Clinical Research Gene Editing RNA 
AuroBlog - Aurous Healthcare Clinical Trials blog
SEPTEMBER 17, 2024
We are witnessing a revolution in healthcare, driven by advances in genetics, Omics, RNA and CRISPR gene-editing technology, to deliver precision and personalised medicine, said Kiran Mazumdar-Shaw, executive chairperson, Biocon and Biocon Biologics. Biology is opening up new frontiers in medicine.
XTalks
SEPTEMBER 4, 2024
Alnylam Pharmaceuticals announced promising results from its HELIOS-B Phase III clinical trial evaluating vutrisiran, an investigational RNA interference (RNAi) therapeutic for treating transthyretin amyloidosis with cardiomyopathy (ATTR-CM). As of March 2024, the FDA has approved six small interfering RNA (siRNA) therapies.
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Worldwide Clinical Trials
MAY 30, 2024
An area of interest has been deciphering ALS’s genetic underpinnings and delivering functional copies of dysfunctional genes to the patient. The targeted ASOs aim to downregulate the expression of gene mutations that are associated with gain-of-function toxicity that leads to motor neuron loss in some ALS cases.
Pharmaceutical Technology
FEBRUARY 20, 2023
This level of homogeneity within the clinical trial population may prevent the identification of variable therapy responses within population subgroups. Gene therapy, while in its infancy, could be a gamechanger for cancer treatment and certain rare diseases and advancements in gene-editing technology relies on knowledge of viral factors.
XTalks
FEBRUARY 11, 2025
Gene therapy has shown promise in treating cancers that are particularly difficult to manage such as neuroblastoma and Wilms tumor using CAR-T cell therapy, CRISPR-Cas9 gene editing and RNA-based interventions to target genetic drivers of disease.
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