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.

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...

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Scrip for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required

More from Gene Therapies

More Pressure on Elevidys As The EU’s CHMP Says ‘No’

 

Just days after Roche stopped shipments of the DMD gene therapy following safety concerns raised by two patient deaths, its EU approval application has hit a block.

Rocket Comes Back To Earth With Restructuring Plan

 

The biotech will lay off nearly one-third of its workforce and focus on its cardiovascular gene therapy programs.

The Duchenne Gene Therapies Lining Up To Take Elevidys’ Place

 

Regenxbio and Solid Biosciences believe they have a next-generation approach to AAV-based gene therapies – but must distance themselves from Sarepta’s safety and efficacy problems.

Sarepta Voluntarily Stops Shipments Of Elevidys After Previously Refusing FDA Request

 
• By 

The company had planned to keep its DMD gene therapy available for ambulatory patients, but now says pausing shipments may enable a better working relationship with the US FDA while safety labeling is updated.

More from Advanced Therapies

Sarepta Voluntarily Stops Shipments Of Elevidys After Previously Refusing FDA Request

 
• By 

The company had planned to keep its DMD gene therapy available for ambulatory patients, but now says pausing shipments may enable a better working relationship with the US FDA while safety labeling is updated.

FDA Asked, Sarepta Refused To Stop Elevidys Shipments After Third Death With Gene Therapy Vector

 
• By 

Sarepta knew about the death in a Phase I trial of SRP-9004 for LGMD when it recently revealed a restructuring plan, but did not disclose the event. The now-discontinued gene therapy candidate uses the same AAV vector as Elevidys, which has been linked to two DMD patient deaths.

Hopes Rise For Sickle Cell Gene Therapy Access As 33 States Join CMS Program

 

CMS said that 33 US states plus the District of Columbia and Puerto Rico had joined a program whereby the agency negotiates outcomes-based contracts on their behalf.