Clinical Trials | May 26, 2026
Lilly posts strong early clinical results for one-shot gene-editing cholesterol therapy
A one-shot gene-editing cholesterol therapy could soon be a reality
A one-shot gene-editing cholesterol therapy could soon be a reality
The agreement targets a long-standing technical hurdle in the field: maintaining chemical purity as RNA constructs extend beyond 100 nucleotides
Genome editing holds extraordinary promise for treating previously incurable genetic diseases
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
Four-year research collaboration combines Pfizer’s deep experience in global drug development, including programs utilizing messenger RNA (mRNA), lipid nanoparticles (LNP), and gene therapy, with Beam’s leadership in base editing and mRNA/LNP delivery technologies
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
Growing CRISPR genome editing portfolio supports researchers on path to future therapeutic applications
The collaboration will combine Metagenomi’s next-generation CRISPR-based and other novel gene editing systems with Moderna’s mRNA and LNP technologies
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