It’s time to unlock the full potential of genetic medicine.

Imagine a world not 
limited by genetic disease.
Envisioning a future where people everywhere live free from the devastation of genetic disease.
Our mission to offer people everywhere the chance to live unburdened by disease is fueled by our two distinct and complementary platforms. 

Our highly potent and selective cell-targeted lipid nanoparticle (ctLNP) delivery platform is designed to drive redosable ligand-directed targeting carrying a variety of therapeutic cargos to tissues and cell types of interest, while avoiding uptake by off-target cells and clearance organs such as the liver and spleen. 

Our iqDNA cargo platform evades innate immunity while providing durable expression, creating the potential for durable genetic medicines that can be dosed and redosed according to the needs of each individual patient. 

ctLNPs and iqDNA can be produced at scale, meaning we could have the ability to provide life-changing therapies for hundreds of millions of people living with a broad range of rare and prevalent diseases.
Our mission to offer people everywhere the chance to live unburdened by disease is fueled by our two distinct and complementary platforms.

Our highly potent and selective cell-targeted lipid nanoparticle delivery platform is designed to drive redosable ligand-directed targeting carrying a variety of therapeutic cargos to tissues and cell types of interest, while avoiding uptake by off-target cells and clearance organs such as the liver and spleen.

Our iqDNA cargo platform evades innate immunity while providing durable expression, creating the potential for durable genetic medicines that can be dosed and redosed according to the needs of each individual patient.

ctLNPs and iqDNA can be produced at scale, meaning we could have the ability to provide life-changing therapies for hundreds of millions of people living with a broad range of rare and prevalent diseases.
Our Scientific Presentations
As we push the limits of genetic medicine, we document our breakthroughs and learnings. Find out more about our ctLNP and iqDNA platforms and progress.
BROAD THERAPEUTIC REACH
By changing the way genetic medicine is delivered, we’re working to change what’s possible.
Our ctLNP is designed to employ highly selective, potent ligands to deliver a variety of nucleic acid cargos to specific and even previously unreachable tissues and cell types, while avoiding off-target clearance, thus enabling them to persist in systemic circulation so that they can access their target. 

This has the potential to make the ctLNP platform the ideal approach for creating cell therapies in vivo – reprogramming T cells or hematopoietic stem cells inside a person’s own body without transplantation. And because the targeting system is modular, identifying and optimizing new ligands to target new tissues and cell types is efficient and flexible, which may allow us to rapidly expand our portfolio into new therapeutic areas.
Take a deeper dive
into ctLNP.
Redosable
Potent & Selective
MULTI-TISSUE
BROAD THERAPEUTIC REACH.
By changing the way genetic medicine is delivered, we’re working to change what’s possible.
Redosable
Potent & Selective
MULTI-TISSUE
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.
Our ctLNP is designed to employ highly selective, potent ligands to deliver a variety of nucleic acid cargos to specific and even previously unreachable tissues and cell types, while avoiding off-target clearance, thus enabling them to persist in systemic circulation so that they can access their target. 

