Developing potential functional cures for patients with chronic diseases
A new treatment modality
For many years, researchers have investigated cell and gene therapies as potential replacement therapies for severe chronic diseases. Despite major advancements in the field, these approaches carry a number of limitations including immune rejection, requirement for immune suppression, limited eligibility, durability and variability challenges, inability to re-dose and high manufacturing costs. To address these drawbacks, we developed our novel Shielded Living Therapeutics™ (SLTx) platform, which we believe has the potential to result in functional cures for patients with a wide range of chronic diseases.
Leveraging the modularity of our SLTx platform, we have developed a growing pipeline of cell-based product candidates that have the potential to produce a wide range of missing or deficient therapeutic molecules (e.g. proteins, antibodies and enzymes). Moreover, our product candidates are designed to be off-the-shelf, durable, controllable and redosable, without requiring modification of the patient’s genes or immunosuppression.
We are presently focused on three therapeutic areas: rare blood disorders, lysosomal storage diseases, and endocrine and other chronic diseases. While the diseases we aim to treat are quite different, our products candidates themselves are generated through three common elements: the cell, the sphere, and our streamlined manufacturing process.
The Shielded Living Therapeutics™ platform
Sigilon has developed proprietary engineered human cells that:
- Express high levels of desired protein(s)
- Deliver proteins at a constant rate or are programmed to “sense and respond” to the changing environment
- Survive well in an encapsulation environment
- Can be produced at large scale
- Are similar to cells previously used in clinical applications
- Provide the ability to easily insert new gene expression cassettes for different applications
The sphere is composed of our proprietary Afibromer™ matrix, which exhibits ideal properties for housing thousands of therapeutic cells. Building upon foundational discoveries made at MIT, Sigilon has developed this dual-layer sphere that can remain in the body while generating minimal fibrosis or immune rejection.
1) Outer layer with Afibromer™
• Preventing immune response and fibrosis
• Allowing influx of nutrients and efflux of protein
2) Inner compartment
• Optimized for viability and productivity
Sigilon has created a state-of-the-art manufacturing platform that is modular for all potential product candidates developed. With virtually all aspects of the platform shared across the development programs, our manufacturing process is designed for reproducibility, speed and cost-efficiency, with the hopes of providing a true “off-the-shelf” product for patients.
In preclinical studies, components of our product candidates were well-tolerated with no notable adverse effects for up to 12 months. In addition, the spheres were successfully retrieved and redosed.