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What’s in Today’s Brief? (September 18th Preview)
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Roche Acquires 89bio for $3.5 Billion to Target Liver Disease
Roche has agreed to purchase 89bio in a deal valued up to $3.5 billion, acquiring the biotech's late-stage drug candidate pegozafermin for metabolic dysfunction-associated steatohepatitis (MASH). The acquisition at a 79% premium aims to strengthen Roche's presence in the cardiometabolic disease sector. Pegozafermin, an FGF21 analog designed to reduce liver fat and fibrosis, is in phase 3 clinical trials. This move follows recent FDA approvals and regulatory openness toward MASH therapies, highlighting the competitive landscape to address this common liver condition linked to obesity.
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AI Drives Genome Design and Accelerates Gene Therapy Development
Researchers have harnessed artificial intelligence to design complete functional bacteriophage genomes and to assist CRISPR gene-editing experiments. AI models trained on millions of viral genomes generated novel phage variants capable of killing bacteria, opening new avenues against antibiotic resistance. Separately, Stanford Medicine introduced CRISPR-GPT, an AI tool that guides researchers in designing gene-editing studies, streamlining development workflows and lowering barriers to genomic therapies. These advances represent significant strides in synthetic biology and genetic medicine, enhancing precision, speed, and innovation.
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Advancements in Cancer Immunotherapy and Targeted Therapeutics
Several studies reveal promising approaches to combat treatment-resistant cancers. Researchers developed antibody-bottlebrush conjugates that deliver high drug loads to tumors, potentially surpassing traditional antibody-drug conjugates in efficacy and safety. Another study identified lymph nodes as key orchestrators of immune responses essential for successful immunotherapy. Emerging fusion proteins combining immune checkpoint blockade with selective tumor microenvironment activation have shown preclinical activity. These innovations hold potential to improve outcomes in hard-to-treat cancers such as melanoma, neuroblastoma, and osteosarcoma.
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Breakthroughs in Rare Diseases and Precision Medicine
Regeneron achieved a pivotal phase 3 win with garetosmab for fibrodysplasia ossificans progressiva (FOP), an ultra-rare bone disease, reducing aberrant bone growth significantly and gearing up for FDA filing. Roivant and Priovant reported Phase 3 success for brepocitinib in dermatomyositis, planning regulatory submission. UC San Diego and Yale teams discovered safer small nuclear RNA base editing as an alternative to CRISPR, offering potential new gene therapies. These developments underscore progress in targeted treatment approaches for rare, debilitating conditions.
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Needle-Free Vaccine Delivery via Skin Stretching Technique
Researchers have demonstrated that mechanically stretching the skin activates immune surveillance and increases skin permeability, enabling more effective vaccine delivery without needles. Using a suction pressure device, topical vaccines applied during skin stretching in mice produced stronger immune responses than traditional subcutaneous injections. This novel approach utilizes the opening of hair follicles and local immune activation, offering a promising platform for painless immunization and potentially broader drug or cell therapy delivery.
...and 5 more selected Biotech stories in today’s full edition.
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