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, 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...
Lysosomal diseases are a group of rare, genetic metabolic diseases in which cellular waste products build up in the body. These disorders are generally progressive and there is no cure. Many lysosomal diseases have no FDA-approved treatment.
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.
Rare blood disorders are a group of rare genetic disorders where the blood does not clot properly. Existing therapies for blood disorders place a high burden on patients and caregivers, requiring infusions as often as multiple times each week over a patient’s lifetime.