A pioneering gene therapy could help treat a rare seizure disorder called Dravet syndrome, according to new clinical trial ...
Bayer has discontinued an early-stage clinical gene therapy for a rare genetic disorder in favor of a similar candidate. | Bayer has discontinued an early-stage clinical gene therapy for a rare ...
In a strikingly blunt briefing, a senior FDA official accused uniQure of pushing “distorted” data to mask a “failed” therapy ...
UniQure’s Path for Huntington’s Gene Therapy Clouded by Ethical Questions as Potential Phase 3 Looms
While the FDA appears to be adamant that uniQure conduct a sham surgery–controlled Phase 3 trial before AMT-130 can be ...
Days after FDA Commissioner Marty Makary appeared to malign uniQure’s AMT-130 in an interview with CNBC, the agency confirmed ...
CRISPR gene therapy Casgevy can nearly eliminate sickle cell crises, but difficult stem‑cell collection is delaying access ...
UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new avenues for CF therapy.
Gene therapies have the potential to cure some diseases, but they are extraordinarily expensive. Location can also be a big ...
AFTX-201 uses a proprietary capsid to deliver a human BAG3 transgene, aiming to restore cardiac function with lower doses than conventional gene therapies.
Successful program buildout prioritizes defined patient flow, infusion access, cold-chain capabilities, and accountability ...
Intellia is now able to continue both Phase III trials of nex-z. Credit: Piotr Swat / Shutterstock.com (Piotr Swat / Shutterstock.com) The US Food and Drug Administration (FDA) has released the ...
A Frisco mom’s yearslong fight led to a gene therapy breakthrough for the rare SLC6A1 disorder. Her son Maxwell is the first patient treated and is already showing promising progress.
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