This has the potential to make the ctLNP platform the ideal approach for creating cell therapies in vivo – reprogramming T cells or hematopoietic stem cells inside a person’s own body without transplantation. And because the targeting system is modular, identifying and optimizing new ligands to target new tissues and cell types is efficient and flexible, which may allow us to rapidly expand our portfolio into new therapeutic areas.
LIFELONG IMPACT.
Forget genetic disease. For years at a time.
If we could deliver long-lasting, high levels of gene expression without triggering an immune response, how would that change how we live with disease? Our iqDNA is designed to enable robust and durable gene expression from non-integrating episomes that express for the life of the cell. This means that each dose of iqDNA would build on the last, allowing for the expression level to be adjusted based on the needs of each individual and for redosing when needed, which could allow people to be treated throughout their lifetime.
Take a deeper dive
into iqDNA.
Durable
Redosable
GAIN OF FUNCTION
LIFELONG IMPACT.
Forget genetic disease. For years at a time.
Durable
Redosable
Gain of Function
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 iqDNA.
LIFELONG IMPACT.
Forget genetic disease. For years at a time.
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 iqDNA.
If we could deliver long-lasting, high levels of gene expression without triggering an immune response, how would that change how we live with disease? Our iqDNA is designed to enable robust and durable gene expression from non-integrating episomes that express for the life of the cell. This means that each dose of iqDNA would build on the last, allowing for the expression level to be adjusted based on the needs of each individual and for redosing when needed, which could allow people to be treated throughout their lifetime.
UNTOLD POTENTIAL
Our highly scalable, cell-free manufacturing process is called rapid enzymatic synthesis, or RES. RES produces highly pure iqDNA at scale, and is key to enabling our vision of a future where genetic medicines are available to more people living with more diseases, in more places around the world.
GLOBAL SCALE
Everyone. Everywhere. It’s why we come to our work every day.
By advancing our ctLNP and iqDNA platforms, we aim to usher in a new day of non-viral genetic medicines—one that provides transformative, life-long treatment for hundreds of millions of people living with both rare and prevalent diseases. We believe our platforms have the potential to reach previously untreatable or under-treated patients and address new indications—unlocking the full potential of genetic medicine and changing the way people all over the world live with disease.
Learn about our current and
future programs.
Global scale.
Everyone. Everywhere. It’s why we come to our work every day.
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.
Learn about our current and future programs.
By advancing our ctLNP and iqDNA platforms, we aim to usher in a new day of non-viral genetic medicines—one that provides transformative, life-long treatment for hundreds of millions of people living with both rare and prevalent diseases. We believe our platforms have the potential to reach previously untreatable or under-treated patients and address new indications—unlocking the full potential of genetic medicine and changing the way people all over the world live with disease.
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IMMUNE-QUIET STRUCTURE
Our RES manufacturing process offers precise control over the structure of DNA, allowing exploration of novel variants that led to the identification of iqDNA. iqDNA is the first DNA construct shown to limit innate immune activation to levels seen with mRNA, now a global therapeutic modality.
TITRATABLE
Because iqDNA is designed to be redosed, treatment can be individualized wherein a second or third dose adds to (or “stacks” on top of) the original dose, something called “titration.” Titration has the potential to adjust the level of expression to match the specific needs of each patient.
LARGE PAYLOAD CAPACITY
iqDNA is designed to have significant payload capacity that creates the opportunity for full gene replacement, which could lead to enhanced efficacy as well as the ability to treat diseases caused by mutations across larger genes. It also allows for the potential to include novel multigene constructs and address conditions that need more than one type of genetic correction.
GAIN OF FUNCTION
Replacing a broken gene or adding new functionality to a cell is called gain of function. This is a powerful therapeutic approach for restoring or improving function, especially because the changes occur in the nucleus of the cell, where most control and regulation of cellular function resides.
Durable Expression
Once in the host cell nucleus, iqDNA 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 patients’ treatment burden.
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STEALTH BASE LNP
Our ctLNP is based on an underlying LNP structure that avoids uptake by the liver and spleen and allows our ctLNPs to remain in systemic circulation where they can reach their targets. This unique property, which we call stealth, allows us to attach specific biological targeting ligands that are designed to engage receptors on the surface of specific tissues and cell types beyond the liver and spleen.
MULTIPLE CARGOS
Our ctLNPs have the capacity to carry specific genetic materials—either RNA, DNA, or a combination. This versatility allows us to explore a wide variety of treatment modalities, such as gene therapy (repairing faulty genes), gene editing (modifying genes), and cellular reprogramming (changing cell functions) in specific tissues and cell types, including those that are currently unreachable.
SELECTIVE TARGETING
By selectively targeting a variety of tissues and cell types, we aim to deliver cargo to exactly where it's needed, with limited delivery to off target cells and tissues where it isn’t. We believe this will contribute to the safety and efficacy of our potential medicines. And because the system is modular, we can exchange ligands to reach different areas as needed, to reach previously unreachable tissues and cell types.
IN VIVO DELIVERY
Our highly selective, potent ctLNP delivery is an ideal therapeutic approach for modifying target cells in vivo. This means giving cells the instructions to become cell therapy without the lengthy, complex and expensive process of taking them out of the body. We are developing in vivo cell therapies that would not require toxic preconditioning and could be administered much more quickly and at a much lower cost than current ex vivo approaches.
REDOSABLE
Because LNPs don't trigger an immune response, they can be dosed more than once, which would allow therapy to begin at low doses, to be adjusted upward as needed, and to extend therapy as needed, and to be extended as needed.
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UNPRECEDENTED CONTROL
Our rapid enzymatic synthesis offers us exquisite control over the structure of DNA. This ability has led to the identification of iqDNA, a novel immune-quiet DNA variant. Delivering iqDNA with our ctLNP enables treatments for a wide range of rare and prevalent diseases of the liver with multiple modalities, including gain of function and gene editing.
TAREGETING 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 ACCESS
We have established a proprietary, cell-free manufacturing platform called RES
that can produce enough material to supply not only every patient in the world for a given rare disease, but also enough to bring genetic medicines to highly prevalent diseases. Crucially, our RES is efficient, allowing for radically lower costs compared to current gene therapies and enabling expanded global access to our therapies.
FOUR-WEEK RESEARCH CYCLE
We have established a highly efficient four-week research cycle to design, produce and screen iqDNAconstructs. This process enables rapid identification of development candidates that achieve disease correction in animal models for our programs. Our research cycle utilizes in vitro activity screens of novel plasmid DNA designs, followed by rapid enzymatic synthesis (RES) and in vivo activity screens of select iqDNA constructs.
HIGH QUALITY
Our cell-free RES manufacturing process yields highly consistent, highly pure iqDNA drug substance in just one day. Because RES enables such a short research cycle, we are able to discover and optimize new iqDNA constructs quickly. In addition, the productivity of RES has allowed us to take advantage of an efficient, flexible GMP approach.
NEWS AND EVENTS
Our mission calls for us to stay connected to both our science community and the world around us. Read the latest on where we’ve been and where we’re headed.

Take a seat at the table.

We are always looking for those who are ready to share their vision, talent, and tenacity—who believe in our mission and themselves.

Imagine a world without genetic disease.

What do you see? More health? Fewer doctor appointments? More bike riding? More peanut butter and jelly sandwiches? More life?
See our shared vision.
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