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Gene Therapies

Set Alert for Gene Therapies

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.

Japan Gene Therapy

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.

Clinical Trials Commercial

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.

Gene Therapy Cardiovascular

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.

Regenerative Medicine Clinical Trials

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. 

Japan Regulation

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.

Commercial India

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.

Approvals Gene Therapy

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.

Deals StartUps and SMEs

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.

M & A Gene Therapy

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.

Gene Therapy Clinical Trials

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.

China Clinical Trials

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.

China Business Strategies

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.

Clinical Trials Companies

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.

Clinical Trials M & A

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.

Gene Therapy Market Access

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.

Commercial Gene Therapy
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