Unlocking Blocked Differentiation in MicroRNA-Deficient Embryonic Stem Cells
Why are pluripotent stem cells that have mutations to block the production of microRNAs unable to differentiate?
Gene Therapy
Transgenic Skin Grafts Treat 7 Year Old
A 7 year old boy with a skin condition called Junctional Epidermolysis Bullosa has been successfully treated using autologous transgenic skin grafts
Hold on Cellectis’ CAR-T Trial Lifted
The hold that the FDA placed on Cellectis’ CAR-T Phase I trials in September has been lifted, provided new trial protocols are implemented
“Be More Proactive in Seeking Out Advice” – Maciej Maselko
Maciej Maselko tells us about the troubles with public opinion of GMOs and how synthetic incompatibility may be the answer
Cell Biology: Cellular Power Outage
Deposits can accumulate in mitochondria, blocking energy production, but a newly described quality control system can mitigate the problem.
Alexion Wins Approval for Expanded Use of its Ultra-Expensive Drug for Ultra-Rare Disease
A flexible Food and Drug Administration has once again rescued a biotech company seeking a drug approval based on flawed clinical trial data.
Genetic Risk Factors for Breast Cancer Identified
72 new genetic variants that contribute to the risk of developing breast cancer have been identified in a by a major international collaboration.
3 Gene Therapy Biotechs to Watch Out For
The approval of some of the first CAR-T therapies recently, has further exploded the field of immune-oncology and gene therapy.
NICE Approves Novel GSK Gene Therapy
NICE has released draft guidance which would enable patients with ADA-SCID, or ‘bubble baby syndrome,’ to receive GSK’s gene therapy Strimvelis
The FDA Just Approved a Second Revolutionary Cancer-Killing Gene Therapy
Gilead Sciences’ gene therapy that uses a patient’s own genetically modified immune cells to treat certain types of non-Hodgkin lymphoma has been approved.
New Research Opens Door to ‘Functional Cure’ for HIV
Scientists have for the first time shown that a novel compound effectively suppresses production of HIV virus in chronically infected cells.
“Developing New Treatment Options for Unmet Medical Needs is the Cause That Inspires Us” – Julia Brown
Julia Brown explains why high standards of performance and high standards of ethical behaviour are essential to be successful in pharma.
The FDA Vote in Favour of Blindness Gene Therapy
The FDA has voted 16-0 in favour of an experimental gene therapy to treat patients with a rare kind of hereditary blindness.
Stem Cell Research Undermined by False Therapies
The reputation of stem cell research is being undermined by dodgy clinics offering unrealistic therapies for unproven diseases.
Gene Therapy for Blindness Appears Initially Effective
Spark Therapeutic’s Luxturna, a gene therapy for a rare, genetic form of blindness, will be reviewed by a U.S. Food and Drug Administration panel this week.
