Spark Therapeutics is preparing to seek the first-ever US FDA approval for a gene therapy after several people who couldn't read a menu in a dimly lit restaurant or take a walk alone at dusk had their vision restored in a Phase III clinical trial testing SPK-RPE65.
Spark Therapeutics is preparing to seek the first-ever US FDA approval for a gene therapy after several people who couldn't read a menu in a dimly lit restaurant or take a walk alone at dusk had their vision restored in a Phase III clinical trial testing SPK-RPE65.
Two-thirds of the 21 people with RPE65-mediated inherited retinal dystrophies (IRDs) who were treated with SPK-RPE65 in a 31-patient Phase...
Optimizing Drug Pipelines With Biophysical Methods”, your view into how using biophysical techniques can enhance and streamline processes throughout the drug discovery and validation cycle.
CEO Paul Stoffels said gaining US clearance for an IND for its novel CAR-T product was demanding, but now opens up a pathway towards a pivotal study starting in 2025.
A final rejection of Leqembi could also spell the same fate for Lilly’s rival drug but public outcry and demand for Alzheimer’s therapies might force the regulator’s hand
GSK will assume full control of mRNA shots in infectious disease, allowing CureVac to cut costs and pivot its research to oncology.
Lilly's first Phase III triplet win opens the door to a label expansion in BTK-pretreated CLL, with analysts suggesting Jaypica could attain blockbuster status. However, the window to capitalize may be brief as next-generation BTK degraders close in with head-to-head trials.
Lilly's first Phase III triplet win opens the door to a label expansion in BTK-pretreated CLL, with analysts suggesting Jaypica could attain blockbuster status. However, the window to capitalize may be brief as next-generation BTK degraders close in with head-to-head trials.
