Protein Production Small Molecule Therapies 
Codon
JULY 28, 2025
The gene therapy is designed to protect this infant from the neurological disorders that otherwise would have resulted from this rare, and often fatal, condition. And while matching a gene therapy to its targeted mutation is an intricate and nuanced process, much boils down to two core elements: a vector and its cargo.
Dark Matter Blog
JULY 16, 2021
My last stop at Arrakis Therapeutics is with a company targeting RNA with small molecules. I was enticed by Arrakis’ founder Jen Petter to serve in one more operational role, leading the research group at Arrakis as the company launched as one of the first biotechs to target RNA with drug-like small molecules.
This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.
The Pharma Data
MARCH 23, 2022
In fact, antiviral therapies designed to treat viruses such as hepatitis C (Sofosbuvir/SOVALDI) work by blocking viral polymerase. Without the stop sign, viral transcription no longer follows the correct order, which has a negative impact on viral protein production,” says Fang. The problems with Ebola polymerase.
Codon
MARCH 20, 2023
Signal Transduction and Targeted Therapy. Point-of-care peptide hormone production enabled by cell-free protein synthesis. Marine biofilm engineered to produce current in response to small molecules. Peroxisomal metabolic coupling improves fatty alcohol production from sole methanol in yeast. Gene Therapy.
Codon
MAY 30, 2023
This means that IscB base editors might be easier to package inside of AAVs, which can only store 4,700 bases of DNA for gene therapies. 3/ Cell Membrane Editor A cell is a lipid shell with lots of DNA, proteins, and RNA molecules inside. Molecular Therapy. Base editors made from Cas9 are about twice as big.
Codon
MAY 30, 2023
This means that IscB base editors might be easier to package inside of AAVs, which can only store 4,700 bases of DNA for gene therapies. 3/ Cell Membrane Editor A cell is a lipid shell with lots of DNA, proteins, and RNA molecules inside. Molecular Therapy. Base editors made from Cas9 are about twice as big.
Codon
MAY 29, 2025
These abilities are precisely what make them promising as potential therapies, particularly as we find ways to enhance their safety profiles. While small-molecule drugs continue to play a critical role in modern medicine, we are witnessing a shift toward the increasing development of amino acid-based therapeutics.
Expert insights. Personalized for you.
Let's personalize your content