Gene Therapies
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Stock Watch: Advanced Therapies Deliver Fourth-Quarter Flop
As drug pricing continues its ascent among hot button issues, the multi-million-dollar price tags for one-and-done advanced therapies, their justification and reimbursement all remain unaligned.
FDA Delay Will Not Push Rocket Pharma Off Its Trajectory
The gene therapy company could have two products approved by the end of 2024 – with ambitions for long-term expansion beyond rare disease.
Sales Of BioMarin’s Hemophilia Gene Therapy Move At Glacial Pace
Analysts had forecast Q4 Roctavian sales of $5m-$6m, but they came in shy of $3m, and uptake has not yet come to reflect apparent demand.
Sangamo Shoots Up As Regulatory Path Clears For Fabry Gene Therapy
The US group has had a tough year but the signs look more promising for isaralgagene civaparvovec as the FDA agrees on the design for a registrational trial for the gene therapy. Now Sangamo needs to secure a partner.
Adverum Climbs Out Of Abyss With Promising Early Gene Therapy Data In Wet AMD
The Phase II LUNA study of ixo-vec showed a reduction in anti-VEGF use and stabilization of visual acuity and anatomic parameters that Adverum – and some analysts – called best in class.
Scribe Therapeutics Picks Cholesterol-Lowering As First In Vivo Gene Target
The company claims its gene editing tech is the most potent yet – and is joining others in making established cardiovascular disease gene PCSK9 its first therapeutic target
Scrip Asks…What Does 2024 Hold For Biopharma? Part 4: Transformative Technology
More than 75 industry executives and experts gazed into the crystal ball and shared with Scrip their predictions around transformative technologies. Perhaps unsurprisingly, AI was the dominant theme – but gene editing, cell therapies and new targeting modalities were also among the highlights.
Solid Data For Sarepta’s Next-Gen PPMO Treatment But Future Uncertain
A Phase II study testing a next-generation exon skipping medicine vesleteplirsen in Duchenne muscular dystrophy amendable to exon 51 outpaced Sarepta’s Exondys 51.
Quick Listen: Scrip's Five Must-Know Things
In this week's podcast edition of Five Must-Know Things: Bayer’s pipeline refresh; J&J’s selective pharma M&A approach; silver lining for Gilead’s Trodelvy miss?; paying for obesity drugs; and the story behind Vertex and CRISPR’s gene therapy breakthrough.
Scrip Asks…What Does 2024 Hold For Biopharma? Part 3: Manufacturing And Supply
Looking ahead on manufacturing and supply-related issues in 2024, respondents told Scrip to expect companies to bring their supply chains closer to home, improvements for delivery of cell and gene therapies, a focus by contract manufacturers and developers on sustainability and continued emphasis on access to critical drugs.
Genentech Places Bet On Polymer Nanoparticle-Based Delivery With GenEdit Deal
Deal Snapshot: The Roche subsidiary will use GenEdit’s hydrophilic nanoparticles to develop genetic medicines for autoimmune diseases meant to overcome the limitations of existing delivery methods.
Bluebird’s Fast Lyfgenia Launch Was A Year And A Half In The Making
The company said when Zynteglo was approved for beta-thalassemia that its launch would lay the groundwork for its sickle cell gene therapy Lyfgenia, and the strategy is paying off so far with a fast ramp-up.
CRISPR Therapeutics And Vertex’s CRISPR Breakthrough: How And Why They Got There First
Leaders from the companies explain how they navigated to success with the world’s first CRISPR-based medicine – and avoided the pitfalls that claimed other programs.
Regenxbio Aims For Big Chunk Of Retinal Disease Market With AbbVie-Partnered Gene Therapy
The company presented promising Phase II data, but it didn’t reveal its plans for a pivotal trial of ABBV-RGX-314, which would compete in a growing – but increasingly crowded – market.
What’s Next For ICER: An Interview With New President Sarah Emond
The Institute for Clinical and Economic Review’s new president talked to Scrip in an interview about cell and gene therapy, obesity drugs and the new Medicare drug price negotiation program.
The ‘Haves And Have-Nots’ Are Becoming Clearer In Cell And Gene Therapy
2023 saw a number of gene therapy companies go to the wall, but those that can combine clinical significance and commercial success can build sustainable businesses.
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