A big pharma has entered the fray, teaming up with a biotech to create the first long-term stand-alone joint venture exploring the gene-editing technology Crispr-Cas9. German conglomerate Bayer AG and Switzerland-based CRISPR Therapeutics have created a joint venture that will explore the technology in three therapeutic areas.
A big pharma has entered the fray, teaming up with a biotech to create the first long-term stand-alone joint venture exploring the gene-editing technology Crispr-Cas9. German conglomerate Bayer AG and Switzerland-based CRISPR Therapeutics have created a joint venture that will explore the technology in three therapeutic areas.
The pair announced on Dec. 21 their London-based JV will develop therapies using new technology for "editing" defective genes to...
Regenxbio reported functional and biomarker data from an ongoing study of RGX-202, which it hopes to file for approval in Duchenne muscular dystrophy in 2026.
With its exon 53-skipping candidate already showing promise in 24-week data, Wave now has 48-week data showing improvements in muscle health and functional outcomes.
Encouraging results in younger children with Duchenne is keeping Regenxbio on track for a potential mid-2026 filing for its gene therapy, RGX-202.
The gene therapy for Duchenne muscular dystrophy passed the blockbuster sales threshold in 2024. Analysts said the drug’s overall risk/benefit profile still appears solid.
Otsuka bested Vera on Phase III immunoglobulin A nephropathy data for proteinuria, but analysts wonder if the difference is especially meaningful.
The company presented Phase I data for CART-EGFR-IL13Rα2, showing patient responses in the notoriously hard-to-treat and deadly brain cancer.
The low-dose atropine eye drop slowed the progression of pediatric myopia by 30%.
