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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 editing tool shows promise for treating diseases with multiple mutations

Science Daily: Pharmacology News

The system can be formulated completely as RNA, dramatically simplifying delivery logistics compared to traditional systems that use both RNA and DNA.

RNA 347
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Novartis Acquires Regulus Therapeutics, Expanding its RNA-Based Drug Pipeline

The Pharma Data

Novartis Finalizes Acquisition of Regulus Therapeutics, Strengthening Its Renal Disease Portfolio with Promising ADPKD Therapy Novartis AG, a leading global pharmaceutical company, has officially completed its acquisition of Regulus Therapeutics Inc. , a biotechnology firm known for its expertise in microRNA-targeting therapies.

RNA 52
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Antisense therapy restores fragile X protein production in human cells

Science Daily: Pharmacology News

An antisense therapy restores production of the protein FMRP in cell samples taken from patients with fragile X syndrome.

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Editing without 'cutting': Molecular mechanisms of new gene-editing tool revealed

Science Daily: Pharmacology News

Functional analysis based on these structures also revealed how a 'prime editor' could achieve reverse transcription, synthesizing DNA from RNA, without 'cutting' both strands of the double helix. Clarifying these molecular mechanisms contributes greatly to designing gene-editing tools accurate enough for gene therapy treatments.

RNA 290
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Thinking inside the box: How SmartLabs is supporting the science of tomorrow

BioPharma Drive: Drug Pricing

With new modalities such as gene and cell therapies, RNA therapeutics, complex biologics and more, today’s science brings unprecedented opportunities to address diseases that have long remained out of reach.

Science 141
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Biogen Advances Investigational SMA Therapy to Registrational Trials After Positive Phase 1 Data

The Pharma Data

Biogen Reports Promising Interim Phase 1 Results for Salanersen in Spinal Muscular Atrophy, Prepares for Registrational Trials Biogen has announced encouraging topline results from its Phase 1 clinical trial evaluating salanersen (BIIB115/ION306) , an investigational antisense oligonucleotide (ASO) therapy for spinal muscular atrophy (SMA).