gene therapy
Ultragenyx Advances Gene Therapy for Glycogen Storage Disease Type Ia Following Phase 3 Success
Ultragenyx, gene therapy, glycogen storage disease type Ia, DTX401, Phase 3 study, GlucoGene, rare genetic diseases
BioNTech Expands ADC Alliance with MediLink, Investing $1.8 Billion
BioNTech, MediLink, ADC alliance, $1.8 billion, oligonucleotide therapy, gene therapy, molecular biology
Revolutionizing CAR-Ts: The Future of Automation and Decentralization in Cell and Gene Therapy Manufacturing
Automation, Decentralization, CAR-Ts, Cell and gene therapy manufacturing, Advanced therapies, Personalized medicine, Regulatory compliance, Quality management systems, Supply chain logistics, Digital solutions, Real-time monitoring, Patient-centered care
Evotec Discontinues Gene Therapy Operations, Shuts Down Austria Facility
Evotec, gene therapy, Austria, facility closure, strategic decision, business operations, research and development, biotechnology
How do the 12 FDA-approved gene therapies break down by medical indication
The 12 FDA-approved gene therapies cover a range of medical indications, primarily focusing on genetic disorders, cancers, and hematological conditions. Here’s a breakdown of these therapies by their medical indications: Genetic Disorders: Luxturna (voretigene neparvovec-rzyl): Approved for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy, which leads to vision loss and may […]
Revolutionizing Healthcare: Streamlining Cell and Gene Therapy Manufacturing (Part 1)
Cell therapy, gene therapy, manufacturing, biotechnology, healthcare innovation, personalized medicine, production challenges, regulatory compliance, scalability, cost-effectiveness.
Revolutionizing Medicine: The Evolution and Future of Cell and Gene Therapy Manufacturing
Cell therapy manufacturing, gene therapy manufacturing, biomanufacturing, advanced therapies, CAR-T cell therapy, stem cell therapy, viral vectors, bioreactors, automation, scale-up, regulatory compliance, personalized medicine.
Revolutionary Gene Therapy Advancements: Early Trial Results from Leading Biotech Companies
ASGCT24, gene therapy, early trial results, Freeline, Rocket, Creyon, biotech companies, genetic diseases, hemophilia, liver diseases, Parkinson’s disease.
Pioneering Gene Therapy Restores Hearing in Children with Otoferlin-Linked Deafness
Gene therapy, otoferlin-related deafness, hearing restoration, children, genetic mutation, clinical trial, auditory function.
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 […]