Recap from the November 2024 Bridging the Gap webinar
Pictured in photo: Sickle cell warrior Jimi Olaghere climbed Mt. Kilimanjaro after receiving advanced gene therapy.
The incredible potential of gene therapies like CAR-T and CRISPR to cure genetic diseases and cancers marks a new era in medicine, yet most patients still lack access to these life-saving treatments. For many, the promise of gene therapy remains out of reach due to barriers like high costs, limited treatment availability, and healthcare inequities.
In the November 2024 Bridging the Gap webinar, guest speakers Jimi Olaghere, a sickle cell survivor and advocate, and Dr. Fyodor Urnov, Professor of Molecular Therapeutics at UC Berkeley, tackled this pressing issue. Their conversation explored the revolutionary impact of gene-editing therapies and highlighted the obstacles—financial, logistical, and systemic—that prevent equitable access.
Together, they brought insights on what must change to make these therapies accessible to patients worldwide. Their stories and perspectives offer a powerful reminder: the potential of gene therapies means little if only a select few benefit.
Overcoming the Challenges of Sickle Cell Disease: A Patient’s Journey
Jimi Olaghere’s story is nothing short of life-changing. Diagnosed with sickle cell disease at birth, Jimi’s life was defined by frequent medical crises and ongoing struggles with physical and mental health. As he shared in the webinar, “I felt like I was in a prison that wasn’t of my own making… every year it was some organ failing.”
Despite the challenges, a profound change began when he saw an article about Victoria Gray, the first patient in the U.S. to receive CRISPR-based gene therapy for sickle cell disease. Inspired by her journey, Jimi reached out to a research team and soon became part of a clinical trial for the treatment himself.
Reflecting on his life since receiving the therapy, Jimi described it as “transformative,” stating that he has not experienced a single crisis since his treatment and now lives a life free from the daily threats posed by sickle cell disease. However, with this new beginning came a realization of the broader issues: the treatment, valued at $2.3 million, remains far beyond reach for most people worldwide.
This staggering cost is a barrier that he is determined to help overcome. Recently, Jimi climbed Mount Kilimanjaro to raise awareness and funds for newborn screening programs and the distribution of hydroxyurea, a medicine that could significantly improve the lives of sickle cell patients in low-income parts of the world. “It was the perfect opportunity,” he said, “to take actionable steps to make these therapies accessible, while ensuring that patients are alive to one day benefit from them.”
The Road Ahead: Making Advanced Therapies Accessible
Dr. Urnov, a key contributor to the development of CRISPR technology and a fervent advocate for affordable gene therapies, joined Jimi in advocating for a major shift in how these treatments are developed and distributed. Dr. Urnov emphasized that the success of CRISPR therapies isn’t just about medical advances; it’s about addressing disparities in access. “When will CRISPR be like KYMRIA?” he asked, pointing to the high costs and limited reach of current gene therapies.
Dr. Urnov also highlighted the need for collaboration across sectors, including partnerships with governments and private industry, to create sustainable pathways for making these therapies more affordable and available worldwide. He noted that while the technology to produce gene therapies affordably is within reach, there is no current system or global council to ensure that these treatments are accessible to the people who need them the most.
Reflecting on his work with the Innovative Genomics Institute (IGI), Dr. Urnov outlined the Institute’s mission to “build a platform for a CRISPR cure” that would not only address rare diseases but also be scalable and affordable enough for large patient populations.
Key Takeaways from the Webinar
- Transformative Impact of Gene Therapy: Jimi Olaghere’s experience shows the life-altering potential of CRISPR-based therapies. With no medical crises since his treatment, he is living proof of what gene-editing technology can achieve.
- Financial and Geographical Access Barriers: Despite the success stories, the high cost of gene therapy presents a major obstacle, particularly for patients in low- and middle-income countries. Jimi’s advocacy and Dr. Urnov’s mission reflect the urgent need to make these therapies widely accessible.
- Collaboration for Scalable Solutions: Dr. Urnov emphasized that partnerships between research institutes, governments, and private companies are essential to overcome logistical and financial barriers to advanced therapies.
- Patient and Family Advocacy: Stories like those of Jimi and Victoria Gray highlight the critical role of patient advocates in promoting awareness and access, with the Emily Whitehead Foundation also playing a vital part in supporting these efforts.
- The Power of Perseverance: Both speakers agreed that the journey to equitable healthcare for all patients with genetic diseases requires resilience and a relentless commitment to innovation, accessibility, and awareness.
Moving Forward: A Call for Systemic Change
The November 2024 Bridging the Gap webinar not only celebrated one year of groundbreaking discussions but also reinforced the need for change. Jimi and Dr. Urnov’s stories illustrate the potential of gene therapy and the challenges that still lie ahead. Whether through global partnerships, new business models, or policy changes, it is clear that making gene-editing therapies accessible to all requires a multifaceted approach.
As Dr. Urnov stated, “We need to deliver therapies to those that are eligible… we cannot let innovation stagnate.”
For patients like Jimi, who have seen the “before and after” of sickle cell disease thanks to CRISPR, there is no going back. Yet for millions worldwide, the promise of these therapies remains out of reach. The Bridging the Gap community remains committed to sharing insights, driving awareness, and fostering collaborations that will help overcome the obstacles to accessible and affordable gene therapy solutions.
A Pathway to Hope
This edition of the Bridging the Gap webinar exemplified the power of patient voices in influencing medical advancements and accessibility. As the life sciences community continues its efforts, the words and actions of advocates like Jimi and pioneering scientists like Dr. Urnov serve as a rallying cry for accessible care.
Their message is clear: with the right commitment, we can make life-changing therapies a reality for all, bridging the gap between innovation and the people who stand to benefit from it the most.
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About the Guests
Fyodor Urnov, Ph.D.
Professor; Scientific Director, Innovative Genomics Institute (IGI)
Dr. Fyodor Urnov is a Professor of Molecular Therapeutics at UC Berkeley and Scientific Director at the Innovative Genomics Institute (IGI), where he focuses on advancing CRISPR-based genome and epigenome editing for clinical use, including treatments for sickle cell disease, beta-thalassemia, and immune disorders. He co-developed the first human genome editing tools and led efforts to bring them to clinical applications. A co-founder of Tune Therapeutics, he also directs the Danaher-IGI Beacon for CRISPR Cures. Dr. Urnov holds a Ph.D. from Brown University and completed postdoctoral research at the NIH.
Jimi Olaghere
Patient Advocate, functionally cured from sickle cell disease by CRISPR
Jimi Olaghere, a technology entrepreneur, is a sickle cell disease survivor who shares his story of resilience and healing after undergoing a transformative gene therapy treatment. Born in Washington D.C. and fortunate to receive life-saving newborn screening, his journey highlights the disparity in healthcare access across Africa. Now, Jimi uses his experience to inspire change, advocating for widespread newborn screening and better healthcare for children affected by sickle cell disease.