Adverum Biotechnologies, Inc. (Nasdaq:ADVM), a clinical-stage gene therapy company targeting unmet medical needs in serious rare and ocular diseases, today announced that the United States (U.S.) Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to ADVM-053, a preclinical gene therapy candidate being investigated as a potential singleadministration treatment which has the potential to provide sustained levels of the C1 esterase inhibitor (“C1EI”) protein.
HAE affects approximately 8,000 individuals in the U.S. This disease is caused by a genetic mutation that results in low levels of C1 esterase inhibitor which can be associated with sudden swelling and edema of respiratory airways, gastrointestinal tract, and extremities.
“We are pleased to receive the Orphan Drug Designation for ADVM-053 from the FDA,” said Leone Patterson, interim president and chief executive officer of Adverum Biotechnologies. “We are committed to developing effective treatments for patients living with HAE and the support from the FDA will be invaluable towards this goal. We look forward to submitting our IND application in the fourth quarter.”
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