OcyonBio

At OcyonBio, we are thrilled to see that patients with rare diseases have a brighter outlook than ever before. The advancements in cell therapy have opened up new possibilities for treating diseases that were once thought to be incurable. We believe that these breakthroughs in science have the potential to transform the lives of millions of patients and their families. This year, 13 new cell or gene therapies could be approved in the US, Europe or both by the end of 2023.1 However, the challenges that stop patients from accessing new therapies remain. Could this year be the turning point when our health care systems start to catch up with our science? 

The status quo for the future of patients with serious diseases is often poor quality of life, disability, or death. But 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 treatment never tested before in children. Now, 10 years later, Emily is considered cured by this CAR-T therapy.1 

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 set of promising cell and gene therapies to address rare and prevalent conditions. There are over 2,000 clinical trials happening today, with 200 in Phase III – a wave of hope for patients waiting on durable, possibly curative treatments.1 

But investment, policy, and regulatory challenges remain for the sector, and many patients will not benefit unless we modernize our approach to health care. 

The Pipeline of Transformative Therapies is Accelerating Rapidly 

2022 was a record year for the approval of never-before-authorized gene therapies, with three new therapies approved to treat rare diseases and another approved to treat bladder cancer.1 But this is just the initial swell of the coming wave. 

As of this writing, as many as 13 brand new cell or gene therapies could be approved for use in the US, Europe, or both by the end of 20231. We are in reach of the FDA’s often-cited 2019 prediction that it would approve 10-20 new cell and gene therapies a year by 2025. 

A gene therapy for Duchenne Muscular Dystrophy – a disease whose R&D path has been beset with disappointment – could receive accelerated approval by the FDA in 2023. 

Two therapies to treat sickle cell disease – one of the most prevalent rare diseases – are also expected to receive approval in 2023. When these new therapies become receive approval, there will be intense focus on whether the US health care system, and particularly state Medicaid programs, can provide access to these therapies. 

OcyonBio at ASGCT 2023 

OcyonBio is committed to being at the forefront of these new trends and helping accelerate your pipeline. We will also be attending the American Society of Gene & Cell Therapy (ASGCT) 2023 to stay up-to-date on the latest developments in cell therapy. Our team is eager to network with industry leaders, share insights, and explore new opportunities to bring life-changing treatments to patients in need.   

We are dedicated to accelerating pipeline success and delivering treatments to patients faster. That’s why we are excited to invite you to our upcoming presentation at the startup session on May 17 at 12:00 pm. We’ll be sharing our expertise on the latest strategies and technologies to accelerate pipeline success and improve patient access to life-changing therapies. Join us to learn more about the trends and developments in cell therapy and how we can work together to bring new treatments to patients more efficiently. 

Access to our presentation at the ASGCT 2023

The future of cell therapy looks bright with many new treatments on the horizon. However, there are still challenges that need to be addressed in order for these treatments to become widely available. It will be interesting to see how these trends develop over the course of 2023. 

References: 

[1]Hunt, Timothy . “The Cell And Gene Therapy Sector In 2023: A Wave Is Coming – Are We Ready?” In Vivo Pharma Intelligence, 9 Jan. 2023, invivo.pharmaintelligence.informa.com/IV146781/The-Cell-And-Gene-Therapy-Sector-In-2023-A-Wave-Is-Coming–Are-We-Ready. Accessed 5 May 2023.

By: Ashley Cortés, Marketing Specialist at OcyonBio