The Clinical Trial Application (CTA) approval clears the way for the company to launch trial locations in France and Romania
PBGENE-DMD is designed to permanently correct mutations in the dystrophin gene between exons 45 and 55, the “hot-spot” region affecting roughly 60% of DMD patients
As CRISPR and prime editing applications advance, researchers increasingly rely on longer guide RNAs to boost editing efficiency and reduce off-target effects
Under the terms of the agreement, Mammoth Biosciences will receive an upfront payment of USD 40 million, potential future payments of more than one billion USD upon successful achievement of certain research, development, and commercial milestones
The collaboration will combine Metagenomi’s next-generation CRISPR-based and other novel gene editing systems with Moderna’s mRNA and LNP technologies
This linear, covalently closed, double-stranded DNA offers a rapid, cell-free alternative to traditional plasmid DNA, addressing a critical bottleneck in nucleic acid therapeutics development
The applications aim to launch Phase Ib/II trials for relapsed/refractory multiple myeloma and primary plasma cell leukemia, respectively
The study is being conducted under a USFDA IND, has received U.S. Fast Track Designation for SLE-ITP, and has obtained IND approval from China’s Center for Drug Evaluation
The expanded mbDNA portfolio delivers enhanced stability, reduced immunogenicity, and broad compatibility with gene insertion and expression technologies
This is the first industry-academia collaboration in the country, initiated through the CSR route, for advancing research in the areas of immuno-therapeutics and regenerative medicines
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