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

The Pharma Data

The goal is to boost SMN protein production more effectively and offer a more convenient, once-a-year dosing schedule , thereby improving long-term disease management and patient compliance. a leader in RNA-targeted therapeutics.

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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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Protein Science

Sygnature Discovery

We’ve successfully tackled targets for: Unprecedented membrane proteins Intrinsically disordered proteins Refolding from inclusion bodies Large protein complexes Labelled proteins for NMR studies RNA structures by crystallography Across our capabilities Construct Design Structure-guided sequence design.

Science 52
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Scientists pinpoint where thousands of individual proteins are made in intact tissue and single cells

Broad Institute

Each colored dot in a vast RIBOmap readout represents one mRNA molecule as it is being used to produce the corresponding protein — a process called translation. This means we’re seeing only RNA that is actively being used to produce proteins,” said Jingyi Ren, a co-first author of the new paper and a graduate student in Wang’s lab.

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Protein Science & Structural Biology

Sygnature Discovery

We’ve successfully tackled targets for: Unprecedented membrane proteins Intrinsically disordered proteins Refolding from inclusion bodies Large protein complexes Labelled proteins for NMR studies RNA structures by crystallography Across our capabilities Protein Expression & Purification Vast diversity of proteins produced.

Science 52
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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.