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Vision

Sigilon Therapeutics鈥� mission is to create immune-protected, engineered human cells that restore normal physiology in a wide range of diseases without generating fibrosis or immune rejection, liberating patients from challenges associated with serious chronic diseases. Treatments based on the Sigilon Therapeutics platform combine advanced cell engineering with cutting-edge innovations in biocompatible materials to pioneer a new class of therapeutics, Shielded Living Therapeutics鈩� (SLTx), that provide durable, controllable, and safe treatment for serious chronic diseases. In April 2018, Sigilon and Eli Lilly announced a global collaboration to develop encapsulated cell therapies for the potential treatment of type 1 diabetes.

Technology

The Shielded Living Therapeutics鈩� platform combines proprietary Afibromer鈩� technology with novel, engineereed human cell lines that can produce a wide range of proteins to treat serious chronic disorders.

A New Treatment Modality

For many years, researchers have worked towards implanted cell-based treatments to replace therapies for several chronic diseases. In the case of diabetes, one area of focus has been the development of implanted donor cells that can sense blood glucose and respond by secreting insulin. However, this approach suffers from a critical limitation: a patient鈥檚 immune system will recognize donor cells as foreign and then destroy them, unless the treatment is co-administered with immunosuppressant drugs: use of the latter on a long-term basis is associated with significant risks including infection and cancer. This challenge partly explains why cell-based therapeutic implants are not yet more widespread. It also underscores the tremendous potential of Sigilon鈥檚 key innovation: The Shielded Living Therapeutics鈩� platform harnesses the power of therapeutic human cells without inducing an immune response.

These engineered human cells can either deliver proteins at a constant rate or be programmed to respond to changing conditions using the techniques of synthetic biology, such as intermittent flare-ups of disease symptoms (e.g., flare-ups that are characteristic of certain autoimmune diseases) or fluctuating levels of circulating factors such as glucose (e.g., conditions relevant to the treatment of diabetes).

Synthetic biology tools and programmable genetic circuits create new possibilities for the treatment of complex and serious chronic diseases. Shielded Living Therapeutics鈩� can be programmed to sense changing conditions and respond appropriately, acting as 鈥渟mart鈥� medicines inside the body. This is unlike gene therapies or RNA interference, which cannot be adjusted once they are introduced into the body.

Cell Technology

Sigilon has developed proprietary engineered human cells that are combined with Afibromer鈩� technology to create effective therapeutics. These human cells:

Express high levels of desired protein(s)

Survive well in an encapsulation environment

Can be produced at large scale

Are like those previously used in clinical applications

Provide the ability to easily insert and remove gene expression cassettes for different applications

Afibromer鈩� Chemistry

In experimental animal models, Shielded Living Therapeutics鈩� using Afibromer鈩� technology have resisted fibrosis for 12 months or more. Assuming this success can be repeated in humans, Sigilon鈥檚 hope is that Shielded Living Therapeutics鈩� could generate and secrete the natural biomolecules a patient needs over long periods of time, even years, maintaining constant therapeutic protein replacement. This goes beyond convenience and adherence and is expected to produce superior longer- term patient outcome.

Pipeline

Sigilon鈥檚 pipeline of products in hemophilia, lysosomal storage diseases, and, in collaboration with Eli Lilly, type 1 diabetes, are rapidly advancing toward the clinic, and the company is carrying out preclinical studies in other chronic disease indications. The company also believes it will find uses for Afibromer鈩� beyond cell therapy; for instance, to reduce the incidence of fibrosis when using glass, plastic, or metal in medical implants such as coronary stents.