Genenta Science (NASDAQ: GNTA) is thrilled to announce that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Temferon™, their innovative cell-based platform. This ground-breaking technology harnesses the power of hematopoietic stem cells to deliver durable and safe treatments for solid tumors, specifically glioblastoma multiforme (GBM). This exciting development marks a major milestone in Genenta Science’s mission to revolutionize cancer treatment.
Genenta is thrilled to announce that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its cell therapy, Temferon, for the treatment of glioblastoma multiforme – a life-threatening brain cancer. This milestone marks a critical step forward in the development of a potential therapy to address the unmet medical need of patients. As CEO Pierluigi Paracchi puts it, “We believe this designation will greatly enhance our clinical program and bring us one step closer to providing a much-needed treatment for those suffering from this devastating disease.”
Genenta is pioneering a groundbreaking new cell therapy, Temferon, to revolutionize the treatment of glioblastoma (GBM). This proprietary therapy seeks to reprogram the tumor microenvironment by directly delivering immunomodulatory molecules to tumors. Currently, a Phase 1/2a clinical trial is being conducted in newly diagnosed uMGMT-GBM patients to evaluate the efficacy of Temferon. This pioneering approach has the potential to revolutionize the treatment of GBM, offering hope to patients and their families.
GBM, or Glioblastoma Multiforme, is a devastating and aggressive form of brain cancer. It is the most common malignant primary brain tumor, and affects roughly 60% of the GBM population with an unmethylated MGMT promoter status. This formidable foe has caused much distress and hardship for many, making it a priority for the medical community to find effective treatment options.
The Orphan Drug Designation program (ODD) allows treatments for rare diseases, which affect fewer than 200,000 people in the US, to receive various forms of assistance. This includes eligibility for federal grants, tax credits for qualified clinical trials, prescription drug user fee exemptions, and a seven-year marketing exclusivity period upon FDA approval. By providing much-needed support, the ODD program is helping to find cures for diseases that would otherwise go untreated.
About Genenta Science
Genenta, a clinical-stage biotechnology company, is developing a revolutionary hematopoietic stem cell gene therapy, Temferon™, to treat a variety of solid tumor cancers. Temferon™ represents a major breakthrough in immuno-oncology as it uses ex-vivo gene transfer to infuse immunomodulatory molecules directly into tumor-infiltrating monocytes/macrophages, thus allowing for a targeted, durable immune response that is free of systemic toxicity. Currently, Temferon™ is undergoing a phase 1/2a clinical trial in newly diagnosed Glioblastoma Multiforme patients with an unmethylated MGMT gene promoter.