FDA Grants Orphan Drug Designation to Temferon for Treatment of Glioblastoma Multiforme
"We expect that the FDA’s decision to grant Orphan Drug Designation to Temferon will enhance the development of our cell therapy, which we believe has the potential to address the unmet medical need of patients and strengthen our clinical program," said Pierluigi Paracchi, Chief Executive Officer of Genenta. "The Orphan Drug Designation program highlights the significant need for an efficacious therapy for patients suffering from glioblastoma multiforme."
Temferon is a proprietary cell therapy designed to reprogram the tumor microenvironment by delivering immunomodulatory molecules directly to tumors. Genenta is testing Temferon in an ongoing Phase 1/2a clinical trial in newly diagnosed patients with GBM who have an unmethylated MGMT gene promoter (uMGMT-GBM).
GBM is the most common malignant primary brain tumor and the most aggressive diffuse glioma, with unmethylated MGMT promoter status identified in approximately 60% of the GBM population.
The ODD program supports the development of treatments that address diseases affecting fewer than 200,000 people in
Genenta (www.genenta.com) is a clinical-stage biotechnology company engaged in the development of a proprietary hematopoietic stem cell gene therapy for the treatment of a variety of solid tumor cancers. Temferon™ is based on ex-vivo gene transfer into autologous Tie2+ hematopoietic stem/progenitor cells (HSPCs) to deliver immunomodulatory molecules directly via tumor-infiltrating monocytes/macrophages (Tie2 Expressing Monocytes - TEMs). Temferon™, which is under investigation in a phase 1/2a clinical trial in newly diagnosed Glioblastoma Multiforme patients who have an unmethylated MGMT gene promoter (uMGMT-GBM), is designed to reach solid tumors, induce a durable immune response not restricted to pre-selected tumor antigens nor type, and avoid systemic toxicity, which are some of the main unresolved challenges in immuno-oncology.
Statements in this press release contain “forward-looking statements,” within the meaning of the
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Source: Genenta Science