Genetic Disease Genetics Genomics Protein 
Drug Discovery World
APRIL 17, 2024
JMB: Regulatory RNAs (or regRNAS) are molecules that directly control the expression of nearby protein-coding genes. CAMP4 is exploiting these regulatory interactions to specifically control the expression of genes tied to disease. What diseases are you currently targeting? What are the challenges of drug discovery in this area?
pharmaphorum
JULY 20, 2022
The Cambridge, Massachusetts biotech has discovered that areas of the non-coding parts of the human genome – referred to by its chief executive Josh Mandel-Brehm (pictured above) as the “dark side” of the genome – actually produce regRNAs that control the expression of the 2% that codes for proteins.
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pharmaphorum
SEPTEMBER 9, 2020
Geneticist Dr Charles Steward has spent his career studying the human genome – but his work became much more personal when his children were diagnosed with severe neurological diseases. Charlie told pharmaphorum how his search for a genetic cause has led him to straddle the divide between scientist and patient advocate.
pharmaphorum
SEPTEMBER 9, 2020
Geneticist Dr Charles Steward has spent his career studying the human genome – but his work became much more personal when his children were diagnosed with severe neurological diseases. Charlie told pharmaphorum how his search for a genetic cause has led him to straddle the divide between scientist and patient advocate.
pharmaphorum
OCTOBER 5, 2022
Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder, from US biotech miRecule. billion deal in 2018.
Drug Discovery World
MAY 11, 2023
However, she also presented thoughts in a technical update, Targeted Protein Degradation – Progress, Direction and Prospects. Ubiquigent’s Sheelagh Frame, said: “It was wonderful to share experiences and thoughts about building better workplaces alongside some other inspiring women in life science.”
Drug Discovery World
APRIL 30, 2024
Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. This is essential to its use as a therapeutic agent and gives the technology a vast versatility making it suitable to treating a wide range of diseases – especially those that have high protein expression.
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