LEXINGTON, MA, USA I January 22, 2019 I Translate Bio (Nasdaq: TBIO), a clinical-stage messenger RNA (mRNA) therapeutics company developing a new class of potentially transformative medicines to treat diseases caused by protein or gene dysfunction, today announced that the Company has received verbal notification from the U.S. Food and Drug Administration (FDA) that the Agency has completed its review of the Company’s Investigational New Drug Application (IND) submission for MRT5201 for the treatment of ornithine transcarbamylase (OTC) deficiency and has additional clinical and nonclinical questions. The Company submitted the IND in December 2018 to support the initiation of a Phase 1/2 clinical trial in patients with OTC deficiency. The FDA is placing the IND on clinical hold until these questions are resolved. The Company expects to receive formal written communication with additional information from the FDA in the near future and plans to work with the FDA in an effort to resolve its questions as promptly as possible.
About MRT5201
MRT5201 is designed to treat patients with OTC deficiency by intravenous delivery of mRNA encoding fully functional OTC enzyme to the liver to enable the hepatocytes, the predominant type of liver cell, to produce the normal OTC enzyme. MRT5201 has been granted orphan drug designation for the treatment of OTC deficiency in the U.S. and EU.
About Translate Bio
Translate Bio is a clinical-stage mRNA therapeutics company developing a new class of potentially transformative medicines to treat diseases caused by protein or gene dysfunction. The Company’s MRT platform is designed to develop product candidates that deliver mRNA carrying instructions to produce intracellular, transmembrane and secreted proteins for therapeutic benefit. Translate Bio believes that its MRT platform and its MRT delivery systems are applicable to a broad range of diseases caused by insufficient protein production or where production of proteins can modify disease, including diseases that affect the lung, liver, eye, central nervous system and lymphatic system. The Company also believes its MRT platform and MRT delivery systems may be applied to various classes of treatments, such as therapeutic antibodies or vaccines in areas such as infectious disease and oncology. Translate Bio’s two lead programs are being developed as treatments for cystic fibrosis (CF) and ornithine transcarbamylase (OTC) deficiency. For more information about the Company, please visit www.translate.bio or on Twitter at @TranslateBio.
SOURCE: Translate Bio
Post Views: 31
LEXINGTON, MA, USA I January 22, 2019 I Translate Bio (Nasdaq: TBIO), a clinical-stage messenger RNA (mRNA) therapeutics company developing a new class of potentially transformative medicines to treat diseases caused by protein or gene dysfunction, today announced that the Company has received verbal notification from the U.S. Food and Drug Administration (FDA) that the Agency has completed its review of the Company’s Investigational New Drug Application (IND) submission for MRT5201 for the treatment of ornithine transcarbamylase (OTC) deficiency and has additional clinical and nonclinical questions. The Company submitted the IND in December 2018 to support the initiation of a Phase 1/2 clinical trial in patients with OTC deficiency. The FDA is placing the IND on clinical hold until these questions are resolved. The Company expects to receive formal written communication with additional information from the FDA in the near future and plans to work with the FDA in an effort to resolve its questions as promptly as possible.
About MRT5201
MRT5201 is designed to treat patients with OTC deficiency by intravenous delivery of mRNA encoding fully functional OTC enzyme to the liver to enable the hepatocytes, the predominant type of liver cell, to produce the normal OTC enzyme. MRT5201 has been granted orphan drug designation for the treatment of OTC deficiency in the U.S. and EU.
About Translate Bio
Translate Bio is a clinical-stage mRNA therapeutics company developing a new class of potentially transformative medicines to treat diseases caused by protein or gene dysfunction. The Company’s MRT platform is designed to develop product candidates that deliver mRNA carrying instructions to produce intracellular, transmembrane and secreted proteins for therapeutic benefit. Translate Bio believes that its MRT platform and its MRT delivery systems are applicable to a broad range of diseases caused by insufficient protein production or where production of proteins can modify disease, including diseases that affect the lung, liver, eye, central nervous system and lymphatic system. The Company also believes its MRT platform and MRT delivery systems may be applied to various classes of treatments, such as therapeutic antibodies or vaccines in areas such as infectious disease and oncology. Translate Bio’s two lead programs are being developed as treatments for cystic fibrosis (CF) and ornithine transcarbamylase (OTC) deficiency. For more information about the Company, please visit www.translate.bio or on Twitter at @TranslateBio.
SOURCE: Translate Bio
Post Views: 31
