May 2024
Gene therapy technique used to restore hearing in congenitally deaf children
The gene therapy technique used to restore hearing in congenitally deaf children involves the use of adeno-associated viruses (AAVs) to deliver a functional copy of the gene responsible for producing otoferlin, a protein necessary for the transmission of sound signals from the inner ear to the brain. This gene therapy approach targets children with a […]
Why do CRISPR/Cas9 gene editing therapies fail after showing efficacy in rodent models
CRISPR/Cas9 gene editing therapies often show promising results in rodent models but can encounter several challenges when translated to human clinical settings, leading to failures or reduced efficacy. These challenges include differences in biology between species, immune responses, off-target effects, and delivery mechanisms. Differences in Biology Between Species Rodent models, such as mice, are extensively […]
Uzedy, Teva, and MedinCell’s Collaborative Effort Yields Success in Phase 3 Trials for Long-Acting Schizophrenia Treatment
Uzedy, Teva, MedinCell, long-acting, schizophrenia, phase 3 trials, treatment, collaboration, mental health, neuroscience, psychiatry, injectable medication.
Europe Temporarily Halts AstraZeneca’s Vaxzevria COVID-19 Vaccine on Company’s Request
AstraZeneca, Vaxzevria, COVID-19 vaccine, marketing authorization, suspension, Europe, European Medicines Agency (EMA), pharmacovigilance, safety concerns.
Revolutionizing Public Health: CBER’s Peter Marks and the Vision to Reduce FDA Visits
CBER, Peter Marks, FDA, public health, healthcare innovation, regulatory affairs, biologics, vaccines, medical devices, preventive measures.
Revival of Gene Editing Clinical Trials in China Post CRISPR Baby Scandal
CRISPR baby scandal, China, gene editing, clinical trials, restart, regulations, ethics, CRISPR-Cas9, genetic diseases, biotechnology.
Revolutionizing Biology: AlphaFold 3 by Isomorphic and DeepMind Expands Beyond Protein Predictions
AlphaFold 3, Isomorphic, DeepMind, protein predictions, biology revolution, artificial intelligence, machine learning, structural biology, drug discovery, disease research.
Lykos Therapeutics’ MDMA-Assisted Therapy for PTSD: Awaiting FDA Advisory Committee Review
Lykos Therapeutics, MDMA-assisted therapy, PTSD treatment, FDA Advisory Committee, clinical trials, drug approval process.
Pfizer Temporarily Suspends Dosing in Advanced Duchenne Muscular Dystrophy (DMD) Trial After Patient Fatality
Pfizer, Duchenne Muscular Dystrophy (DMD), clinical trial, dosing halt, patient death, late-stage study, temporary suspension, safety concerns, investigation.
Prologue Ventures: Revolutionizing Viral Protein Drug Development with $50 Million Investment
Prologue Ventures, viral protein drugs, $50 million investment, drug development, biotechnology, healthcare innovation.