Pfizer Inc. won U.S. approval for its first gene therapy, expanding into a promising field of medicine that has proved to be a challenging business. The treatment, called Beqvez, gained clearance ...
An iEcure spokesperson said the company anticipates dosing the first patient its phase 1 study of the experimental ...
The first patient received the therapy ... navigates the pivotal phase of awaiting FDA approval for Upstaza, its gene therapy candidate, the financial landscape of the company is a key metric ...
N.J., biopharmaceutical company on Tuesday said the FDA set a target action date of Nov. 13 for a decision on Upstaza, which won European Union approval in 2022 as the first gene therapy for AADC ...
In an important step toward more effective gene therapies for brain diseases, researchers have engineered a gene-delivery vehicle that uses a human protein to efficiently cross the blood-brain barrier ...
The green light from the FDA to begin the Phase 3 clinical trial marks a pivotal milestone as OCU400 becomes the first gene therapy to progress ... meet 2026 BLA and MAA approval targets.
The Food and Drug Administration (FDA ... the first months of life and affects physical, mental, and behavioral functions. Eladocagene exuparvovec is a one-time, recombinant adeno-associated virus ...
Welcome to 2024's regulatory tracker for Fierce Pharma. On this page, we're recording the regulatory progress of in-market products, including expansions into key geographies and new indications.
Upstaza, which delivers a functional copy of the DDC gene, is already approved in Europe, Great Britain, and Israel.