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New tRNA tech aims to rewrite rare disease treatment

Drug Target Review

To do this, Alltrna leverages the unique biology of tRNA and its AI-enabled platform to design engineered tRNAs that can restore full-length protein production in diseases caused by shared genetic mutations, such as premature termination codons. Alltrna focuses on a mutation-driven approach.

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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. Some patients demonstrate suboptimal clinical outcomes or experience treatment fatigue due to ongoing, frequent dosing regimens.

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Gene editing extends lifespan in mouse model of prion disease

Broad Institute

His lab had just developed base editing, a gene-editing approach that makes single-letter changes in DNA and can shut down protein production using strategies including installing a stop signal in the genetic code. In 2018, Liu, who works on the same floor as Minikel and Vallabh at Broad, approached them and proposed a collaboration.

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First Patient Dosed in Phase 3 REVEAL Trial of ION582 for Angelman Syndrome

The Pharma Data

This mechanism has the potential to partially restore UBE3A protein production in neurons, addressing the root cause of the disorder. ION582 works by inhibiting a natural antisense transcript that suppresses the expression of the paternal UBE3A allele, thereby allowing it to be reactivated.

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A Visual Guide to Gene Delivery

Codon

LNPs measure between 50 and 120 nanometers and primarily carry functional RNA — such as mRNA or guide RNAs for CRISPR — rather than DNA.

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Dyadic International Rebrands as Dyadic Applied BioSolutions

The Pharma Data

The C1 expression platform , originally derived from a fungus used in industrial enzyme production, has been optimized by Dyadic for robust, scalable, and cost-efficient protein production. Its counterpart, the Dapibus™ platform , is designed for producing proteins in a non-GMP environment for food and industrial use.

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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.