We are committed, both individually and collectively, to unlocking the full potential of genetic medicine.
Our pioneering platform has the potential to extend the power of gene therapy to many more diseases, affecting many more people, than what is possible with existing gene therapy technology. We are advancing a broad and expansive portfolio in both rare and prevalent diseases.
Initially, we are focusing on diseases of the liver and retina with great unmet need and for which our non-viral gene therapy platform may significantly improve lives. We plan to expand into additional rare and prevalent diseases of the liver and retina, as well as in skeletal muscle, the central nervous system and oncology. We also plan to advance antibody gene therapy programs where we see a possibility of directing the body’s own cells to produce consistent levels of therapeutic antibodies for years at a time from a single dose.
Explore the platform behind the promise.
Our pioneering platform has the potential to extend the power of gene therapy to many more diseases, affecting many more people, than what is possible with existing gene therapy technology. We are advancing a broad and expansive portfolio in both rare and prevalent diseases.
Initially, we are focusing on diseases of the liver and retina with great unmet need and for which our non-viral gene therapy platform may significantly improve patients' lives. We plan to expand into additional rare and prevalent diseases of the liver and retina, as well as in skeletal muscle, the central nervous system and oncology. We also plan to advance antibody gene therapy programs where we see a possibility of directing the body’s own cells to produce consistent levels of therapeutic antibodies for years at a time from a single dose.
Hemophilia A is a rare X-linked hereditary bleeding disorder in which the blood does not clot properly. It is a result of deficiency in the production or function of coagulation Factor VIII. We aim to titrate dosing of our ceDNA therapy to achieve individualized therapeutic Factor VIII levels for each child and adult, restoring normal clotting function, preventing or reducing the risk of bleeding episodes and potentially eliminating the need for recurring Factor VIII injections.
Hemophilia A affects approximately 16,000 people in the United States and 320,000 worldwide.
PKU is a rare autosomal recessive genetic disorder caused by deficiency of the hepatic enzyme phenylalanine hydroxylase. This enzyme metabolizes the essential amino acid phenylalanine, or Phe. Since Phe is found in all protein containing foods, eating a normal diet with PKU leads to elevated levels of Phe in the blood, and toxic Phe accumulation in the brain. This results in symptoms like skin rashes, neurological problems including intellectual disability, and behavioral, social and emotional issues. We aim to achieve stable correction of Phe levels in people of all ages with PKU, which may allow them to have more normal diets, eliminate the burden of ongoing treatment and stabilize and/or preserve neurocognitive function. PKU affects approximately 15,000 people in the United States and 41,000 in the EU.
Wilson disease is a rare autosomal recessive disease due to a loss-of-function mutation in the ATB7B copper transporter. We aim to achieve full hepatic correction of copper metabolism early in the disease by delivering the full-length ATP7B gene with an optimized expression cassette, re-directing copper to be incorporated into ceruloplasmin and normalizing bile excretion. This offers the potential to overcome liver fibrosis and prevent irreversible neurocognitive decline.
There are approximately 11,000 people with Wilson disease in the United States and 17,000 in the EU.
Gaucher disease is a rare inherited autosomal recessive disorder caused by the insufficient expression of lysosomal enzyme glucocerebrosidase, or GCase. Gaucher disease is the most common inherited lysosomal storage disease. We aim to treat early in the disease, before the onset of inflammation, fibrosis and irreversible tissue injury and to provide consistent GCase expression levels, with the potential to significantly reduce the treatment burden relative to enzyme replacement therapy (ERT) infusions.
There are approximately 6,000 people with Gaucher disease in the United States and 9,000 in the EU.
Our antibody gene therapy program seeks to deliver to the liver the genes needed to produce specific antibodies. With a single dose, our non-viral gene therapy platform may enable durable secretion of antibodies to provide consistent therapeutic effects over time. We aim to advance antibody gene therapy programs across multiple therapeutic areas.
Stargardt disease is the most common inherited macular dystrophy. We aim to deliver the full ABCA4 gene to photoreceptors and RPE cells and halt the progressive loss of retinal rods/cones to preserve vision.
Stargardt disease affects approximately 37,000 people in the United States and 66,000 in the EU.
Leber’s Congenital Amaurosis 10 (LCA10)
LCA10 is the most common genetic cause of childhood vision loss. We aim to treat all people with LCA10, irrespective of their CEP290 mutation, utilizing ctLNP to specifically deliver durable CEP290 expression to photoreceptors, potentially recovering visual function for patients with a one-time treatment.
LCA10 affects approximately 2,200 people in the United States and 3,400 in the EU.
Wet Age-Related Macular Degeneration (wet AMD)
AMD is the leading cause of vision loss occurring in approximately 10 million people in the United States. Wet AMD is the most severe form of AMD, characterized by neovascularization of the retina, leading to significant loss in visual acuity and rapid progression to blindness. We aim to deliver intravitreally to enable retinal cells to sustainably produce and secrete anti-VEGF. This may enhance regression of neovascularization and improve visual acuity and may also enable administration only a few times over a person's life, while reducing lapses in treatment coverage.
There are approximately 1.2 million people with wet AMD in the United States and 2.5 million in the EU.
mRNA vaccines are revolutionizing public health. They are scalable and effective against COVID-19, and they hold promise in treating endemic viruses and addressing or even preventing future pandemics. We are leveraging our proprietary LNP platform to explore the expansion of mRNA vaccines and the potential of DNA vaccines to provide increased vaccine stability and a more robust memory response. We aim to deliver vaccines that are effective against a wider set of diseases and available to more people in more places around the world.
There are a variety of genetic muscle disorders, including muscular dystrophies, that may be treated by efficient and systemic gene therapy to skeletal muscle. We are currently developing a ctLNP utilizing a targeting ligand to deliver ceDNA specifically to skeletal muscle.
Central Nervous System (CNS)
We are exploring the use of ceDNA to correct disorders of the CNS. The work we are doing with local delivery in the retina to photoreceptors may inform expansion into the CNS.
We are exploring the use of ceDNA to treat a variety of cancer indications. We believe that we can use distinct targeting ligands to deliver ceDNA specifically and efficiently to tumors and can utilize ceDNA to express high levels of relevant proteins within the tumor.
