1. Scrip
  2. >>Advanced Therapies
  3. >>Gene Therapies

Gene Therapy Patient Pipeline Builds, But Slowly, In Q2

 
• By Jessica Merrill

Bluebird, Vertex and Sarepta are starting to see some commercial gene therapy traction and expect momentum to pick up in the second half, while hemophilia remains a challenging area. 

gene therapy
gene therapies slowly picked up momentum in Q2 • Source: Shutterstock

Some gene therapy manufacturers, like bluebird bio, Vertex Pharmaceuticals Incorporated and Sarepta Therapeutics, Inc., are beginning to see a ramp up in their commercial businesses and reported an expanding pipeline of patients beginning the process to start treatment in the second quarter. But the number of patients treated with new gene therapies remains limited and companies focused on hemophilia, including CSL Limited and BioMarin Pharmaceutical Inc., have continued to experience slow uptake.

Key Takeaways
  • With several gene therapy launches under way, manufacturers are gaining valuable commercial experience.

Of the handful of companies that have launched new gene therapies during the last two years, Sarepta stands out for...

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
Start Free Trial

Already a subscriber?

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

Sanofi Plays It Safer With Blueprint Buy After Bumpy 2025

 
• By Kevin Grogan

But the French major is still keen on early-stage M&A.

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.