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The Cell And Gene Therapy Sector In 2023: A Wave Is Coming – Are We Ready?

 
• By Timothy Hunt

From a scientific perspective, the outlook for patient with rare disease has never been brighter. This year, 13 new cell or gene therapies could be approved in the US, Europe or both by the end of 2023. However, the challenges that stop patients accessing new therapies remain. Could this year be the turning point when our health care systems start to catch up with our science?  

2023
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The status quo for patients with serious diseases is often death, disability, or poor quality of life. What if I told you that status quo is changing? Ten years ago, Emily Whitehead had run out of treatment options for her leukemia. As a last resort, Emily’s parents agreed to an experimental cell therapy never tested in children. Now, 10 years later, Emily is considered cured by this CAR-T therapy.

About The Author

Timothy Hunt is CEO of the Alliance for Regenerative Medicine, an international advocacy organization dedicated to realizing the promise of regenerative medicines and advanced therapies.

There are now six FDA-approved CAR-T cell therapies available to patients in both the US and Europe to treat various blood cancers. They are but one part of a growing...

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US Health System Redesign Critical, NAM’s Medical Experts Warn Trump Government

 
• By Ashley Yeo

The whirlwind back-and-forth on US tariffs and Robert F. Kennedy jr.’s plans to deregulate health care have become all-preoccupying, but the National Academy of Medicine was first to set out President Trump’s health administration priorities.

Podcast: “Powerful Yet Unexplored”: Commit Biologics Tackles The Complement System

 
• By Anabel Costa-Ferreira

Mikkel Wandahl Pederson, CEO and chief scientific officer at Commit Biologics, discussed the company’s mission to harness the powerful complement system for the treatment of serious diseases.

ML-Assisted Genetic Risk Score Predicts GLP-1 Adverse Events

 
• By David Wild

Phenomix Science presented new data at Digestive Disease Week 2025 showing its machine learning-assisted genetic risk score can predict nausea and side effects from GLP-1 receptor agonists, aiding personalized obesity treatment.