CRISPR in 2025: Clinical Use, Regulatory Pressure, and Future Promise
Part 1 of a mini-series on gene therapies, from the perspective of a scientist and an investor.
Welcome back to Biotech Blueprint!
In this week’s special episode, I team up with the Biotech Capital Compass to examine where gene editing stands today, scientifically, clinically, and commercially. This kicks off a multi-part series on gene therapies, viewed through both scientific and investment lenses.
CRISPR-based technologies have seen extraordinary momentum over the past decade, moving from basic research to FDA-approved therapies. But despite early clinical wins and billions in investment, serious challenges remain, from off-target effects and delivery barriers to complex regulatory frameworks. In this episode, we focus on the current state of the science, how CRISPR is being used and regulated, and what still stands between proof-of-concept and scalable care.
Since its emergence in 2012, CRISPR has outpaced earlier gene-editing platforms like zinc finger nucleases and TALENs, becoming the dominant tool across research and development. Scientific output has exploded, and commercial interest has followed. Between 2013 and 2021, CRISPR-focused companies in the U.S. raised over $2 billion, backed by venture capital and strategic partnerships. Notable examples include Bayer’s $335 million investment in CRISPR Therapeutics and Vertex’s collaboration that led to Casgevy, the first FDA-approved CRISPR-based therapy.
In part 1 of our series, we establish the scientific and regulatory foundation. We break discuss the distinction between broader gene therapy and gene editing, discuss the significance of early approvals like Casgevy, and look at the regulatory scaffolding now emerging around this field, from the FDA’s RMAT designation to evolving EMA guidance. We also talk about what’s still missing: delivery remains unsolved, durability is not well established, and manufacturing at scale poses real challenges for commercialization.
Translating gene-editing science into reliable medicine is anything but straightforward. Off-target effects, though reduced with high-fidelity Cas variants and optimized guide RNAs, still pose risks in in vivo settings. And gene-edited products demand long-term patient monitoring to assess both safety and therapeutic durability. That raises logistical, regulatory, and economic hurdles that must be factored into trial design, investor expectations, and payer decisions.
Still, the regulatory path is beginning to take shape. Programs like RMAT and parallel initiatives in Europe are accelerating timelines, but also raising expectations. Regulators increasingly demand not just strong data, but durable outcomes across diverse populations. Clinical trial design is evolving in parallel, with single-arm studies, synthetic controls, and real-world evidence all playing a larger role in what ultimately earns approval.
Gene editing is no longer theoretical, but it’s far from mature. In upcoming episodes, we’ll explore how companies are building the infrastructure to support this modality: from investor strategy to AI-enabled design tools and platform scalability. CRISPR may be the breakthrough. But what comes after the breakthrough is what will define its role in medicine.
Stay tuned!
DISCLAIMER: This content is for informational purposes only. It should not be taken as legal, tax, investment, financial, or other advice. The views expressed here are my own and do not reflect the opinions of any company or institution.
DISCLOSURE: I have no business relationships with any company mentioned in this article.