Our Science
Bringing the power of in vivo multi-cellular genome editing to complex diseases.
The Engenious™ Platform
With a single outpatient treatment, Engenious empowers patients’ own cells to cure disease. Its unprecedented delivery and engineering capabilities open up new diseases and populations to the power of in vivo gene editing.
Ex vivo approaches began the story of cell therapies – now we’re rewriting their narrative to be simpler, safer, smarter – and inside the body.
A new standard for versatile cell programming and control
As the source of the blood and immune cells throughout our bodies, the hematopoietic system is intrinsically linked with health and disease. Our platform, Engenious, is the first to deliver in vivo editing of any or all hematopoietic cells with a one-time, off-the-shelf treatment. It achieves this through exquisitely precise delivery of expansive payloads to the cell types of our choice.
We believe Engenious’ combination of delivery and capacity for a broad suite of DNA editing tools creates the foundation for the future of in vivo genomic medicines.
Smart cell therapies for cancer
In vivo editing of the immune system offers the potential to engineer the body’s natural defenses to overcome cancer’s immuno-suppressive abilities. Engenious suggests a new way forward for treating cancer built on multi-functional combinations of engineered cell therapies produced by the patient. In practice, this could mean several engineered cell types simultaneously expressing recombinant receptors that direct an immune attack at tumors.
Engenious™ First-in-class delivery
Two decades of research & development support our virus-like-particle (VLP) delivery system. Devoid of any viral genes, its capsid design minimizes the chance of an immune response. Crucially, it allows for both direct transduction of any combination of immune cell types, as well as hematopoietic stem cells directly – putting the full breadth of the immune and blood cell armamentarium at our disposal.
Applying the full DNA engineering toolkit
Our VLPs have a vast payload capacity – up to 35 kilobases of packaged DNA, which is more than seven times the limit of AAV, a leading in vivo delivery tool. This lets us incorporate a full range of modern gene modification technologies, including gene replacement, editing, endogenous protein therapies, regulatory elements, and more.
The ability to use several of these tools in a single therapy holds the full promise of multiplexed therapies: several multi-functional engineered cell types working in concert to fight disease within the patient.
Clinical breakthroughs:
What Engenious is designed to deliver
Simple treatment process
Single outpatient treatment step
In-vivo off-the-shelf therapy
No toxic pre-treatment
No complex manufacturing per patient
Unmatched durability and efficacy
Immediate functionality delivered by directly targeting mature immune cells
Durable functionality delivered by directly targeting stem cells
One-shot solution to complex diseases offered by multiplexed combination therapies
Tuning of therapeutic efficacy by use of an existing drug to boost the proportion of engineered cells in the blood and bone marrow
Re-dosing not required: Removes risk of repeat-dose immunogenicity
Our Pipeline
At the nexus of scientific validation & unmet need
