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
Program Cell Product Research &
Discovery
Lead
Optimization
Preclinical
Development
Phase 1/2 Phase 3
SIG-005: MPS-1 Human IDUA
SIG-002: T1 Diabetes Human Insulin
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
SIG-007: Fabry Human GLA
SIG-018: MPS-2 + CNS Human IDS + BBB
SIG-020: MPS-6 Human ARSB
SIG-XXX: Liver Disease Undisclosed
SIG-001: Hemophilia A* BDD-FVIII

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