Pharmaceutical Technology
JANUARY 19, 2023
This DUX4 protein abnormal expression leads to modifications in gene expression in muscle cells which are associated with progressive muscle function loss in FSHD patients. Avidity Biosciences’ AOC 1020 is intended to reduce the DUX4 mRNA and DUX4 protein expression in muscles in these patients.
Drug Discovery World
JULY 10, 2023
Artificial intelligence (AI), gene editing, synthetic biology and more, are beginning to prove their usefulness in drug design and development, taking therapies away from the one-size-fits-all approach, towards more definable targets that operate more effectively. This was done to assess the accuracy of the team’s machine-learning algorithms.
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pharmaphorum
FEBRUARY 9, 2021
million ($22 million) in second-round financing as it prepares to start clinical trials of its lead drug candidate INT-1B3 for solid tumours. InteRNA is focusing on the development of gene-silencing drugs based on microRNA, naturally occurring, non-coding strands of RNA that are thought to regulate gene expression in cells.
Roots Analysis
NOVEMBER 3, 2023
NAD + is involved in a wide range of cellular processes, including energy metabolism, DNA repair, and gene expression. There are several types of NAMPT inhibitors, including small molecules, peptides, and antibodies. NAMPT inhibitors work by blocking the activity of NAMPT, which in turn reduces the production of NAD +.
The Pharma Data
OCTOBER 15, 2020
On the clinical side, cell and gene therapies must find places where small molecules, antibodies or other traditional approaches may not be the best option. Small molecule and antibody therapies haven’t been able to do that.”. So far, 25 people have been treated in a Phase I/II trial.
pharmaphorum
JANUARY 10, 2023
Xork is designed as a pre-treatment to overcome one of the key limitations to adeno-associated virus (AAV) based gene therapies like AT845, namely that some patients have existing antibodies against the AAV vector that interfere with the treatment. The post Astellas licenses companion drug for Pompe gene therapy appeared first on.
Drug Discovery World
FEBRUARY 6, 2023
Typically, the data obtained from such studies is large and high dimensional with thousands of molecules being measured for each individual sample requiring special statistical, data science, and bioinformatics skills to integrate the information and correlate it with the clinical outcome of interest.
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