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New tRNA tech aims to rewrite rare disease treatment

Drug Target Review

The landscape of genetic medicine is undergoing a profound transformation, driven by innovative approaches that challenge the traditional, disease-specific paradigms. My transition to biotech and rare diseases was deeply personal – my son was diagnosed with Duchenne muscular dystrophy in 2020. “My

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An immune factor in the brain plays critical roles in neuron function and aging

Broad Institute

A new study reveals that C1q can also enter neurons, where it influences protein production, and can accumulate within neurons over time. They also suggest that C1q influences learning, memory, and flexibility in the adult brain, potentially by interacting with specific complexes within neurons that impact protein production.

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

Broad Institute

Now, researchers at MIT and the Broad Institute of MIT and Harvard have developed RIBOmap, a technique that lets them pinpoint and visualize the precise locations of thousands of proteins being produced within an intact tissue and even individual cells. By Sarah C.P. Williams June 29, 2023 Credit: Zeng H, Huang J, Ren J, et al.

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

The Pharma Data

The study represents a major step toward addressing the unmet medical needs of individuals affected by this rare and severe neurodevelopmental disorder, which currently has no approved disease-modifying treatments. Encouraging results from the previously completed Phase 1/2 HALOS study support the continued development of ION582.

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Sino Biological: new US centre for bioprocessing

Drug Target Review

The company specialises in recombinant protein production and antibody development. All of Sino Biological’s products are independently developed and produced with a stringent quality management system, and include unique bioreagents addressing areas such as cell therapy, stem cell, and infectious disease research.

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Eloxx Pharmaceuticals to Report Third Quarter 2020 Financial Results and Provide Business Update on November 5, 2020

The Pharma Data

is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates (designed to be eukaryotic ribosomal selective glycosides) that are formulated to treat rare and ultra-rare premature stop codon diseases. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA.

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Biogen Announces Topline Results from the Tofersen Phase 3 Study and its Open-Label Extension in SOD1-ALS

The Pharma Data

Tofersen binds to SOD1 mRNA, allowing for its declination by RNase-H in an trouble to reduce conflation of SOD1 protein product. About Tofersen Tofersen is an antisense medicine being estimated for the implicit treatment of SOD1-ALS. Source link: [link].