Disease Protein Production RNA 
Drug Target Review
JUNE 9, 2025
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
The Pharma Data
JUNE 25, 2025
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. These reductions were sustained through one year , highlighting salanersen’s potential to meaningfully slow disease progression.
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Codon
JULY 28, 2025
Researchers have identified nearly 10,000 monogenic diseases , or conditions caused by errors in a single gene. Fortunately, as readily as these diseases are discovered and characterized, so too are many of their cures. Fortunately, as readily as these diseases are discovered and characterized, so too are many of their cures.
Broad Institute
JULY 19, 2024
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.
Broad Institute
JUNE 29, 2023
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.
The Pharma Data
OCTOBER 14, 2020
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.
Dark Matter Blog
JULY 16, 2021
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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