Novartis announced it is scrapping plans to move OAV201, an investigational gene therapy for Rett syndrome, into clinical trials. “The totality of the pre-clinical data [on OAV201] does not support a path forward to human clinical trials,” according to the company’s third quarter report for this year.
Novartis Gene Therapies is planning to submit an investigational new drug application (IND) for OAV201, its Rett syndrome gene therapy candidate, by the end of the year, the company announced in a letter to the Rett community. If the IND is approved by the U.S. Food and Drug Administration…
Most animal studies needed to support plans to move AVXS-201 — AveXis’ investigational gene therapy for Rett syndrome — into clinical testing in patients are complete, the company reports. AveXis hopes to soon start meeting with the U.S. Food and Drug Administration (FDA) in advance of filing…
AveXis is postponing plans to open a clinical trial of its Rett syndrome gene therapy candidate AVXS-201. “Out of caution,” it will instead conduct additional preclinical tests and quality controls checks before asking for new regulatory approval for a study in patients. In a statement to the…
Preclinical data from mouse studies support initiating first-in-human clinical trials to explore the safety and effectiveness of the investigational gene therapy AVXS-201 in patients with Rett syndrome. Recent results have shown that one-time treatment with AVXS-201 is safe and well-tolerated in mice and non-human primates and supports the production…
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