FDA-approved drug may prevent Cytokine Release Syndrome (CRS) associated with cell therapy

Recently published research findings suggest that we may be able to prevent Cytokine Release Syndrome (CRS) through the oral administration of an FDA-approved drug. CAR-T therapy is a promising new approach to cancer treatment. However, a devastating side effect of CAR-T cell infusion is CRS. The patient’s infused activated T cells release interferon gamma (IFN-γ)

FDA approves first gene therapy for hereditary disease — Spark Therapeutics’ Luxturna

On December 19, 2017, the US FDA approved a new gene therapy to treat children and adults with a genetic condition that often results in blindness. Spark Therapeutics’ Luxturna (voretigene neparvovec-rzyl) is a one-time gene therapy product that treats biallelic RPE65 mutation-associated retinal dystrophy, an inherited retinal disease that frequently progresses to complete blindness. Approval

FDA and the future of medicine: Advancing regenerative medicine options for patients with safety and science at the forefront

On November 16, 2017, the US FDA announced a policy framework for regenerative medicine, releasing two final and two draft guidance documents. These documents reflect the agency’s commitment to modernize the system to incorporate promising cell and gene therapies and provide a structure for these options to be introduced in a fast, effective, and safe

With CAR-T approval, FDA steps up efforts at regulating stem cell clinics

About two months and two weeks after the FDA’s advisory committe recommended for approval Novartis’ CAR-T therapy for the treatment of  relapsed or refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL), the FDA followed up with an official approval. Tisagenlecleucel – or, as the therapy is commonly known, Kymriah – will head

Big CAR-T Win: The FDA recommends Novartis’ CAR-T therapy for approval

A commercial and scientific first, and a breakthrough for the emerging field of CAR-T therapy, occurred this week with a pivotal regulatory win for cell therapy. On June 12, 2017, the FDA unanimously recommended for approval Novartis’ CAR-T therapy for the treatment of  relapsed or refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL),

FDA Introduces ‘Regenerative Medicine Advanced Therapy’ Drug Designation

At the end of 2016, the United States Congress passed the 21st Century Cures Act. Addressing some of the provisions included in the Act, the FDA followed through, on January 17, 2017, by introducing a new designation process for regenerative advanced therapies, called Regenerative Medicine Advanced Therapies (RMAT). On a fundamental level, this new designation provides an