Expression Therapeutics Announces IND Approval by the FDA for Hemophilia A Gene Therapy
- Category: DNA RNA and Cells
- Published on Tuesday, 26 May 2020 18:08
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ATLANTA, GA, USA I May 26, 2020 I Expression Therapeutics has announced that it has received clearance by the United States Food and Drug Administration (FDA) to proceed following review of its Investigational New Drug Application (IND) for clinical testing of its novel lentiviral vector-based gene therapy — ET3 — for hemophilia A. Hemophilia A is the most common severe congenital bleeding disorder and afflicts approximately 1 in 8,000 people. Without treatment, severe hemophilia is crippling and fatal by late adolescence to early adulthood.
The ET3 gene therapy developed by Expression Therapeutics combines innovative platform technologies in protein bioengineering and tissue-directed expression. ET3 consists of autologous mobilized peripheral blood stem and progenitor cells transduced with a recombinant lentiviral vector, encoding a bioengineered coagulation factor VIII transgene designed for high‑level expression at low vector copy number. In the ET3 trial, subjects will be preconditioned with low-dose stem and immune cell suppressing agents prior to receiving a single infusion of ET3. The high-expression factor VIII can correct the bleeding tendency in hemophilia A. The duration of ET3 activity is expected to be the normal lifetime of the patient. Expression Therapeutics expects to initiate a Phase 1 clinical trial — titled ET3-201 — at Emory University and enroll patients shortly.
"We are extremely pleased that the FDA has granted permission to proceed with this clinical study," said Trent Spencer, Ph.D., President of Expression Therapeutics and Director of the Cell and Gene Therapy Program in the Aflac Cancer and Blood Disorders Center at Emory University.
Hematopoietic stem and progenitor cell lentiviral gene therapy is currently the only approach that offers the possibility of permanent cure of hemophilia A and provides an opportunity to reach both pediatric and adult populations.
"We are very excited to get the hemophilia A clinical trial underway, the first of six gene therapy products currently under development at Expression Therapeutics," said Mohan Rao, Ph.D., CEO of Expression Therapeutics.
Expression Therapeutics is a biotechnology company based in Atlanta. The current therapeutic pipeline includes advanced gene therapies for hemophilia A and B; neuroblastoma, T-cell leukemia/lymphoma, and acute myeloid leukemia (AML); and primary immunodeficiencies such as hemophagocytic lymphohistiocytosis (HLH).
SOURCE: Expression Therapeutics