Biologics

Neurogene Gene Therapy Patient Dies After Dosing

Neurogene is reporting that a patient dosed in an ongoing Phase 1/2 gene therapy clinical trial for Rett syndrome has died following complications from a rare and life-threatening hyperinflammatory syndrome associated with systemic exposure to high doses of adeno-associated virus (AAV), the company says.

Neurogene says FDA has allowed it to proceed with the trial using a lower dose (1E15 vg) for both the pediatric and adolescent/adult cohorts. “Neurogene will also incorporate the 1E15 vg dose in its future registrational trial design planning,” it says.

NGN-401 is described as an investigational AAV9 gene therapy being developed as a one-time treatment for Rett syndrome. “It is the first clinical candidate to deliver the full-length human MECP2 gene under the control of Neurogene’s EXACT transgene regulation technology,” the company says. EXACT technology is designed to address a treatment approach that enables targeted levels of MECP2 transgene expression without causing toxic effects from overexpression that is often associated with conventional gene therapies, it says.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome