May 2024
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 […]
Differences and potential advantages of aficamten compared to BMS’s Camzyos
The recent analysis from Cytokinetics has highlighted several key differences and potential advantages of aficamten compared to Bristol Myers Squibb’s Camzyos, both of which are cardiac myosin inhibitors used in the treatment of obstructive hypertrophic cardiomyopathy (HCM). Key Differences Pharmacodynamic Profile: Aficamten, being a next-generation myosin inhibitor, has a different pharmacodynamic profile compared to mavacamten […]
Merck Pauses Phase 3 TIGIT Trial Due to Immune-Mediated Adverse Events
Merck, Phase 3 Trial, TIGIT, Immune-mediated Adverse Events, Discontinuations
Preliminary CRISPR-Based HIV Treatment Study Shows Limited Success
CRISPR, HIV, gene editing, treatment, study, curative potential, early study, limited success
Maze Therapeutics Secures New Partnership for Pompe Drug Development After Sanofi Deal Collapse
Maze Therapeutics, Pompe drug, partnership, Sanofi deal collapse, drug development, genetic diseases
Moderna’s Diversification Strategy Hindered by FDA Delay in RSV Vaccine Review
Moderna, FDA, RSV vaccine, diversification strategy, vaccine review, delay
Duchenne Muscular Dystrophy: Potential Inclusion in Newborn Screening Programs
Duchenne muscular dystrophy, newborn screening, medical experts, decision-making, early diagnosis, treatment options.
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.
Moderna’s RSV Vaccine Decision Postponed by FDA to Late May
FDA, Moderna, RSV vaccine, decision, delay, May
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.