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CRISPR Therapeutics And Vertex’s CRISPR Breakthrough: How And Why They Got There First

 
• By Andrew McConaghie

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.

Among the many innovative new medicines launched in 2023, one approval stands out in terms of innovation – Vertex and CRISPR Therapeutics’ sickle cell and beta thalassemia gene therapy Casgevy, the first CRISPR-edited gene therapy to reach the market.

The story of how Casgevy (exagamglogene autotemcel, exa-cel) was developed is a remarkable one, not least because of the exceptional speed with which it has reached the market, taking barely nine

Key Takeaways

  • Casgevy became the first ever CRISPR-based gene therapy to gain approval in November

  • The sickle cell disease therapy has reached the market less than four years after the first human trial was launched

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Bluebird, Private Equity Firms Look To Sweeten Buyout Deal

 
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Shareholders are being offered a flat fee of $5 per share as an alternative to $3 plus a contingent value right, which could be a more attractive option if they want cash up front.

Which Firms Are Most Exposed To Prasad’s Possible Accelerated Approvals Clampdown?

 
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Verve Moves Toward Lilly Opt-In For PCSK9 Program With Phase Ib Results

 
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The company posted positive initial results from the Heart-2 trial in HeFH and CAD, with strong LDL cholesterol lowering and a clean safety profile.

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Abeona Sets $3.1m Price For Rare Skin Disorder Gene Therapy

 
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Gilead Pipeline Progress Offsets Oncology Sales Slowdown

 
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Sales of the company’s approved cancer therapies were weaker or down, but Trodelvy is likely to see a label expansion and cell therapies are making progress.

In Brief: BriaCell’s Breast Cancer Cell Therapy Boosts Survival In Phase II

 
• By Sushmita Panda

BriaCell announced its cell therapy Bria-IMT plus checkpoint inhibitors bested Gilead’s Trodelvy in a Phase II trial in HR+ breast cancer; a Phase III trial is enrolling.