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FDA proposes new approval pathway for rare disease gene therapy
Will consider data that includes "the experience of individuals," FDA commissioner says ...
After teasing a new regulatory process for personalized genetic medicines at the end of last year, the FDA today unveiled draft guidance for an approval pathway that could see custom CRISPR therapies, ...
As cell and gene therapies move into more hospitals and cancer centers, health system leaders are learning that clinical ...
UCLA researchers use lipid nanoparticles to insert full CFTR gene into airway cells, restoring function and opening new ...
Forget Vertex Pharmaceuticals -- CRISPR Therapeutics is a better buy right now.
A gene therapy that patients breathe in has been found to shrink lung tumours by inserting immune-boosting genes into ...
The Food and Drug Administration aims to evaluate treatments for rare diseases based on plausible evidence that they would work — without requiring a clinical trial first.
The FDA is now expected to decide on the application by Aug. 23. ・The company intends to manufacture the therapy at its new gene therapy manufacturing facility in Bedford, Massachusetts, if approved.
Despite innovative new research, many cell and gene therapies do not make it all the way to the patients. Researchers and ...
Japanese Patent Covers Acclaim-3 Clinical Trial Combining REQORSA Gene Therapy with Genentech, Inc.'sTecentriq® AUSTIN, Texas, Feb. 23, 2026 /PRNewswire/ -- Genprex ...
For patients with rare and devastating neurological disorders, treatment options can seem few and far between. However, gene therapy research, led by Dr. Russell Lonser and Dr. Brad Elder, is hoping ...
Boehringer Ingelheim is discarding an inhaled gene therapy designed to treat cystic fibrosis (CF) after terminating a phase 1/2 trial. | Boehringer Ingelheim is discarding an inhaled gene therapy ...
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