Gene Therapies
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AnGes To Withdraw, Refile HGF Gene Therapy In Japan
If finally fully approved in its first indication, the HGF gene therapy could become a first-in-class therapy for PAD - but only after years of challenges in clinical trials.
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Quick Listen: Scrip's Five Must-Know Things
In this week's podcast edition of Five Must-Know Things: global CEO remuneration; another late-stage loss for Merch KgaA; deals shift to smaller M&A, partnering; Bayer’s Parkinson’s bets bear fruit; and India looks to innovate CAR-T models.
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Intellia Becomes First To Show Potential For CRISPR Therapy Re-Dosing
The company presented data showing it is possible to safely and effectively re-dose its ATTR amyloidosis CRISPR/Cas9-based therapy.
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Bayer's Bets In Parkinson's Start To Bear Fruit
The German major is kicking off Phase II trials for a cell therapy and a gene therapy that could be gamechangers for the progressive neurodegenerative disorder that affects more than 10 million people worldwide.
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Conditional Global-First Nod For SanBio's Cell Therapy for TBI
SanBio’s lead cell therapy asset has been on a bumpy journey to its global-first approval and while a nod has now come in Japan in a high-need indication, a commercial launch is conditional on additional data to establish product equivalence and manufacturing consistency.
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As India Scales CAR-T Efforts, Can It Innovate Commercial Models?
As India seeks to build on early gains in CAR-T cell therapy, experts call for ecosystem reform and also the need to demonstrate value to payer models of large economies to potentially back international reimbursements.
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Sarepta Obtains Broader DMD Label For Elevidys
The gene therapy is now approved for all ambulatory boys with Duchenne muscular dystrophy ages 4 and older. The revised label also includes accelerated approval for non-ambulatory patients.
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Roche Adds Ascidian’s RNA-Writing To Its Neurology Push
A first big pharma partnership for Ascidian, headed up by ex-Biogen research head Michael Ehlers, as the Swiss firm expands its gene editing and neuroscience efforts.
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Two Better Than One As Syncona Creates Spur
The merger of Freeline and SwanBio is establishing an entity with secure financing to advance their respective gene therapies for Gaucher’s disease and adrenomyeloneuropathy.
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Sarepta Retains DMD Gene Therapy Lead As Pfizer Stumbles
Pfizer’s Phase III CIFFREO study in DMD failed to meet primary and key secondary endpoints, likely removes it as a potential competitor to Sarepta’s Elevidys.
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New Promise, Looming Competition For DMD In China
With several new drugs progressing for the estimated 70,000 patients in China with DMD, a recent setback for Nippon Shinyaku's contender and progress for Chinese gene therapy developers are adding both uncertainty and likely competition in an untapped market with pressing medical need.
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China Biotech Podcast: Going Global Trends, Rare Disease Drug Development
Guest speaker Professor Guangzuo Luo joins Brian Yang and Dexter Yang to discuss orphan drug development and going global trends for Chinese bioventures, along with the latest business moves from Shanghai Fosun and Jiangsu Hengrui.
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Intellia Builds Case For NTLA-2002’s Functional Cure In Angiodema
The hereditary angioedema candidate could become the first ever in vivo gene-editing therapy – but whether it will truly transform treatment remains to be seen.
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Quick Listen: Scrip's Five Must-Know Things
In this week's podcast edition of Five Must-Know Things: ASCO preview; Asahi Kasei’s US acquisition; Novartis aims for renal disease dominance; paying for gene therapies; and US BIOSECURE Act diluted.
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Could Combining Strategies Be A Way Forward To Pay For Gene Therapies?
A report by ICER and Tufts Medical Center’s NEWDIGS said a single strategy cannot adequately address payment challenges for all payers.
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Quick Listen: Scrip's Five Must-Know Things
In this week's podcast edition of Five Must-Know Things: the life of a blockbuster; blockbusters by indication; gene therapy struggles; sun shines on AstraZeneca and GSK; and Korean ventures look to Japan.
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