StemCell Therapy

AVROBIO, a US-based company, developing lentiviral-based gene therapies, has announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation for the Companys investigational gene therapy, AVR-RD-02, for the treatment of Gaucher disease.

Get a complete list of the presentations,here.

What is AVR-RD-02: AVR-RD-02 consists of the patients own hematopoietic stem cells, genetically modified to express glucocerebrosidase (GCase), the enzyme that is deficient in Gaucher disease. It is an ex vivo lentiviral-based investigational gene therapy. The stem cells are genetically modified to express functional glucocerebrosidase (GCase), which reduces levels of glucosylceramide and glucosylsphingosine, the accumulated substances which cause the symptoms of Gaucher disease.

How advanced is AVR-RD-02 development program: The Company is actively recruiting in Canada for its Phase 1/2 clinical trial of AVR-RD-02, which seeks to evaluate the safety and efficacy of the therapy in patients with Type 1 Gaucher disease.

What is an orphan drug designation: Orphan-drug designation provides special status to a drug or biological product to treat a rare disease or condition upon request of a sponsor. For a drug to qualify for orphan designation both the drug and the disease or condition must meet certain criteria specified in the ODA and FDAs implementing regulations at 21 CFR Part 316. Orphan designation qualifies the sponsor of the drug for various development incentives of the ODA, including tax credits for qualified clinical testing. A marketing application for a prescription drug product that has received orphan designation is not subject to a prescription drug user fee unless the application includes an indication for other than the rare disease or condition for which the drug was designated.

What is the gene therapy pipeline like for the treatment of Gaucher disease: There are only a few gene therapy candidates that are being developed for Gaucher disease.

For further information, check out the report here

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Gene Therapy Pipeline Update: AVROBIO Receives Orphan-Drug Designation | Roots Analysis - The News Brok

This entry was posted on September 8, 2020 at 12:53 am and is filed under Gene therapy. You can follow any responses to this entry through the RSS 2.0 feed. Both comments and pings are currently closed. |