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Orphan drug biopharmaceutical company: Inozyme Pharma, Inc. (INZY)

Inozyme Pharma, Inc. (NASDAQ: INZY) was founded in 2015 and is headquartered in Boston, Massachusetts, USA. It has 24 full-time employees. It is a rare disease biopharmaceutical company dedicated to developing novel therapies to treat blood vessels, soft tissues and bones. Abnormal mineralization disease.

Inozyme Pharma

Inozyme Pharma, Inc. (INZY):

The founder of Inozyme Pharma has observed the role of biological pathways in the mineralization process, which is the root cause of various rare, debilitating and life-threatening metabolic diseases. These diseases can lead to excessive calcification of soft tissues and insufficient bone mineralization, with devastating consequences for patients. Based on a deep understanding of mineralization biology, Inozyme Pharma is developing new drugs with the potential to greatly improve the standard of care and change the treatment paradigm.

Inozyme Pharma’s in-depth understanding of the biological pathways involved in mineralization, we are seeking the development of therapeutic agents to solve the root causes of these debilitating diseases. It is well known that the two genes ENPP1 and ABCC6 play a key role in key mineralization pathways, and defects in these genes can lead to abnormal mineralization. Inozyme Pharma initially focused on developing a new therapy to treat rare genetic diseases of ENPP1 and ABCC6 deficiency.

  • ENPP1 deficiency is a rare occurrence caused by mutations in the ENPP1 gene. Inozyme Pharma believes that 11,000 to 12,000 people worldwide suffer from ENPP1 deficiency. The condition is inherited as a recessive feature, in which genetic mutations result in reduced or missing ENPP1 enzyme activity. Deficiency of ENPP1 leads to low plasma pyrophosphate levels (precursors of PPi and AMP-adenosine), which can lead to neointimal hyperplasia, and is associated with higher early mortality and long-term morbidity. The manifestations of ENPP1 deficiency include acute infancy, progressive childhood and adulthood.
  • ABCC6 deficiency is a rare, inherited and inherited congenital metabolic error caused by mutations in the ABCC6 gene, affecting more than 67,000 people worldwide. ABCC6 deficiency is a recessive inheritance, in which genetic mutations lead to reduced or missing ABCC6 protein activity. ABCC6 deficiency can lead to decreased plasma levels of PPi and is associated with pathological mineralization of blood vessels and entire soft tissues throughout the body, leading to serious morbidity, including blindness, potentially life-threatening cardiovascular complications, and skin calcification.

INZ-701 aims to restore the normal balance of PPi and adenosine for ENPP1 deficiency, and to replace other genetically inadequate ENPP1 enzyme functions, and to provide treatments for other diseases, such as ABCC6 deficiency, involving low PPi levels.

Inozyme Pharma, Inc. (INZY) financing:

  • On November 15, 2017, in the A round of financing, Inozyme Pharma received a lead investment from Longitude Capital, and a $49 million investment from New Enterprise Associates (NEA), Novo Ventures and Sanofi Ventures.
  • On November 9, 2018, in Series B financing, Inozyme Pharma received an investment of USD 33.7 million.
  • On April 10, 2019, C round of financing, Inozyme Pharma received the lead investment of Pivotal bioVenture Partners and Sofinnova Investments, and 7 institutions including New Enterprise Associates (NEA), Novo Holdings, RA Capital Management, Longitude Capital and other 7 institutions followed up with US$67 million in funding .

Inozyme Pharma, Inc. (INZY) investment:

Inozyme Pharma, Inc. (NASDAQ: INZY) was listed on IPO on 7/24/2020 and listed on Nasdaq. The issue price is $16.00, 5 million shares are issued, and the capital is 80 million US dollars, underwritten by BofA Securities/ Cowen/ Piper Sandler, Wedbush PacGrow Joint.

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