Brain-Directed Gene Therapy Advancements Post Neurogene Trial
A recent tragic event has prompted Neurogene to stop high-dose testing of their gene therapy for Rett syndrome after a young girl in the trial passed away. The U.S. Securities and Exchange Commission filing shared this news, conveying that the company is committed to continuing their research at a lower dose in light of this development.
In early November, the girl received a high dose of the therapy as part of the clinical trial, which caused her to exhibit signs of a systemic immune reaction. Following this incident, the U.S. Food and Drug Administration (FDA) permitted testing to proceed at a lower dose based on safety data from other participants. Although this unfortunate event has led to the cessation of high-dose testing in the trial, Neurogene remains focused on the overall study’s continuation at the lower dose.
Neurogene disclosed that the first four participants who received the lower dose of the gene therapy showed improvements in various areas, demonstrating the promise of the treatment. The therapy provides a copy of the gene responsible for Rett syndrome’s symptoms, aiming to address the deficits caused by the condition.
NGN-401 is part of a group of therapies being developed for Rett syndrome, alongside other treatments like TSHA-102 from Taysha Gene Therapies. While this adverse event is a setback, experts emphasize that it offers valuable insight into gene therapy safety and delivery methods. The goal remains to make progress in the field and learn from these experiences to advance treatment options for patients.
Neurogene’s decision to adjust the trial protocol underscores the complexities of gene therapy research and the importance of thorough safety evaluations. By carefully navigating these challenges, researchers aim to develop effective treatments for individuals affected by Rett syndrome and related conditions.
