Gene Therapy’s Next Big Challenge: Manufacturing

 
• By Amanda Micklus
  
Generate a summary of this article
Listen to this article
Beta

As the pipeline and market for novel cell and gene therapies expands, it is time for drug developers to address other critical aspects in the logistical chain – and for these complex therapies a lot of those issues relate to manufacturing. Looking at specific cases, In Vivo has underlined the main manufacturing challenges facing cell and gene therapy developers, and highlighted methods and approaches being used to generate solutions. 

DNA detail background with selective focus
• Source: Shutterstock

Commercialization of modern-day gene therapies is now a reality. Next-generation modalities such as chimeric antigen receptor T-cell (CAR-T) therapies are fully in launch mode in the US, where Novartis AG and Gilead Sciences Inc. are banking on the success of their one-time hematological cancer treatments Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel), respectively. Final approval of those CAR-T therapies has also occurred in the EU, with funding arrangements in place in the UK. In addition, the first in vivo gene therapy for an inherited disease is now available in the US, by way of Spark Therapeutics Inc.'s Luxturna (voretigene neparvovec - rzyl), and the EMA is currently evaluating what could be the next approval in the market, bluebird bio’s LentiGlobin (lentiviral beta-globin gene transfer) for transfusion-dependent beta thalassemia.

Presently, there are 25 unique gene therapies that have reached Phase III or have been filed for approval. Approximately 33...

Pink Sheet Podcast

Learn what's happening at the US FDA. On the go.

Derrick Gingery and the team bring you a weekly "Drug Fix".

SpotifyApple PodcastsWebsite

More from Cell & Gene Therapies

test carousel-PINKSHEET

 
• By Saba Afreen

test carousel-test

Diversity Action Plans Should Be Brief And Waiver Requests Filed Early, US FDA Says - Clone

 
• By Sue Sutter

In what could amount to a de facto enforcement mechanism, the new draft guidance also “strongly encourages” sponsors to share details about their diversity action plan and enrollment goals with the public.

Testing all options - Akash

 
• By Akash

This is an test article.

More from Advanced Technologies

CAR-T Therapies: Rethinking Of Postmarketing Requirements And REMS Urged

 
• By Sue Sutter

Long-term follow-up requirements have taken a conservative approach but could be ripe for re-examination and global harmonization given the years of experience with the products, Kite Pharma executive director says; former FDA gene/cell therapy office head Wilson Bryan calls for elimination of the classwide REMS.

test

 
• By Sabbah and Saba Afreen

test

Gene Therapies: CoGenT Pilot Could Help With Regulatory Convergence Earlier In Development TEST

 
• By Sue Sutter

Program for collaborative reviews of gene therapy applications is getting under way with the European Medicines Agency. After launch, the pilot could be expanded to earlier discussions around CMC and pharm/tox data requirements, FDA’s Nicole Verdun said.