changing what’s possible.
In order to do its important work, our ceDNA has to carry a gene or genes to the right location in the body and access the cell’s nucleus. For this, we’ve engineered our cell-targeted lipid nanoparticle (ctLNP). Being non-viral, this delivery system can be available to everyone, and unlike viral gene therapy, does not exclude patients with preexisting immunity. Further, since our ctLNP has been designed to avoid activation of the immune system upon initial dose, a patient’s treatment can be titrated in steps to achieve the right individual outcome, without overshooting, and redosed if needed to extend the effect of treatment over a lifetime.
TAKE A DEEPER DIVE INTO ctLNP.
In order to do its important work, our ceDNA has to carry a gene or genes to the right location in the body and access the cell’s nucleus. For this, we’ve engineered our cell-targeted lipid nanoparticle (ctLNP). Being non-viral, this delivery system can be available to everyone, and does not disqualify patients with preexisting immunity.
Further, since our ctLNP has been designed to avoid activation of the immune system upon initial dose, a patient’s treatment can be titrated in steps to achieve the right individual outcome, without overshooting, and if needed, re-dosed to extend the effect of treatment over a lifetime.
Genetic disease is a family thing.
When genetic disease touches a life, it affects a whole family. We look to the day when parents, grandparents, brothers, sisters and spouses can spend less time focused on their loved one’s health and more time enjoying their loved one’s life.
Global potential.
If you can efficiently manufacture hundreds of millions of doses you can effectively change hundreds of millions of lives.
Our aim to create a new class of genetic medicine that leaves no one behind calls for a manufacturing process that is efficient, reliable and scalable. Our capsid-free manufacturing process uses cell-free rapid enzymatic synthesis (RES) to produce highly consistent, highly pure ceDNA in just one day, compared to the 28 days required with traditional cell-based manufacturing. RES can be rapidly scaled to produce not millions, but hundreds of millions of doses to enable our vision: a future where genetic medicine is available to more people, living with more diseases, in more places around the world.
Take a deeper dive into enzymatic manufacturing.
Global potential.
If you can efficiently manufacture hundreds of millions of doses you can effectively change hundreds of millions of lives.
Our goal to create a new class of non-viral gene therapy that leaves no patient behind calls for a manufacturing process that is reliable and scalable. Our capsid-free manufacturing process is engineered to use existing biologics facilities to enable not thousands, but millions of doses. We seek to create unprecedented access for patients everywhere. Together with our large-capacity ceDNA and our non-viral ctLNP delivery system, our scalable manufacturing enables our vision: a future where gene therapy is available to more people living with more diseases, in more places around the world.
Take a deeper dive into enzymatic manufacturing.
Our aim to create a new class of genetic medicine that leaves no one behind calls for a manufacturing process that is efficient, reliable and scalable. Our capsid-free manufacturing process uses cell-free rapid enzymatic synthesis (RES) to produce highly consistent, highly pure ceDNA in just one day, compared to the 28 days required with traditional cell-based manufacturing. RES can be rapidly scaled to produce not millions, but hundreds of millions of doses to enable our vision: a future where genetic medicine is available to more people, living with more diseases, in more places around the world.
Unlocking the potential of genetic medicine and the potential of Zac and Jonathan
Zac and Jonathan were born with a rare genetic disease called phenylketonuria, or PKU, that severely restricts their diets. Their big, loving family spends every dinnertime laughing around the same long dining table, even if they have to eat very different foods.
Our Scientific Presentations
As we push the limits of genetic medicine, we document our breakthroughs and learnings. Find out more about our platform and progress.
Our high-capacity ceDNA is an engineered, double-stranded, linear, covalently closed-ended DNA construct that includes the gene of interest and other regulatory elements that drive expression. This genetic payload is flanked by two inverted terminal repeat (ITR) sequences which are responsible for many of ceDNA’s unique features.
Native Gene Regulation
The large capacity of our ceDNA constructs provides us the freedom to incorporate native regulatory elements naturally associated with the gene we are replacing. These regulatory elements act as powerful “on/off” switches which may enable expression of the therapeutic protein to adjust in response to changes in the person’s body.
LARGE PAYLOAD CAPACITY. NEW INDICATIONS.
ceDNA has significant payload capacity that can deliver constructs of 12 kilobases, almost three times the capacity of AAV. This increased capacity creates the opportunity to treat diseases caused by larger genes such as Wilson disease, LCA10 and Stargardt disease. It also allows for the potential to include novel multi-gene constructs to produce complex biologics such as monoclonal antibodies and to address conditions that need more than one type of genetic correction.
