Addressing a broad range of chronic diseases
Our Shielded Living TherapeuticsTM platform combines advanced cell engineering with innovations in biocompatible materials, potentially enabling our products to produce a wide range of therapeutic molecules that may be missing or deficient in the body, including proteins, enzymes or other therapeutic molecules.
Our pipeline primarily spans a range of lysosomal and endocrine diseases, with the current focus on MPS-1 and diabetes. Our product candidates are designed to be off-the-shelf, durable, controllable and redosable, without requiring modification of the patient’s genes or complete suppression of the patient’s immune system.
Prioritized Areas of Development | |||||||||||
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Program | Cell Product | Research & Discovery |
Lead Optimization |
Preclinical Development |
Phase 1/2 | Phase 3 | |||||
SIG-005: MPS-1 | Human IDUA |
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SIG-002: T1 Diabetes | Human Insulin |
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Expansion Areas of Development | |||||||||||
Program | Cell Product | Research & Discovery |
Lead Optimization |
Preclinical Development |
Phase 1/2 | Phase 3 | |||||
SIG-XXX: MPS-1 + CNS | Human IDUA + BBB |
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SIG-007: Fabry | Human GLA |
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SIG-018: MPS-2 + CNS | Human IDS + BBB |
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SIG-020: MPS-6 | Human ARSB |
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SIG-XXX: Liver Disease | Undisclosed |
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SIG-001: Hemophilia A* | BDD-FVIII |
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Notes: IDUA = alpha-L-iduronidase; GLA= alpha-galactosidase A ; IDS= iduronate sulfatase; ARSB= Arylsulfatase b ; BBB= Blood brain Barrier penetrants
* Phase 1/2 clinical trial currently on hold – continuing to follow first three patients