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 ...
Patients treated with RGX-202 demonstrate consistent, robust microdystrophin expression and functional improvement compared to natural history in Phase ...
News Medical on MSN
AI Revolutionizes Solid Tumor Drug Development
Artificial intelligence (AI) is fundamentally reshaping the landscape of solid tumor (ST) drug development. By integrating multi-omics data, 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.
Researchers at Monash University in Melbourne, Australia, have uncovered the hidden code governing how genetic mutations affect RNA splicing and result in disease. The researchers were able to ...
Ashlee is an insurance editor, journalist and business professional with an MBA and more than 17 years of hands-on experience in both business and personal finance. She is passionate about empowering ...
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