The START pilot program aims to accelerate the development of novel drug and biological products for rare diseases rAAV-Olig001-ASPA was selected as one of a few CBER-regulated products based on eligibility criteria, including clinical benefit for rare diseases with...
In a recent discussion, Peter Marks, the director of the FDA’s Center for Biologics Evaluation and Research, and Janet Woodcock, former FDA deputy commissioner, highlighted the critical need for regulatory flexibility in developing rare disease therapies. They...
Gene therapy can be a promising therapy for both genetic and acquired brain disease. However, the limiting problem in brain gene therapy is delivery to the brain followed by regulation of the expression of the transgene. Present day gene vectors do not cross the...
–Supplemental New Drug Application (sNDA) submission based on Phase 3 study of vibegron 75mg (GEMTESA) demonstrating statistically significant reductions in daily micturition and urgency episodes– –If approved, vibegron will be the first and only beta-3 agonist for...
CHENGDU, China, May 10, 2024 /PRNewswire/ — WestGene, a biotech company dedicated to mRNA technology, announces a historic milestone with the FDA IND approval of its mRNA therapeutic cancer vaccine, WGc-043. This landmark achievement marks the world’s...
