Living with serious chronic diseases

People with serious chronic diseases face a number of challenges in managing their condition. The standard treatments for chronic diseases are chronic therapies — for instance, in the case of hemophilia or diabetes, factors or proteins delivered every week, every day, or even every hour, like clockwork. With current therapies, patients don’t have the luxury of forgetting about their illness or taking a day off from tending to their health. There is no peace of mind. And because treatments are given intermittently, their effects wax and wane, leading to higher rates of complications from illness and the need for frequent blood tests to gauge treatments’ efficacy.

 

We at Sigilon believe patients’ lives could be transformed by the invention of a single, long-lasting therapy that delivered steady levels of the necessary protein or factor constantly, day and night, liberating patients from the burden of their disease. That’s why we’re creating Shielded Living Therapeutics™.

Our Shielded Living Therapeutics™ product platform

Sigilon’s 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.

Our initial target therapeutic focus areas

  • Rare blood disorders

    Existing therapies for blood disorders like hemophilia place a high burden on patients and caregivers, requiring infusions as often as multiple times each week over a patient’s lifetime. These therapies also do not entirely restore natural protein function, leaving patients vulnerable to long-term complications. Access to prophylaxis therapy is limited, especially in developing countries.

    Our Shielded Living Therapeutics™ products have the potential to provide durable, consistent delivery of blood clotting factors, and thus may provide a better option for people with hemophilia — delivering both improved efficacy and improved quality of life for these patients.

    Patient resources:
    Hemophilia Federation of America
    National Hemophilia Foundation
    World Federation of Hemophilia

     

     

  • Lysosomal storage disorders

    Lysosomal storage disorders are a group of rare metabolic diseases in which cellular waste products build up in the body. These disorders are generally progressive, and there is no cure. Many lysosomal storage disorders have no FDA-approved treatment.

    Even for diseases with an approved treatment, patients face myriad challenges. For instance, protein-replacement therapy, the standard of care for Fabry disease, requires a lifelong infusion burden with high costs associated. Furthermore, the variable half-life of these treatments means that patients must be constantly vigilant to protect themselves against the effects of fluctuating enzyme levels.

    Shielded Living Therapeutics™ products could provide better efficacy and better outcomes for patients with lysosomal storage disorders. By providing consistent delivery of therapeutics, patients could avoid the peaks and troughs associated with present-day treatments — and many other lysosomal storage disorders could gain an effective treatment.

    Patient resources:

    NORD
    National Gaucher Foundation
    Lysosomal Disease Network

  • Type 1 diabetes

    Type 1 diabetes is a common chronic disease, affecting millions of people throughout the world, in which the immune system attacks the body’s own pancreatic cells, stopping the production of insulin. Though it was previously considered a childhood disease, type 1 diabetes is known to affect people at every age, and it has no cure. Treatment for type 1 diabetes requires constant vigilance, including frequent injections or infusions ofinsulin, blood sugar testing multiple times daily, and dietary and lifestyle modifications. Still, an important percentage of patients will develop chronic complications such as diabetic polyneuropathy or diabetic kidney disease in their lifetime, due to challenges adhering to this schedule or to the peaks and troughs of insulin delivery.

    A potential future treatment being studied is islet cell transplantation. However, this approach suffers from a critical flaw: a patient’s immune system will reject the islet cells unless the treatment is co-administered with immunosuppressant drugs, which carry their own risks.

    We are working with our partner Eli Lilly to develop Shielded Living Therapeutics™ products for the treatment of type 1 diabetes. The therapeutic goal of these products will be to restore normal cell function by both sensing blood glucose levels and releasing insulin over the long term, freeing patients from constant disease management.

    Patient resources:

    American Diabetes Association
    National Institute of Diabetes and Digestive and Kidney Diseases
    JDRF