Inozyme Pharma

This week our team had the privilege of welcoming a mother whose children live every day with #ABCC6Deficiency, a rare disease for which we’re working to develop a first-in-class treatment. Hearing firsthand about the challenges her family faces, the resilience of her children, and the impact this disease has on their daily lives brought into focus the reason we push forward through the challenges of drug development. Creating new therapies is an incredibly complex, lengthy, and rigorous journey, especially for rare diseases, where science must often break new ground. Collaborating closely with patients and families is not only essential for our science but also a powerful reminder of why we’re here—to bring hope where there currently is none. Thank you to this remarkable mom for sharing her family’s story, her kind note (an excerpt is shown below), and for reinforcing our commitment to making a meaningful difference. And thank you to the dedicated team at Inozyme Pharma, who work tirelessly every day to turn hope into real, life-changing possibilities. Together we will continue to advance innovation and work toward a better future for patients everywhere. #RareDisease #PatientImpact #Innovation #Community #ABBC6 #ENPP1 #Team #GACIGlobal

Thank you Brian Orelli, PhD and BiotechTV for discussing #calciphylaxis and the latest INZ-701 data with Dr. Sagar Nigwekar M.D., Co-Director of the Kidney Research Center at Massachusetts General Hospital and our CSO Yves Sabbagh, PhD at #Kidneywk.   We look forward to continuing to build on these encouraging results, which provide a strong foundation for advancing INZ-701 into a registrational trial in calciphylaxis.   #RareDisease #ASN2024

Today, we presented positive interim data from our Phase 1 SEAPORT 1 trial of our lead program, INZ-701, in patients with end-stage kidney disease at the American Society of Nephrology’s Kidney Week 2024.   The data show that INZ-701 elevated PPi levels in patients with end-stage kidney disease undergoing hemodialysis. Low PPi levels are associated with calciphylaxis, one of the most challenging complications for these patients. We plan to advance this program as we continue our work to develop the first treatment for people affected by this life-threatening condition.   You can read the full press release here: https://bit.ly/3BZE4AU   #KidneyWk #ASN

We’re pleased to share that Interim data from our ongoing Phase 1 trial SEAPORT 1, exploring the use of our lead candidate, INZ-701, in patients with end-stage kidney disease receiving hemodialysis, will be presented at the American Society of Nephrology’s Kidney Week 2024.     Asghar Chaudhry, M.D., South Florida Nephrology Research, will present the data on Thursday, October 24, 2024 at 10:00 a.m. Pacific time. Learn more: https://bit.ly/3BX6bjT #ASN #KidneyWeek

Great video from CheckRare of Dr. David Weber discussing ENPP1 Deficiency, caring for children with the disease, new data presented at the American Society for Bone and Mineral Research on disease progession and emerging treatments    #RareDiseases #ASBMR #ENPP1Deficiency

Welcome the newest member of our Board of Directors, Erik Harris! Mr. Harris brings extensive commercial expertise in the rare disease space to Inozyme Pharma, including his current position as Chief Commercial Officer at Ultragenyx. His experience will be helpful as we lay the groundwork for our transition into a commercial entity. Learn more at: bit.ly/4h91a8n  

Thank you CheckRare for speaking to our Chief Medical Officer, Kurt Gunter, MD, FASCP, about our recent data presented at the American Society for Bone and Mineral Research on the significant short- and long-term cardiovascular and musculoskeletal consequences of ENPP1 Deficiency and ABCC6 Deficiency in children.

We’re excited to see the launch of Genomics England’s Generation Study by the UK’s National Health Service (NHS), which will advance early detection and treatment of rare genetic conditions, including mutations in the ENPP1 and ABCC6 genes that can lead to Generalized Arterial Calcification of Infancy (GACI). The Generation Study will sequence the genomes of more than 100,000 infants, helping families to get better care and treatment for conditions that often go undiagnosed for years.  https://lnkd.in/epig6YDg

We are at the American Society for Bone and Mineral Research (ASBMR) 2024 Annual Meeting in Toronto, Canada, through this weekend!    We are thrilled to connect with colleagues, peers, and partners to discuss the many unmet needs in bone and mineralization diseases.     If you are attending, stop by our booth to learn more about our work to develop potential treatments for #ENPP1 and #ABCC6 Deficiencies.     #ASBMR2024 #RareDiseases 

We are presenting new data at the American Society for Bone and Mineral Research (ASBMR) 2024 Annual Meeting today through Sept. 30. This data underscores the urgent need for therapies that address the complications and long-term systemic effects of #ENPP1 Deficiency and #ABCC6 Deficiency.   We look forward to sharing four posters at the conference highlighting the significant burden associated with these #RareDiseases and the promise of our lead candidate, INZ-701.   Learn more: https://bit.ly/3XUtpjj    #ASBMR2024