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Gene Therapies

Set Alert for Gene Therapies

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

Gene Therapy Q1 Roundup: The Struggle Is Real

Vertex and bluebird compete for sickle cell patients, BioMarin talks about divesting Roctavian, and Sarepta warned that sales of Elevidys will plateau with a narrow label.  

Gene Therapy Launches

Regeneron Gene Therapy Reaches New Heights In Restoring Hearing

The US biotech’s DB-OTO has made headlines worldwide after a baby with profound genetic deafness experienced improved hearing to normal levels within 24 weeks after a single intracochlear injection.

Ear Gene Therapy

Metagenomi Looks For Upside To Moderna’s Exit

Moderna has cut short its alliance with Metagenomi, giving it more work to do in persuading skeptical investors about its gene-editing platform.

Deals Companies

Pfizer Matches Beqvez Price To Hemgenix In Hemophilia B, Plans Warranty Program

The drug maker said the program would allow for continued reimbursement when patients change insurance plans. A Sangamo-partnered gene therapy for hemophilia A is also in the works.

Approvals Gene Therapy

How Ochre Bio Bagged Boehringer For Its RNA-Based Regenerative MASH Therapies

UK-based Ochre Bio has signed its first major deal with Boehringer Ingelheim. Scrip talked to its co-founder and CEO, Jack O’Meara, about its human tissue-based drug discovery platform, its resulting RNA platform for liver disease and how the fledgling drug company's early work mirrored that seen in diagnostics.

Companies Deals

Precision Keeps Faith In Duchenne Gene-Editing After Lilly’s Exit

The company has pledged to continue developing its Duchenne in vivo gene editing therapy without Lilly, but will remain focused on its lead program in hepatitis B.

Deals Companies

Keeping Up The Pressure In Pulmonary Arterial Hypertension

Merck & Co leads a market expected to exceed $10bn by 2028, but others are aiming to muscle in.

Clinical Trials Commercial

Verve Not Ready To Pull Plug On Anti-PCSK9 Gene Editing Candidate

The company told Scrip that the sixth patient with elevated liver enzymes and low platelets recovered after hospital observation and oral steroids, and it may still continue VERVE-101’s development.

Gene Therapy Clinical Trials

Orchard Will Launch Lenmeldy At Just Five Centers

Orchard, which Kyowa Kirin bought in October, gave Lenmeldy a $4.25m list price, exceeding the upper bound of about $3.9m set by ICER.

Approvals Pricing Strategies
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