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Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare eye diseases, and Synpromics Limited, a leading synthetic promoter development company backed by Calculus Capital, today announced they have entered into a broad, multi-target collaboration agreement.

Researchers have taken a significant step forward in developing gene therapy against a fatal neurodegenerative disease that strikes children. By delivering a working version of a gene to produce a key enzyme that is lacking in Batten disease, the scientists delayed symptoms and extended lifespan in dogs with a comparable disease.

The paper appears online today in Science Translational Medicine.

Researchers found inserting genetic material into intestinal cells to fix the disorder causing cystic fibrosis was effective in human cell cultures and mice, according to a new study.

Cystic fibrosis, which has no cure, is caused by mutations of the CFTR gene. The mutations cause cell proteins that channel chloride ions and water out of cells to malfunction, leading to the build-up of thick mucus that can clog airways or the gastrointestinal tract.

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.

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.

On January 10th, the British Medicine and Healthcare Regulatory Agency (MHRA) approved Genethon’s Phase I / II gene therapy clinical trial application in X-linked chronic granulomatous disease (XCGD) to start at Great Ormond Street Hospital in London. Genethon is also seeking approval for this multicenter trial in Germany, Switzerland and France. In total the trial will include 20 patients (5 per site) to be treated and followed up for 2 years.

New Rochelle, NY, March 8, 2013— Follow-up results from one of several groundbreaking gene therapy studies reported in 2008 in patients with an inherited form of retinal blindness confirmed the earlier positive results of substantial improvement in vision that has now persisted for more than 4 years. However, the new study* also showed that while gene therapy was able to improve patients' vision, it did not slow or stop the underlying disease process.

RetroSense Therapeutics, a biotechnology company dedicated to developing gene therapy approaches to vision restoration, announced today that the U.S. Patent and Trademark Office has issued a Notice of Allowance for U.S. patent application (No. 12/299,574), which broadly covers methods of restoring visual responses with a variety of optogenetic compounds.

According to the BBC (November 2012), geneticists, scientists and the entire medical profession are applauding the European Commission for bringing ‘a new era to medicine in the Western world’ by approving Glycera, a Dutch gene therapy medicine for treating a rare, inherited disorder known as LPLD. While gene therapy has been gaining momentum for years since French scientists first used it in 2000 to treat a rare immune disorder called SCID, Glycera’s approval may be the breakthrough needed to move gene therapy to sufferers of other rare diseases such as mesothelioma.