Sangamo and Pfizer Announce Updated Phase 1/2 Results Showing Sustained Increased Factor VIII Activity Through 44 Weeks Following SB-525 Gene Therapy Treatment

The first two patients treated with the 3e13 vg/kg dose achieved stable Factor VIII (FVIII) levels demonstrating durability in the normal range through 44 and 37 weeks, respectively– All five patients in the 3e13 vg/kg dose cohort achieved normal range FVIII levels within 5-7 weeks following treatment, with no bleeding events with up to 44 weeks

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Buyer beware of this unproven and untested $1 million gene therapy for aging, that might even be harmful to your health

Offshore tests by a startup seek to lengthen people’s telomeres—and their lives. Source MIT Technology Review It’s said that nothing is certain except death and taxes. But doubt has been cast over the former since the 1970s, when scientists picked at the seams of one of the fundamental mysteries of biology: the molecular reasons we

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Hopkins team invents non-viral system for getting gene therapy into cells

Polymer-based nanocontainers could improve the delivery of gene therapies, a Johns Hopkins team reported. One of the most popular methods for inserting therapeutic genes into cells to treat disease is to transport them using a virus that has been stripped of its infectious properties. But those noninfectious viruses can still sometimes touch off dangerous immune

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FDA launches app for healthcare professionals

To report novel uses of existing medicines for patients with difficult-to-treat infectious diseases The US Food and Drug Administration announced the global launch of CURE ID, an Internet-based repository that will allow the clinical community to report their experiences treating difficult-to-treat infectious diseases with novel uses of existing FDA-approved drugs through a website, a smartphone

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FDA sends warning to companies for offering unapproved umbilical cord blood products that may put patients at risk

Liveyon Labs Inc. and Liveyon LLC warned that their stem cell products lack required FDA approval and represent a potential risk to the public health The U.S. Food and Drug Administration has warned Liveyon Labs Inc. (Liveyon Labs) and Liveyon LLC, of Yorba Linda, California, and their presidents and chief executive officers, Roya Panah and John

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Stanford researchers program cancer-fighting cells to resist exhaustion, attack solid tumors in mice

CAR-T cells are remarkably effective against blood cancers, but their effect can be transient as the cells become exhausted. Stanford researchers found a way to keep the cells effective in mice with human tumors. A new approach to programming cancer-fighting immune cells called CAR-T cells can prolong their activity and increase their effectiveness against human

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Mesenchymal stem cell perspective: cell biology to clinical progress. Review

The terms MSC and MSCs have become the preferred acronym to describe a cell and a cell population of multipotential stem/ progenitor cells commonly referred to as mesenchymal stem cells, multipotential stromal cells, mesenchymal stromal cells, and mesenchymal progenitor cells. The MSCs can differentiate to important lineages under defined conditions in vitro and in limited

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