At the Forefront of Transformative Therapies

Inozyme is a leader in developing therapies for rare diseases.

Our Pipeline

Our lead candidate, INZ-701, is in clinical development for the potential treatment of ENPP1 Deficiency and ABCC6 Deficiency, which are driven by low levels of inorganic pyrophosphate (PPi) andadenosine. Currently, there are no therapeutic options for patients suffering from ENPP1 Deficiency and ABCC6 Deficiency –  two rare disorders. We are pioneering the development of effective therapies for these patients and their families.

Learn more about our clinical studies.

One Pathway - Multiple therapeutic opportunities

Planned expansion of INZ-701 into additional indications marked by low PPi and adenosine.

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Pipeline - image updated March 2024


Stage of Development
   Next Anticipated Milestone
Phase 1/2
Phase 2/3
ENPP1 Deficiency
Topline Data
Q1 2023
ABCC6 Deficiency
Topline Data
Q1 2023
Exploratory Trial Initiation
Diseases of Neointimal Proliferation
Generate preclinical proof-of-concept
Diseases of Abnormal Mineralization
Gene Tx
Generate preclinical tox. data

ENPP1 and ABCC6 are key transmembrane proteins regulating mineralization and neointimal proliferation. A properly functioning pathway is illustrated in the 3 steps below:

Genetic mutations to the ENPP1 or ABCC6 genes result in low levels of PPi and adenosine. Low levels of PPi lead to abnormal mineralization and pathologic calcification, and low levels of adenosine lead to intimal proliferation –which can drive systemic and progressive conditions affecting the vasculature, skeleton, and organs such as the skin and eyes.


Native ENPP1 is bound to the cell membrane with the active enzymatic portion of the protein outside the cell. We fused this domain with an antibody Fc fragment to construct INZ-701. In contrast from native ENPP1, INZ-701 is a soluble protein designed to circulate throughout the body and cleave extracellular ATP into PPi and AMP, a precursor of adenosine.


PPi is a potent inhibitor of mineralization which, in balance with phosphate (Pi), enables normal physiologic mineralization. PPi inhibits crystallization of Pi and calcium to form hydroxyapatite and prevents further propagation by binding to the surface of forming hydroxyapatite crystals.

Low levels of PPi lead to pathologic mineralization and pathological calcification in areas of the body where it should not occur, referred to as abnormal mineralization. Abnormal mineralization occurs in the vasculature and soft tissue, including multiple organ systems, and results in disease.


Adenosine is a critical regulator of vascular smooth muscle proliferation. Adenosine is a potent vasodilator, acting through direct mechanisms by binding to adenosine receptors.  Low levels of adenosine lead to the narrowing and obstruction of blood vessels caused by neointimal proliferation and potential development of cardiovascular disease.