Drug Development
Phacilitate’s annual event dawns as cell and gene therapies reach a new tipping point: the science has hit new heights just as regulatory and government policies spark momentum and frustration.
FEATURED STORIES
From opening new therapeutic mechanisms to repairing neuronal damage, investigational molecules from Ventyx Therapeutics, AC Immune, Gain Therapeutics and more could shape the future of Parkinson’s disease treatment.
As drug candidates discovered via AI move into later-stage clinical trials, the technology seems to be doing as promised: speeding drug development.
Following the hard-won success of early anti-amyloid drugs, a new generation of Alzheimer’s modalities—from tau-targeting gene silencers to blood-brain barrier delivery platforms—is entering the pipeline to anchor future combination therapies.
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Proof of concept for the Genetically Engineered Microbial Medicines (GEMM) platform came in a recent first-in-human, Phase I trial.
The company said the study demonstrated broad efficacy, showing statistically significant superiority to placebo over a total of 15 prespecified primary and secondary outcome measures.
ADI-001 generated positive early responses from three of four evaluable participants, two of whom achieved complete responses while one had a partial response that researchers logged as “near complete.”
Austin, Texas-based Aeglea Biotherapeutics reported what initially appears to be positive outcomes for its Phase III PEACE trial of pegzilarginase in Arginase 1 Deficiency (ARG1-D).
An investigational drug for Dravet syndrome is showing promise in treating the progressive form of genetic epilepsy.
Data is still coming in from South Africa and other regions about the new Omicron variant of COVID-19. Early analysis suggests it causes less severe disease than other variants.
The latest updates from Mesoblast Limited’s Phase III trial on rexlemestrocel-L show that the drug provides the highest benefit when used in patients with certain types of chronic heart failure.
The approval of Keytruda also now includes stage III melanoma following complete resection for pediatric patients 12 years and up.
A major factor behind the success of the smallpox eradication campaign was political commitment and leadership.
This afternoon, the U.S. Food and Drug Administration expanded the Emergency Use Authorization for Eli Lilly’s monoclonal antibody treatment combination of bamlanivimab and etesevimab.