Top 20 Milestones in 20 Years of Advanced Therapies
As we mark 20 years of Phacilitate’s contribution to the advanced therapies ecosystem, we take a look back at the moments that have shaped this dynamic field. With insights from industry experts, pioneers, and patient advocates, these top 20 milestones represent progress, resilience, and the boundless future of cell and gene medicine.
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The Top 20 Milestones That Transformed Advanced Therapies1. FDA Approvals That Defined a Generation
Regulatory approvals have marked crucial turning points in the industry:
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2010: PROVENGE – The first FDA-approved cell therapy launched a new treatment paradigm.
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2017: KYMRIAH and YESCARTA – CAR-T therapies achieved landmark cancer remission rates.
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2017: LUXTURNA – First gene therapy for an inherited disease, restoring vision in children.
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2019: ZOLGENSMA – Gene therapy stepped beyond rare diseases to tackle SMA.
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2022: Atara’s Allogeneic T Cell Therapy – Opened doors to off-the-shelf solutions for transplant medicine in Europe.
The wave of innovation wasn’t limited to the U.S.:
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2012: Glybera (EU) – The West’s first approved gene therapy set the tone for global innovation.
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2015–2022: STRIMVELIS & HOLOCLAR – Expanded Europe’s leadership in therapy approval.
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2023: CASGEVY – The world’s first CRISPR-based treatment approval marked a new era for gene editing.
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UPenn’s CD19 Trial – A collaboration that redefined cancer treatment and viral vector application.
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2020: Jennifer Doudna’s Nobel Prize (CRISPR) – Acknowledged gene editing’s potential to rewrite DNA.
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2023: Karikó & Weissman’s Nobel (mRNA) – Honored the science behind COVID-19 vaccines and personalized medicine.
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Alliance for Regenerative Medicine (ARM) – Advocacy, education, and regulatory leadership.
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CBER’s Regulatory Milestones – Provided the evolving framework needed to evaluate safety and efficacy.
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Patient Societies (ARM, ASGCT) – Fostered community, accelerated collaboration, and drove public awareness.
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Emily Whitehead – The first child to receive CAR-T therapy, achieving a historic remission from leukemia.
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Victoria Gray – The first sickle cell patient treated with CRISPR-based gene editing, redefining what’s possible.
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2006: Lonza Acquisition – Marked a turning point in large-scale manufacturing capability.
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2012: Novartis & UPenn Deal – A landmark academic-industry partnership.
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Strategic M&A (Akous, AstraZeneca, Vertex) – Reinforced long-term faith in cell and gene therapy markets.
Initially borrowing tools from other sectors, the industry now benefits from platforms designed specifically for cell and gene workflows. Innovations in closed-system manufacturing, automation, and vector design have helped improve scalability, efficiency, and patient access.
Looking Ahead: A Future Built on 20 Years of InnovationIn just two decades, what once seemed like science fiction has become standard care for many. With groundbreaking FDA approvals, Nobel-recognized research, and inspiring patient journeys, advanced therapies are no longer emerging—they’re here. As infrastructure strengthens and innovation accelerates, the next 20 years promise even more accessible, personalized, and curative treatments.
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