Fast Track Designation was granted for BB-301 following FDA review of positive interim clinical study results and proprietary Responder Analysis ...
Fast Track Designation was granted for BB-301 following FDA review of positive interim clinical study results and proprietary Responder Analysis planned for use in pivotal study for BB-301 BB-301 has ...
About BB-301 BB-301 is a novel, modified AAV9 capsid expressing a unique, single bifunctional construct promoting co-expression of both codon-optimized Poly-A Binding Protein Nuclear-1 (PABPN1) and ...
REGENXBIO completes pivotal enrollment and initiates commercial production in Duchenne gene therapy programme: Rockville, Maryland Friday, October 31, 2025, 18:00 Hrs [IST] REGENX ...
Ultragenyx (RARE) stock is down 40% since March 2024 amid ongoing financial struggles. See more insights on its growth ...
The researchers argue that traditional vaccine development methods, which rely heavily on laboratory experimentation and long ...
Patients treated with RGX-202 demonstrate consistent, robust microdystrophin expression and functional improvement compared to natural history in Phase ...
News-Medical.Net on MSN
AI indelibly transforms solid tumor drug development
The integration of single-cell RNA sequencing (scRNA-seq) with AI has become a cornerstone for deciphering tumor heterogeneity. For instance, in pancreatic ductal adenocarcinoma (PDAC), spatial ...
Morning Overview on MSN
An ancient fossil breaks nature’s secret life code
Scientists have long believed that a universal genetic code serves as a blueprint for all life on Earth, dictating the structure and function of organisms from the simplest bacteria to complex humans.
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