| Ticker | Status | Jurisdiction | Filing Date | CP Start | CP End | CP Loss | Deadline |
|---|
| Ticker | Case Name | Status | CP Start | CP End | Deadline | Settlement Amt |
|---|
| Ticker | Name | Date | Analyst Firm | Up/Down | Target ($) | Rating Change | Rating Current |
|---|
Inozyme Pharma, Inc. (NASDAQ:INZY) ("Inozyme" or the "Company"), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of pathologic mineralization and intimal proliferation, highlighted the inclusion of Generalized Arterial Calcification of Infancy (GACI), which is caused by mutations in the ENPP1 or ABCC6 genes, in the Genomic England's Generation Study. This research initiative, embedded within the UK's National Health Service (NHS), was developed to advance early detection and treatment of rare genetic conditions, with the goal of setting a new standard for newborn screening programs.
"We commend Genomics England for recognizing the crucial importance of early detection of GACI as we advance INZ-701 into clinical trials in infants. Including ENPP1 and ABCC6 in the initial list of genes selected for this groundbreaking study represents the hard work and dedication of the advocates, families, and healthcare professionals who work tirelessly for those living with this disease," said Catherine Nester, R.N., Inozyme's senior vice president of healthcare professionals and patient engagement. "The Generation Study has the potential to make a significant impact on the creation of more comprehensive newborn screening programs that save lives by empowering families to find appropriate care for their babies as quickly as possible."
The Generation Study is scheduled to begin in late 2023, with the goal of sequencing the genomes of more than 100,000 infants and paving the way for potential widespread implementation of whole-genome sequencing in newborn screening. Genomics England collaborated with NHS experts as well as scientists, healthcare professionals (HCPs), and people living with rare conditions to select 223 individual conditions for its current screening program.
GACI is a rare genetic disorder that is fatal in 50 percent of affected infants by six months of age due to severe and pathological vascular calcification and neointimal proliferation (overgrowth of smooth muscle cells inside blood vessels). The disease can lead to the potential failure of major organs, such as the heart, lungs, and kidneys, and it is caused by ENPP1 and ABCC6 Deficiencies, rare disorders for which there are currently no approved treatments. Inozyme is developing INZ-701, an enzyme replacement therapy, for the treatment of rare disorders like GACI that impact the vasculature, soft tissue, or skeleton.
To learn more about Genomics England and its Generation Study, read the full announcement here.
About INZ-701
INZ-701, a recombinant Fc fusion protein, is an ENPP1 enzyme replacement therapy in development for the treatment of rare disorders of the vasculature, soft tissue, and skeleton. In preclinical studies, the experimental therapy has shown potential to prevent pathologic mineralization and intimal proliferation (the overgrowth of smooth muscle cells inside blood vessels), which can drive morbidity and mortality in devastating genetic disorders such as ENPP1 Deficiency and ABCC6 Deficiency. INZ-701 is currently in clinical trials for the treatment of ENPP1 Deficiency and ABCC6 Deficiency.
Posted In: INZY