MORE POTENT CONSTRUCTS
Our large capacity ceDNA constructs have the potential to utilize novel expression elements that exceed the capacity of currently available gene therapy modalities. This may enable improved expression to treat diseases such as hemophilia A and Gaucher disease and potentially lower the dose required to achieve therapeutic efficacy.
Durable Expression
Once in the host cell nucleus, ceDNA drives durable expression of the gene of interest without integrating into the genomic DNA. This durability has the potential to enable years-long expression with each dose, minimizing the treatment burden for people.
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ABILITY TO REACH INFANTS AND CHILDREN
Children grow fast, and their cells divide rapidly. This is a challenge for traditional, viral-based delivery methods because the effect of the treatment “washes out” over time as cells divide. The ability to titrate the initial doses of our ceDNA-ctLNP may offer infants and children appropriate levels of treatment when it matters most – before disease progression is underway — and the ability to redose as needed may allow our therapies to extend expression throughout life.
INDIVIDUALIZED PATIENT TITRATION
Unlike the vectors used to deliver viral-based genetic medicine, our non-viral ctLNP delivery system has been designed to avoid activation of the immune system in patients upon initial dose. This enables individualized patient titration whereby each patient can be redosed until they reach the expression level required to address their specific therapeutic needs.
LIFELONG IMPACT
We expect our genetic medicines to have a durable effect, enabling years long expression with each dose, similar to current viral-based gene therapies. If expression of the gene of interest begins to wane over time, the ability to redose our ceDNA-ctLNP may restore effective expression levels, prolonging the benefits of our therapies throughout a person’s life.
GREATER OPPORTUNITIES FOR HUMAN TRIALS
With the ability to titrate and redose, we believe that a greater proportion of people participating in our early clinical trials may achieve the desired level of gene expression.
TARGETED, MULTI-TISSUE DELIVERY
Our ctLNP uniquely employs biological ligands designed to engage receptors on the surface of specific tissues, such as the liver or the muscle. We aim to deliver ceDNA to the exact tissue where it’s needed, with limited delivery to off-target cells. We believe this will improve the safety and efficacy of our products. Because the system is modular, we can exchange ligands to reach different tissues as needed for different diseases.
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TARGETING RARE AND PREVALENT DISEASE
There are approximately 5000 rare diseases and more than a million common ones. We see the capacity to scale to hundreds of millions of doses offering the ability to treat not only rare disease, but prevalent ones, as well. This is a new frontier for genetic medicine, with the goal of leaving no one behind.
GLOBAL POTENTIAL
With our rapid, highly scalable, RES manufacturing process, we aim to make genetic medicine available for more people, for more diseases, in more places around the world. We are focused not just on rare diseases, but also on prevalent ones affecting millions of patients globally. Our goal is to leave no patient behind.
POTENTIAL TO SCALE FOR WORLDWIDE ACCESS
Our programs for endogenous therapeutic antibody production (ETAP) seek to treat serious and life-threatening diseases by equipping the patient’s own liver cells with the genetic code they need to produce a steady supply of therapeutic antibodies. We believe this approach can enable treatment of infectious and non-infectious diseases and can lead to effective, durable treatments for hundreds of millions of people worldwide (on a scale similar to mRNA manufacturing). Our highly scalable RES manufacturing process is designed to make that dream a reality.
HIGH QUALITY
Our rapid enzymatic synthesis (RES) of ceDNA consistently yields highly pure material and reduces the variability inherent in cell-based manufacturing.
EXPLORE ceDNA
Durable Expression
More Potent Constructs
Large Payload Capacity. New Indications.
Novel Structure
Native Gene Regulation
Durable Expression
Once in the host cell nucleus, ceDNA drives durable expression of the gene of interest without integrating into the genomic DNA. This durability has the potential to enable years-long expression with each dose, minimizing the treatment burden for people.
MORE POTENT CONSTRUCTS
Our large capacity ceDNA constructs have the potential to utilize novel expression elements that exceed the capacity of currently available gene therapy modalities. This may enable improved expression to treat diseases such as hemophilia A and Gaucher disease and potentially lower the dose required to achieve therapeutic efficacy.
