Regenxbio reaches an agreement with FDA on a path toward a potential accelerated approval of Navsunli (clemidsogene lanparvovec-sngl), the first gene therapy for treating Hunter syndrome, or mucopolysaccharidosis type II.
An opinion article calls on FDA to establish a formal regulatory framework for evaluating vaccines as potential interventions to prevent dementia, arguing that the absence of clear guidance is hindering the launch of large-scale clinical trials.
Regenxbio reaches an agreement with FDA on a path toward a potential accelerated approval of Navsunli (clemidsogene lanparvovec-sngl), the first gene therapy for treating Hunter syndrome, or mucopolysaccharidosis type II.
An opinion article calls on FDA to establish a formal regulatory framework for evaluating vaccines as potential interventions to prevent dementia, arguing that the absence of clear guidance is hindering the launch of large-scale clinical trials.
Merck says FDA has approved an expanded indication for its pneumococcal conjugate vaccine, Capvaxive, allowing its use in children and adolescents aged 2 to 17 years who have completed a primary pneumococcal vaccination series and are at increased risk for pneumococcal disease due to chronic medical conditions.
FDA approves a Beam Therapeutics IND for BEAM-304, allowing the company to begin clinical testing of its experimental base-editing treatment for phenylketonuria. The clearance enables the launch of a Phase 1/2 trial evaluating BEAM-304, an in vivo gene-editing therapy designed to directly correct disease-causing mutations in the phenylalanine hydroxylase (PAH) gene, according to the
