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Category Archives: Gene Therapy

Krystal files for IPO to move gene therapies into clinic | FierceBiotech – FierceBiotech

Posted: August 23, 2017 at 3:41 am

Krystal Biotech has filed to raise about $35 million in a Nasdaq IPO. The money will equip Krystals ex-Intrexon executive team to file an IND for its rare skin disease gene therapy in the first quarter of next year. That gene therapy, KB103, is designed to treat dystrophic epidermolysis bullosa, a rare disease that makes the skin fragile and susceptible to blisters and tears. Continue reading

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Apic Bio Launches to Advance First-in-Class Gene Therapy for … – Business Wire (press release)

Posted: at 3:41 am

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Apic Bio, Inc., a pre-clinical stage gene therapy company leveraging its proprietary platform to advance therapies to treat rare diseases with complex mechanisms, in particular Alpha-1 Antitrypsin Deficiency (Alpha 1), launched today with an initial investment led by the venture philanthropy arm of the Alpha-1 Foundation and a private investor with the disease. Its lead product, APB-101, targets the liver via an AAV delivered Dual Function Vector (df-AAV) whereby the Z-AAT protein is silenced and M-AAT protein is augmented. Continue reading

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Gene therapy darling Oxford BioMedica pares losses – Telegraph.co.uk

Posted: August 20, 2017 at 3:42 pm

A darling British biotech that is working on the development of the worlds leading gene therapy for cancer has posted reduced losses and a leap in sales. Oxford BioMedica signed a deal worth up to $100m (77m) with Swiss drugs giant Novartis last month to supply cell material for its potential blockbuster treatment, known as CTL019, for a type of leukaemia. The living drug was recommended for approval by US regulators in June, with a final determination expected this autumn. Continue reading

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Sangamo Therapeutics: The Breakthrough In Gene Therapy – Seeking Alpha

Posted: at 3:42 pm

Investment Thesis With an undervalued stock, a critically-acclaimed drug leading in its space and a barrage of financial and expertise-based backing, Sangamo Therapeutics (NASDAQ:SGMO) is the ideal vehicle for exposure to a market with high growth potential and a hedge against the antiquated methods (CRISPR/Cas9) of treating hemophilia. The same method has also been found to work to a high degree of accuracy on other genetic disorders. Continue reading

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Scientists foresee Russian gene therapy for HIV cure may be registered in 5-10 years – TASS

Posted: at 3:42 pm

MOSCOW, August 17. /TASS/. A Russian gene therapy drug for individuals infected with HIV called Dinavir is undergoing pre-clinical trials, and the drug has already proved its efficiency on cells. Continue reading

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Achromatopsia: Gene Therapy Cures Eye Disease? – NBC 5 Dallas-Fort Worth

Posted: at 3:42 pm

Imagine only being able to see the things in front of you in soft focus, and just in black and white. For people with the genetic eye condition achromatopsia those are just some of the side effects. But, researchers are testing a new treatment designed to cure the condition by fixing the gene responsible. Continue reading

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Gene Therapy is Finally Here, But Who Will Foot the Bill – Wall Street Pit

Posted: August 14, 2017 at 4:45 pm

Human protein-coding genes number from 20,000 up to 25,000. If just one of these genes gets altered or a code gets missing, it can be fatal to an individual. In fact, approximately 30 per cent of infant mortality at birth in developed countries are caused by genetic disease. Continue reading

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A Novel Form of Gene Therapy Can Treat Diabetes With Genetically … – Big Think

Posted: at 4:45 pm

Researchers from the University of Chicago have successfully completed a proof-of-concept studywhere they managed to treat obesity in mice using a new type of gene therapy that utilized skin transplants. Continue reading

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TIMELINE-Gene therapy’s long road to market – Reuters – Reuters

Posted: at 4:45 pm

LONDON, Aug 8 (Reuters) – Gene therapy, which aims to patch faulty genes with working DNA, has been a long time in development. The following are major milestones: 1972 – Researchers first suggest gene therapy as a treatment for genetic diseases but oppose its use in humans “for the foreseeable future”, pending greater understanding of the technology. 1990 – A four-year-old girl with severe immunodeficiency became the first patient to undergo gene therapy in the United States. Continue reading

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Pfizer’s push into gene therapy adds more jobs in Sanford – News & Observer

Posted: at 4:45 pm

Pharmaceutical giant Pfizer is expanding its facilities in Sanford to accommodate the companys push into gene therapy. The state Department of Commerce announced earlier this week that Pfizer would invest $100 million in the site and create 40 jobs there within three years Continue reading

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