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.
As a patient or caregiver, you or your loved one faces an array of challenges. You may be living with many different clinical presentations and disabilities, uncertainty about the future course of your disease, or have significant medical expenses. Even when available, lysosomal disease therapies may have limited efficacy and require regular intravenous infusions. We empathize with you.
Our Shielded Living Therapeutics™ platform has the potential to provide durable, consistent delivery of enzymes for the treatment of a range of lysosomal disease, including MPS-1or Fabry disease.