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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...

Key Takeaways

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

  • The sickle cell disease...

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Bluebird, Private Equity Firms Look To Sweeten Buyout Deal

 
• By Alaric DeArment

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?

 
• By Andrew McConaghie

Vaccine and accelerated approvals are expected to face greater scrutiny under new CBER leader Vinay Prasad, but he has promised “no light switch change” to policy, giving some reassurance to companies whose share prices have been battered.

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

 
• By Joseph Haas

Abeona plans to offer an outcomes-based payment model for Zevaskyn, which likely will see complementary use with Krystal’s Vyjuvek in recessive dystrophic epidermolysis bullosa patients.

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

 
• By Alaric DeArment

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.

More from Advanced Therapies

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

 
• By Joseph Haas

Abeona plans to offer an outcomes-based payment model for Zevaskyn, which likely will see complementary use with Krystal’s Vyjuvek in recessive dystrophic epidermolysis bullosa patients.

Gilead Pipeline Progress Offsets Oncology Sales Slowdown

 
• By Alaric DeArment

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.