Remove Protein Production Remove RNA Remove Therapies
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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).

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New tRNA tech aims to rewrite rare disease treatment

Drug Target Review

One company leading the charge in this revolution is Alltrna , whose pioneering work in engineered transfer RNA (tRNA) therapeutics is offering new hope for patients with rare genetic diseases. These codons signal the premature end of protein translation, resulting in truncated or non-functional proteins.

Disease 80
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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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A Look Back

Dark Matter Blog

As soon as I learned about DNA and RNA, I wanted to be a molecular biologist. Last stops at RNA My last roles in biotech were where my original passion began: DNA and RNA. My last stop at Arrakis Therapeutics is with a company targeting RNA with small molecules. I wanted to use molecular biology to create drugs.

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Targeting a human protein may stop Ebola virus in its tracks

The Pharma Data

In fact, antiviral therapies designed to treat viruses such as hepatitis C (Sofosbuvir/SOVALDI) work by blocking viral polymerase. Back in 2018, researchers tested a broad-spectrum antiviral candidate called remdesivir/VEKLURY, which acts as a nucleotide decoy to get incorporated into the viral RNA genome and stop viral polymerase. .

Virus 52
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Codon Digest: Bacteria Resist Every Virus

Codon

Signal Transduction and Targeted Therapy. Basic Science A trailing ribosome speeds up RNA polymerase at the expense of transcript fidelity via force and allostery. Ribozyme-mediated RNA synthesis and replication in a model Hadean microenvironment. Assembly of metabolons in yeast using Cas6-mediated RNA scaffolding.

Virus 52
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Messenger RNAs with multiple “tails” could lead to more effective therapeutics

Broad Institute

Messenger RNAs with multiple “tails” could lead to more effective therapeutics By Corie Lok March 22, 2024 Breadcrumb Home Messenger RNAs with multiple “tails” could lead to more effective therapeutics Scientists have engineered long lasting mRNAs that increased therapeutic protein production in cells and animals.