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As we get into Phacilitate’s 20th anniversary, Georgi Makin and Susan Nichols have been looking at the top 20 events that have shaped the industry over the past few decades.
Amidst significant advancements in healthcare, the development of advanced therapies has been a defining driver of progress over the last 20 years. From gene editing to CAR-T cells, these once-futuristic treatments are no longer science fiction, but tangible hope for many facing previously insurmountable challenges.
For this blog post, we have enlisted the support from Phacilitate community members with some of the deepest and longest-gathered industry knowledge bases to determine and celebrate our top 20 events from the past 20 years, while considering the future and anticipating the breathtaking possibilities that lie ahead.
FDA Approvals that Defined an Era
- 2010: PROVENGE, the first FDA-approved cell therapy, proved the viability of a completely new treatment paradigm.
- 2017: The CAR-T revolution exploded with KYMRIAH and YESCARTA, offering unprecedented levels of cancer remission and redefining the fight against the disease.
- 2017: LUXTURNA illuminated the path for gene therapy, offering sight to children once shrouded in darkness by a rare genetic disorder.
- 2019: ZOLGENSMA proved the power of gene therapy beyond rare diseases, treating children for a devastating neurodegenerative condition.
- 2022: Atara’s allogeneic T cell therapy in Europe opened a new chapter in transplant medicine, demonstrating the potential to revolutionize organ replacement.
Beyond Borders: Global Recognition and the Spread of Hope
- 2012: Clybera, the first gene therapy approved in the West, signaled Europe’s early commitment to innovation in this revolutionary field.
- 2015–2022: Approvals like HOLOCLAR (Italy) and STRIMVELIS (UK) showcase the global reach of advanced therapies, ensuring that hope transcends borders.
- 2023: CASGEVY, the first CRISPR-based therapy approved anywhere in the world, marks a historic moment in gene editing and a glimpse into the boundless potential of this technology.
From Nobel Laureates to Patients: Celebrating the Science and Stories Behind the Magic
- UPenn’s CD19 trial: This pioneering study, combining cell therapy and viral vectors, stands as a testament to the power of collaborative research and its ability to translate scientific brilliance into tangible patient benefits.
- 2020: Jennifer Doudna’s Nobel Prize for CRISPR underscores the transformative power of gene editing, a technology with the potential to rewrite the very blueprint of life.
- 2023: Katalin Karikó and Drew Weissman’s Nobel Prize for mRNA recognizes the foundation for COVID-19 vaccines and paves the way for a new era of personalized medicine.
Building the Infrastructure for Hope: From Advocacy to Manufacturing
- Alliance for Regenerative Medicine (ARM): Paving the regulatory pathway and ensuring a voice for the industry, this organization has been instrumental in bringing advanced therapies to patients.
- CBER Standards and Milestones: Providing a stable and evolving regulatory framework for advanced therapies, CBER plays a critical role in ensuring safety and efficacy while fostering innovation.
- Patient Organizations and Societies: From ARM to ASGCT, these groups connect patients and developers, driving advocacy and building a community of shared purpose.
Patient Stories that Inspired Progress
- Emily Whitehead: Facing a bleak prognosis after multiple relapses of childhood leukemia, Emily Whitehead became the first pediatric patient to receive groundbreaking CAR T-cell therapy, leading to a stunning remission and inspiring the field of personalized medicine.
- Victoria Gray: The first patient to be treated with a gene edited (CRISPR-based) therapeutic for sickle cell anemia.
Investing in the Future: Deals and Partnerships Fueling Progress
- Lonza’s 2006 acquisition: Launching a major player in the manufacturing space, this deal signaled the growing demand for specialized facilities to produce these complex therapies.
- Novartis and UPenn’s 2012 deal: This early partnership highlights the importance of collaboration between industry and academia in accelerating the development of new therapies.
- Pharma M&A: Akous, AstraZeneca, and Vertex’s acquisitions demonstrated the industry’s unwavering faith in the long-term potential of advanced therapies, fueling further investment and research.
Tools for a New Era: Manufacturing Revolutionizes Accessibility
Historically, tools and technologies were adapted from similar or adjacent industries rather than being developed specifically for cell and gene. However, specialized tools and technologies have emerged, designed specifically for the unique needs of advanced therapy production. These dedicated innovative tools are making these treatments safer, more efficient, and ultimately, more accessible to patients.
Looking Ahead
In just two decades, advanced therapies have gone from scientific dreams to tangible realities, offering hope and cures where none existed before. We’ve witnessed groundbreaking FDA approvals, global recognition, and scientific breakthroughs, all fueled by dedicated advocacy, patient stories, and industry investments. As we stand on the precipice of the future, we can confidently expect a tapestry of innovation, with more effective and accessible therapies woven into the fabric of healthcare.
The revolution is not over; it’s just gathering momentum.
