What Inozyme does

Inozyme Pharma, Inc. was a Boston-based, publicly traded (NASDAQ: INZY) clinical-stage biopharmaceutical company founded in 2016 that developed therapeutics for rare diseases affecting the vasculature, soft tissue, and skeleton. Its primary and essentially sole asset was INZ-701, a late-stage enzyme replacement therapy designed to treat ENPP1 deficiency, an ultra-rare genetic disorder characterized by pathological calcification and impaired mineralization of bone and vasculature. The company was funded through a series of private venture rounds and a July 2023 public offering that raised approximately $60 million at $4.80 per share, bringing disclosed capital raised to well over $300 million across the company's history.

The business model was pre-commercial: Inozyme did not generate product revenue and instead monetized its R&D effort through eventual commercialization or — as ultimately occurred — strategic acquisition. The company's value proposition centered on addressing the underlying cause of ENPP1 deficiency through a targeted enzyme replacement approach, with a horizontal single-indication focus on ENPP1 rather than a broad pipeline. In July 2025, BioMarin Pharmaceutical Inc. completed an all-cash acquisition of Inozyme at a valuation of approximately $270 million, absorbing INZ-701 into BioMarin's broader rare disease enzyme therapy portfolio. Following the acquisition, Inozyme ceased to operate as an independent company.

Name

Inozyme

Legal name

Inozyme Pharma, Inc.

Website

https://inozyme.com

Company type

Public

Founded year

2016

Operating status

Acquired

Headcount range

11–50 employees

Short description

Inozyme Pharma is a Boston-based, clinical-stage biopharmaceutical company developing INZ-701, a late-stage enzyme replacement therapy for ENPP1 deficiency, a rare genetic mineralization disorder, acquired by BioMarin in July 2025 for approximately $270 million.

Ownership category

akta.pro rank

Product category

Rare Disease Biopharmaceuticals

NAICS

Biological Product (except Diagnostic) Manufacturing (325414)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Endocrinology & Metabolic Specialty Pharmaceuticals (HLAIACAD)

Keywords

• Rare disease therapeutics
• Enzyme replacement therapy
• Biopharmaceutical drug development
• ENPP1 deficiency treatment
• Mineralization disorder therapy

Headquarters

HQ city

Boston

HQ country

United States

HQ region

North America

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations

Core offering

Inozyme Pharma, Inc. was a clinical-stage biopharmaceutical company developing INZ-701, a late-stage enzyme replacement therapy for ENPP1 deficiency, a rare genetic disorder affecting mineralization of bones and vasculature. The company focused on therapeutics for diseases impacting the vasculature, soft tissue, and skeleton. It was acquired by BioMarin Pharmaceutical in July 2025 for approximately $270 million.

Product overview

Inozyme Pharma Inc. was a clinical-stage biopharmaceutical company developing therapeutics for rare diseases impacting the vasculature, soft tissue, and skeleton. The company's primary asset was INZ-701, a late-stage enzyme replacement therapy for ENPP1 deficiency. The company was acquired by BioMarin Pharmaceutical in 2025 in an all-cash transaction valued at approximately $270 million.

Differentiator

Problem solved

Functional benefit

Products and services

• INZ-701 A late-stage enzyme replacement therapy candidate for the treatment of ENPP1 deficiency, a rare genetic disorder affecting mineralization of bones and vasculature. It is intended for patients with ENPP1 deficiency, addressing the underlying enzymatic cause of vascular and soft tissue calcification and skeletal abnormalities.

Segments1 record

Ideal customer profiles1 record

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature1 record

Partnerships

One partnership is on record.

• BioMarin Pharmaceutical Inc.coreStrategic or Co-development Partner · 1 July 2025BioMarin Pharmaceutical completed its acquisition of Inozyme Pharma Inc. in an all-cash transaction valued at approximately $270 million. The acquisition includes Inozyme's late-stage therapy INZ-701, aimed at treating ENPP1 deficiency. This represents a significant strategic integration of Inozyme's rare disease pipeline into BioMarin's portfolio.

Scale indicators3 records

Recent moves6 records

Expansion highlights3 records

Broad incumbents

• Recordati Rare Diseases: Dedicated rare disease business unit of Recordati focused on metabolic and endocrine rare disorders. Comparable in addressing small patient populations with specialty distribution and pricing models similar to those INZ-701 would target.
• Genzyme (Sanofi): Pioneer in enzyme replacement therapy with multiple marketed products (Cerezyme, Fabrazyme, Myozyme/Lumizyme). Broader incumbent in rare genetic disorders with overlapping modality to Inozyme's enzyme replacement approach.
• Alexion / AstraZeneca Rare Disease: Established leader in rare disease therapeutics with a portfolio including complement inhibitors and enzyme replacement therapies. Comparable as a large strategic player operating in the same rare genetic disease space.
• Takeda Rare Disease (post-Shire acquisition): Global biopharmaceutical company with a major rare disease franchise inherited from Shire, including enzyme replacement therapies such as Replagal and Elaprase. Comparable as a large strategic serving similar rare disease patient populations.

Direct peers

• Swedish Orphan Biovitrum (Sobi): Dedicated rare disease biopharmaceutical company with a portfolio spanning enzyme replacement therapies (e.g., Kineret, Gamifant) and metabolic genetic disorders. Highly comparable focus on rare enzyme and metabolic conditions.
• Amicus Therapeutics: Rare disease biopharmaceutical company commercializing Galafold for Fabry disease and developing next-generation enzyme and gene therapies for genetic disorders. Comparable focus on enzyme-based mechanisms targeting rare metabolic conditions.
• BioMarin Pharmaceutical: Strategic acquirer of Inozyme and a leading rare disease biopharmaceutical company with a deep enzyme replacement therapy portfolio (e.g., Naglazyme, Vimizim, Aldurazyme). Directly comparable business model focused on enzyme replacement therapies for ultra-rare genetic disorders.
• Ultragenyx Pharmaceutical: Clinical and commercial rare disease biopharmaceutical company developing enzyme replacement and other therapies for ultra-rare genetic and metabolic disorders. Closely comparable approach to Inozyme's enzyme-based treatment of rare mineralization and metabolic conditions.

Emerging players

• Soleno Therapeutics: Clinical-stage rare disease company developing a therapy for Prader-Willi syndrome, an ultra-rare genetic disorder. Comparable as a small, focused clinical-stage rare disease company pursuing orphan indications with similar go-to-market dynamics.
• Travere Therapeutics: Rare disease biopharmaceutical company with metabolic and rare genetic disorder programs, including treatments for conditions affecting kidney and metabolic function. Comparable as a smaller, focused rare disease company developing therapies for metabolic disorders.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks5 records

Key highlights5 records

Customer concentration

• Xx.com/inozyme
• Facebookfacebook.com/InozymePharma
• LinkedInlinkedin.com/company/inozyme

Revenue estimate

Valuation estimate

Number of profiles

Profiles11 records

Funding overview

Funding rounds6 records

Investors11 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Inozyme