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Gene editing: beyond the hype

pharmaphorum

Sangamo CEO Sandy Macrae told us how his company is being cautious about the hype and finding ways to be financially viable in an emerging space. Maybe in 50 years’ time we’ll be using gene editing to lower cholesterol, but it won’t replace statins in anyone but those with life threatening mutations for a long time”. Zinc fingers.

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How epilepsy researchers are moving the needle past anti-seizure treatments

Pharmaceutical Technology

Several biotech companies and researchers are now exploring medical devices and gene therapies to address not just common forms of epilepsy, but also rare conditions such as Dravet Syndrome. Meanwhile, others are researching the link between gene variation and different responses to treatments.

Research 264
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Untangling the Complexities of Cell and Gene Therapy Clinical Trials: A Supply Chain Perspective 

Pharmaceutical Technology

By Luisa Sterkel & Joana Loureiro , Tenthpin Consultants The promise and potential of cell and gene therapies (CGT) has emerged in the recent past and currently over 1.500 CGT are registered for clinical trials holding great hope for the treatment of challenging and uncurable diseases.

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Takeda grows in gene therapies again with $2bn Code Bio deal

pharmaphorum

Takeda has forged another alliance as it continues a push into gene therapy, agreeing a deal worth up to $2 billion with Code Biotherapeutics for opt-in rights to four candidates for rare diseases. The post Takeda grows in gene therapies again with $2bn Code Bio deal appeared first on. Last October it signed a $3.6 billion-plus.

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Can genetic data be a magic bullet for drug R&D?

pharmaphorum

Drug development has long been an issue for the pharma industry, due to the expense and the high failure rate of potential treatments. Ben Hargreaves finds that the vast amount of genetic data that exists today could help provide a faster, more targeted way of developing new drug candidates.

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Italfarmaco’s Duvyzat Wins FDA Approval as First Nonsteroidal Treatment for All Genetic Variants of DMD

XTalks

Duvyzat, a histone deacetylase (HDAC) inhibitor that works to reduce inflammation and muscle loss, is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. Progressive muscle weakness in the disease is caused by genetic mutations in the dystrophin gene that lead to a lack of functional dystrophin protein.

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‘Knowledge is power’ for rare diseases and NAbs

pharmaphorum

Cure Rare Disease’s Rich Horgan discusses the importance of preclinical NAbs screening and helping rare disease patients better understand their eligibility for gene therapies. This is particularly pertinent in rare diseases, as a number of gene therapy products use viral delivery methods to deliver the transgene to target organs.