Inozyme Pharma's ENPP1 Deficiency Data Presentation at CHOP
Inozyme Pharma's Upcoming Presentation on INZ-701
Inozyme Pharma, Inc. (Nasdaq: INZY) is proud to announce that Dr. Kurt Gunter, the Senior Vice President and Chief Medical Officer, will present significant interim data related to INZ-701 during the prestigious CHOP Cardiology Annual Meeting. This meeting will take place in Orlando, and it promises to be a notable event for those involved in pediatric cardiology and rare diseases.
Presentation Details
The presentation will be centered around the findings from the company’s Expanded Access Program (EAP), which studies INZ-701's effects on infants and young children suffering from ENPP1 Deficiency. The details of this presentation are as follows:
- Title: Impact of the Enzyme Replacement Therapy, INZ-701, in Children with ENPP1 Deficiency: Experience from an Expanded Access Program
- Presentation Number: 10 – Poster Session
- Date: Saturday, February 22, 2025
- Time: 10:00 – 11:00am ET
- Presenter: Dr. Kurt Gunter, M.D.
Understanding ENPP1 Deficiency
ENPP1 Deficiency is a rare and progressive disease that significantly impacts blood vessel function, soft tissues, and bone health. Infants diagnosed with generalized arterial calcification of infancy (GACI Type 1) face severe risks, with many not surviving past six months. Those affected later in life may develop additional challenges, including osteomalacia and various musculoskeletal disorders, which can lead to significant pain and mobility issues.
This condition also has a broader impact. Hearing loss, calcification in arteries and joints, and cardiovascular problems can accompany the disease due to the underlying genetic mutations. Current statistics estimate that approximately 1 in 64,000 pregnancies worldwide may result in biallelic mutations leading to ENPP1 Deficiency. However, due to the severe nature of symptoms associated with even monoallelic mutations, the actual prevalence may be notably higher.
The Role of INZ-701
INZ-701 represents Inozyme Pharma’s commitment to addressing the challenges posed by rare diseases like ENPP1 Deficiency. This investigational therapy is an enzyme replacement therapy (ERT) that injects a version of the ENPP1 enzyme, aimed at increasing the levels of inorganic pyrophosphate (PPi) and adenosine. By enhancing these key molecules, INZ-701 seeks to restore mineralization processes and address the overgrowth of smooth muscle cells that can occur in blood vessels.
Currently under clinical investigation, INZ-701 has the potential to offer hope not only for ENPP1 Deficiency but also for related conditions such as ABCC6 Deficiency and calciphylaxis, targeting diseases that result from deficiencies in the PPi-Adenosine pathway. This innovative therapy aims to significantly improve the quality of life for patients by addressing the underlying causes of their conditions rather than just treating the symptoms.
About Inozyme Pharma
Inozyme Pharma is actively forging a path in biopharmaceutical innovation, focusing on rare diseases that impair bone and vascular health. The company harnesses expertise in the PPi-Adenosine Pathway, where the ENPP1 enzyme plays a crucial role. Disruptions in this pathway can lead to severe health conditions, making the developments surrounding INZ-701 not just pioneering, but crucial in the fight against these devastating diseases.
Engaging with Inozyme
For further details on Inozyme Pharma’s innovative work and ongoing trials, visit their official website or connect with them on social media platforms. This engagement helps spread awareness about ENPP1 Deficiency and the potential therapies that can transform the lives of those affected.
Frequently Asked Questions
What is INZ-701?
INZ-701 is an enzyme replacement therapy designed to treat ENPP1 Deficiency and other related conditions by enhancing PPi and adenosine levels in the body.
What is ENPP1 Deficiency?
ENPP1 Deficiency is a rare genetic condition that causes serious complications affecting bone health, blood vessels, and overall developmental health in affected individuals.
When will Inozyme present the interim data?
The presentation of the interim data for INZ-701 will take place on February 22, 2025.
Where will the presentation occur?
The presentation will be held at the CHOP Cardiology Annual Meeting in Orlando.
Is there a therapy approved for ENPP1 Deficiency?
No, currently, there are no approved therapies specifically for ENPP1 Deficiency.
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