2021 Disease Trials 
Broad Institute
APRIL 5, 2025
The organization has honored Liu for the development of the gene editing platforms base editing and prime editing, which can correct the vast majority of known disease-causing genetic variations and have already been used in at least 15 clinical trials, with life-saving results.
PPD
AUGUST 16, 2023
The COVID-19 pandemic rapidly accelerated the adoption of hybrid and decentralized clinical trial (DCT) models. However, as the world settles into its post-pandemic state and returns to pre-pandemic paradigms in many areas, the pharmaceutical industry remains dedicated to moving beyond traditional, centralized clinical trial constructs.
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Drug Target Review
JUNE 21, 2023
Most rare diseases are caused by a single gene defect, but severity can vary considerably among patients. Modifier genes can help explain that variability and can alter or even prevent disease onset and progression, making them appealing therapeutic targets. However, the identification of these genes is challenging.
Drug Target Review
JULY 4, 2023
In practice, some patients may be left managing a syndrome associated with the therapy along with the disease. 2 However, when dosed at the MTD, ADCs display improved efficacy over small molecules in oncology trials. 2 However, when dosed at the MTD, ADCs display improved efficacy over small molecules in oncology trials.
The Pharma Data
AUGUST 30, 2021
Positive high-level results from the FoCus Phase III trial in Wilson disease showed ALXN1840 met the primary endpoint with a statistically significant improvement in daily mean copper mobilisation from tissues, demonstrating superiority compared with standard-of-care (SoC) treatments. Wilson disease. 1, 2, 3.
The Pharma Data
MAY 31, 2022
the completion of enrollment of its Phase 1 (Ph1), open-label trial of pluripotent stem cell-derived dopaminergic neurons in patients with Parkinson’s disease (PD). Ph1 clinical trial is to evaluate the safety, tolerability, and?preliminary?efficacy?of of BRT-DA01 in patients with Parkinson’s disease.?. preliminary?efficacy?of
Chemical Biology and Drug Design
OCTOBER 10, 2023
Abstract Human immunodeficiency virus (HIV) causes acquired immunodeficiency syndrome (AIDS), a lethal disease that is prevalent worldwide. million people worldwide were living with HIV in 2021. Several agents are in clinical trials, including apricitabine, racivir, elvucitabine, doravirine, dapivirine, and elsulfavirine.
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