LARGE PAYLOAD CAPACITY. NEW INDICATIONS.
ceDNA has significant payload capacity that can deliver constructs of 12 kilobases, almost three times the capacity of AAV. This increased capacity creates the opportunity to treat diseases caused by larger genes such as Wilson disease, LCA10 and Stargardt disease. It also allows for the potential to include novel multi-gene constructs to produce complex biologics such as monoclonal antibodies and to address conditions that need more than one type of genetic correction.
Novel Structure
Our high-capacity ceDNA is an engineered, double-stranded, linear, covalently closed-ended DNA construct that includes the gene of interest and other regulatory elements that drive expression. This genetic payload is flanked by two inverted terminal repeat (ITR) sequences which are responsible for many of ceDNA’s unique features.
Native Gene Regulation
The large capacity of our ceDNA constructs provides us the freedom to incorporate native regulatory elements naturally associated with the gene we are replacing. These regulatory elements act as powerful “on/off” switches which may enable expression of the therapeutic protein to adjust in response to changes in the person’s body.
EXPLORE ctLNP
Targeted, Multi-tissue Delivery
Individualized Patient Titration
Ability to reach Infants and Children
Lifelong Impact
Greater Opportunities for human trials
TARGETED, MULTI-TISSUE DELIVERY
Our ctLNP uniquely employs biological ligands designed to engage receptors on the surface of specific tissues, such as the liver or the muscle. We aim to deliver ceDNA to the exact tissue where it’s needed, with limited delivery to off-target cells. We believe this will improve the safety and efficacy of our products. Because the system is modular, we can exchange ligands to reach different tissues as needed for different diseases.
INDIVIDUALIZED PATIENT TITRATION
Unlike the vectors used to deliver viral-based genetic medicine, our non-viral ctLNP delivery system has been designed to avoid activation of the immune system in patients upon initial dose. This enables individualized patient titration whereby each patient can be redosed until they reach the expression level required to address their specific therapeutic needs.
ABILITY TO REACH INFANTS AND CHILDREN
Children grow fast, and their cells divide rapidly. This is a challenge for traditional, viral-based delivery methods because the effect of the treatment “washes out” over time as cells divide. The ability to titrate the initial doses of our ceDNA-ctLNP may offer infants and children appropriate levels of treatment when it matters most – before disease progression is underway — and the ability to redose as needed may allow our therapies to extend expression throughout life.
LIFELONG IMPACT
We expect our genetic medicines to have a durable effect, enabling years long expression with each dose, similar to current viral-based gene therapies. If expression of the gene of interest begins to wane over time, the ability to redose our ceDNA-ctLNP may restore effective expression levels, prolonging the benefits of our therapies throughout a person’s life.
GREATER OPPORTUNITIES FOR HUMAN TRIALS
With the ability to titrate and redose, we believe that a greater proportion of people participating in our early clinical trials may achieve the desired level of gene expression.
EXPLORE ENZYMATIC MANUFACTURING
TARGETING RARE AND PREVALENT DISEASE
POTENTIAL TO SCALE FOR WORLDWIDE ACCESS
Global Potential
High Quality
TARGETING RARE AND PREVALENT DISEASE
There are approximately 5000 rare diseases and more than a million common ones. We see the capacity to scale to hundreds of millions of doses offering the ability to treat not only rare disease, but prevalent ones, as well. This is a new frontier for genetic medicine, with the goal of leaving no one behind.
POTENTIAL TO SCALE FOR WORLDWIDE ACCESS
Our programs for endogenous therapeutic antibody production (ETAP) seek to treat serious and life-threatening diseases by equipping the patient’s own liver cells with the genetic code they need to produce a steady supply of therapeutic antibodies. We believe this approach can enable treatment of infectious and non-infectious diseases and can lead to effective, durable treatments for hundreds of millions of people worldwide (on a scale similar to mRNA manufacturing). Our highly scalable RES manufacturing process is designed to make that dream a reality.
GLOBAL POTENTIAL
With our rapid, highly scalable, RES manufacturing process, we aim to make genetic medicine available for more people, for more diseases, in more places around the world. We are focused not just on rare diseases, but also on prevalent ones affecting millions of patients globally. Our goal is to leave no patient behind.
HIGH QUALITY
Our rapid enzymatic synthesis (RES) of ceDNA consistently yields highly pure material and reduces the variability inherent in cell-based manufacturing.
