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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...

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More from Gene Therapies

New Regenxbio DMD Data Support Gene Therapy Accelerated Approval

 
• By Joseph Haas

Regenxbio reported functional and biomarker data from an ongoing study of RGX-202, which it hopes to file for approval in Duchenne muscular dystrophy in 2026.

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.

More from Advanced Therapies

BREAKING NEWS: Sanofi Bets $9.5bn To Bag Blueprint

 
• By Kevin Grogan

Buy boosts the French major's immunology pipeline.

Senti’s Computer-Logic Inspired Cell Therapy Shows Early Promise

 
• By Andrew McConaghie

The biotech believes its logic-gated cell therapies can get round the need for ‘clean targets’ which limit CAR-Ts and ADCs.

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.