New CRISPR Therapeutics/Vertex Data Dangle The Hope Of Cure For SCD and TDT

With more data reported at ASH, Vertex and CRISPR Therapeutics added heft to the notion that functional cures are on the horizon for the inherited blood disorders sickle cell disease and beta-thalassemia. Attention is now turning to Bluebird’s marketing strategy after a filing delay.    

DNA detail background with selective focus
• Source: Shutterstock

bluebird bio will need to capitalize on its leader position if it is to maintain first-mover advantage over CRISPR Therapeutics AG/Vertex Pharmaceuticals Incorporated in a potential blockbuster market for genetic treatments for sickle cell disease (SCD) and beta-thalassemia.

The companies have each presented data from more patients for their respective products, LentiGlobin for Sickle Cell Disease, betibeglogene autotemcel...

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 Clinical Trials

Biogen Lays Out Cadence For Felzartamab Data In Kidney Diseases

 

The first Phase III data for the CD38-targeting antibody is expected in 2027 in antibody-mediated rejection in kidney transplant patients, followed by other indications.

In Brief: Gilead HIV Trials On Hold Over Safety Signal

 

The FDA has paused Gilead’s HIV trials of GS-1720 and GS-4182 over safety concerns. The company’s upcoming PrEP PDUFA looks to be unaffected.

Spain’s SpliceBio Secures Sanofi and Roche Support In $135m Financing

 
• By 

Series B cash will be used to advance its Stargardt disease gene therapy.

In Brief: PolyPid’s D-PLEX100 Aces Phase III Trial, US Partner, Submission Eyed

 

PolyPid's D-PLEX100 meets Phase III endpoints this time around, significantly reducing surgical point infections in abdominal colorectal surgery, and is set for a US approval submission early next year.

More from R&D

In Brief: PolyPid’s D-PLEX100 Aces Phase III Trial, US Partner, Submission Eyed

 

PolyPid's D-PLEX100 meets Phase III endpoints this time around, significantly reducing surgical point infections in abdominal colorectal surgery, and is set for a US approval submission early next year.

Insmed’s PAH Data Indicate Potential To Surpass Established Therapies

 
• By 

Insmed’s inhaled treprostinil exceeded goals for reducing pulmonary vascular resistance and improving six-minute walk test distance in Phase IIb data.

NewAmsterdam’s CETP Inhibitor Improves Alzheimer’s Biomarkers

 
• By 

Data from the BROADWAY trial testing LDL-C reduction in adults with ASCVD showed that obicetrapib improved Alzheimer’s disease biomarkers, including tau and amyloid. Analysts think the data could differentiate obicetrapib against competitors in the cholesterol market.