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Are You Using Next-Gen Sequencing to inform AAV product and process quality? Here are 4 reasons you should

BioPharma Drive: Drug Pricing

Next-generation sequencing allows for critical insights into gene therapy products, which can help streamline and accelerate everything from process development and production to regulatory approval.

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Targeting KRAS Diversity: Covalent Modulation of G12X and Beyond in Cancer Therapy

Covalent Modifiers

Insights from successful KRASG12C targeting informed the design of molecules addressing other mutations, often in a covalent manner. This breakthrough led to the development and approval of sotorasib (AMG510) and adagrasib (MRTX849), revolutionizing the treatment of KRASG12C-dependent lung cancer.

Therapies 214
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Gene editing extends lifespan in mouse model of prion disease

Broad Institute

We are hopeful the results might inform the future development of a one-time treatment for this important class of diseases. We realized it was this golden opportunity to use human genetics to inform base editing, Minikel said. Theres still a long way to go to make this a therapy, Minikel said. Nature Medicine.

Disease 144
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An ancient RNA-guided system could simplify delivery of gene editing therapies

Broad Institute

An ancient RNA-guided system could simplify delivery of gene editing therapies By Corie Lok February 27, 2025 Breadcrumb Home An ancient RNA-guided system could simplify delivery of gene editing therapies The programmable proteins are compact, modular, and can be directed to modify DNA in human cells.

RNA 66
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New gene delivery vehicle shows promise for human brain gene therapy

Broad Institute

Gene therapy could potentially treat a range of severe genetic brain disorders, which currently have no cures and few treatment options. Since we came to the Broad we’ve been focused on the mission of enabling gene therapies for the central nervous system,” said Deverman, senior author on the study. “If

Therapies 137
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Novel Bispecific therapies in oncology

SugarCone Biotech

Our skepticism was based on our understanding of the biology of those pathways and informed by observations made by the Antonia lab, several Roche groups and others that TGF- expression is sufficient to prevent T cell activation. The lead program here was bintrafusp alfa, now terminated.

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Scaling Phage Therapy

Codon

Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. Read it on our website here. Illustration by David S. Fast forward to 2023.

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