We work to save lives.
Our Story Begins with Science
The founders of Inozyme Pharma observed the integral role of pyrophosphate, or PPi, in rare, debilitating, and life-threatening calcification disorders. These diseases can lead to over-calcification of soft tissues and under-mineralization of bone, with devastating consequences for patients.
In 2016, Inozyme Pharma acquired technology developed in the laboratory of Demetrios Braddock and licensed from Yale University. The name Inozyme Pharma reflects the company’s goal of developing an enzyme replacement therapy using inorganic PPi to treat calcification disorders = Inozyme.
Based on a deep understanding of the biology of calcification, the company is developing new medicines with the potential to drastically improve the standard of care and change treatment paradigms.
We Focus on Calcification Disorders
We are focused on devastating and debilitating rare diseases that largely impact infants, children, and young adults.
In certain rare, life-threatening, and devastating genetic disorders, PPi levels are below the normal levels. These low levels can lead to over-mineralization of soft tissues and/or under-mineralization of bone, which cause damage and disease to bones, arteries, and organs.
Our scientists created and tested INZ-701, an enzyme replacement therapy designed to increase PPi levels and regulate calcification in multiple metabolic diseases, including ENPP1 Deficiency and ABCC6 Deficiency.
ENPP1 Deficiency can manifest as two separate calcification disorders:
- Generalized arterial calcification of infancy (GACI) type 1
- Autosomal recessive hypophosphatemic rickets type 2 (ARHR2)
ABCC6 Deficiency can manifest as two separate calcification disorders:
Given the central role of low PPi in various metabolic diseases, as well as our knowledge of rare bone diseases and the established role of ERT, we plan to expand and diversify its development programs to benefit additional patient populations, including those living with calciphylaxis, and chronic kidney disorders.
We are just getting started. We combine all the tools necessary for success:
- Solid science
- Strong business acumen
- Well-defined regulatory strategy
- Robust financial backing