Applied Genetic Technologies Corporation, a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare eye diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted an orphan drug designation for its gene therapy product candidate for the treatment of achromatopsia caused by mutations in the CNGA3 gene.
Gene Therapy News
The data Demonstrate that SB-728-T Possesses Necessary Immunologic Properties to Support a 'Functional Cure' for HIV/AIDS
RICHMOND, Calif, USA. Sangamo BioSciences, Inc. (Nasdaq: SGMO) announced new data from its program to develop a 'functional cure' for HIV/AIDS in two presentations at the 20TH Conference on Retroviruses and Opportunistic Infections (CROI), held in Atlanta from March 3 to 6, 2013.
LONDON — Mice with up to 200 tumours have been completely cured of lung cancer using a “gene therapy” technique, scientists claim. By blocking production of a protein which drives the development of tumours, researchers were able to eliminate the tumours without any evidence of adverse sideeffects, the Daily Telegraph reported. The study found that the treatment was effective even after several rounds of therapy suggesting that the mice did not become resistant to it – a major problem for cancer doctors.