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The promises of gene therapy are taking a long time to be fulfilled, in part due to challenges in developing gene vectors capable of safe and efficient delivery of therapeutic genes (transgenes) into patients’ cells. Nonetheless, these challenges are being addressed in innovative ways. A variety of first-generation vectors, both viral and non-viral, have been employed in clinical trials with varying degrees of success, and the experience gained is guiding the design of next-generation vectors.
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Learn more in the "Trends in Gene Therapy report", available to buy here
Pink Sheet: global policy and regulatory coverage
02 Aug 2017
Long-term or open-ended treatment with PD-1/PD-L1 inhibitors is a burden for patients and the healthcare system and does not fit the biological paradigm of cancer immunotherapies, experts say; however, challenges to studying shorter durations include fears of undertreatment and patient objections to stopping a therapy that appears to be working.
Topics Clinical trials
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Pharma Intelligence offers a wealth of pharmaceutical industry news and strategic insight into the healthcare & biotechnology markets from around the world. Working in an interconnected global network, our 65 journalists and over 300 in-house analysts supply comprehensive analysis and reports.
Scrip: industry news and insights
The Swiss major's pharma chief tells Scrip that its gene therapy for SMA offers much more than Biogen's Spinraza for both patients and payers, noting that the company is ready with alternative payment models for the pricey treatment as soon as approvals for Zolgensma are granted.
Topics Business strategies Drug approval Drug development landscape Drug review
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