Gene Therapy Q1 Roundup: The Struggle Is Real

 
• By Jessica Merrill
  
Generate a summary of this article
Listen to this article
Beta

Vertex and bluebird compete for sickle cell patients, BioMarin talks about divesting Roctavian, and Sarepta warned that sales of Elevidys will plateau with a narrow label.  

turtle
Gene therapies are making slow progress commercially • Source: Shutterstock

The development of a commercial market for gene therapies is unfolding in real time, and progress remains slow. Several developers of new gene therapies – bluebird bio, Vertex Pharmaceuticals Incorporated, Sarepta Therapeutics, Inc. and BioMarin Pharmaceutical Inc. – updated investors on launch progress in connection with first quarter sales and earnings results.

Complex administration and reimbursement of the expensive therapeutics remain barriers to broad uptake, but a growing pipeline of patients are...

Scrip Podcast

Keep up with the top pharma business news of the week. On the go.

Scrip brings you a weekly bulletin of top picks.

SpotifyApple PodcastsWebsite

More from Gene Therapies

Scrip Asks…What Does 2024 Hold For Biopharma? Part 6: Therapeutic Area Advances KOSH SANDBOX

 
• By Kosh Naran

More than 100 biopharma executives and experts told Scrip their predictions for therapeutic area advances in the coming year. The recent commercial success of GLP-1s in diabetes and obesity and their potential in further disease areas fuelled excitement around the metabolic space. Expectations were also positive in neurology following the launch of Leqembi for Alzheimer’s disease in 2023, while the multiple opportunities to improve cancer treatment kept oncology top of the pile overall.

Quick Listen: Scrip's Five Must-Know Things

 
• By Ian Haydock

In this week's podcast edition of Five Must-Know Things: Merck & Co. steps into CD19 bispecific space; gene therapy patients rise, but slowly; Madrigal’s Rezdiffra plans; Korean biopharma financing recovering?; and approvals to watch out for in Q3.

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. 

More from Advanced Therapies

spo

Akash test for clone

Test article to check the clone issue

Live test

 
• By Mary Jo Laffler

Progress Or Hype? A Decade Of Cell Therapy In Japan

 
• By Lisa Takagi

After 10 years of promised investment following its Nobel Prize for iPS cell research, Japan is cautiously narrowing regulations around the conditional approval of cell therapies and cutting some reimbursement prices. Commercial success remains mixed and some products have been withdrawn from the market.