Forget the next blockbuster drug. The new gold rush in the pharmaceutical industry is for the “blockbuster platform”, the foundational technology that can generate not just one, but a whole pipeline of cures. The first half of 2025 marked a pivotal moment in this shift, with Big Pharma betting billions to acquire companies armed with revolutionary cell and gene therapy engines.
Big Pharma bets billions on technology platforms
Large pharmaceutical companies have been aggressively pursuing high-value cell and gene therapy acquisitions and collaborations in H1 2025. The strategy appears to have shifted towards acquiring entire technology platforms capable of generating multiple future products, rather than single drug candidates. Key M&A deals included:
- AstraZeneca’s acquisition of Belgian biotech EsoBiotec for up to $1 billion, gaining its platform for developing in vivo CAR-T cell therapies.
- Eli Lilly’s $1.3 billion purchase of Verve Therapeutics to access its clinical-stage base editing platform for genetic conditions.
- AbbVie’s $2.1 billion acquisition of Capstan Therapeutics, a deal focused on solving the critical manufacturing and scalability challenges of CAR-T therapies.
- BioNTech’s $1.25 billion takeover of its German rival CureVac to consolidate its leadership in mRNA technology.
Landmark data confirms durability of gene therapies
A pivotal development was the presentation of landmark long-term follow-up data confirming the durable, multi-year efficacy of potentially curative gene therapies. At the European Hematology Association (EHA) 2025 Congress, Vertex presented data showing its CRISPR-based therapy, CASGEVY (exagamglogene autotemcel), provided transformative benefits for over 5.5 years in sickle cell disease and over 6 years in beta thalassemia patients. An overwhelming 95.6% of evaluable sickle cell patients remained free of debilitating pain crises for at least 12 consecutive months. This crucial evidence of sustained clinical value is fundamental to justifying the high upfront costs of these treatments and securing broad market access.
In vivo gene editing platforms show lasting promise
A major scientific advance was the validation of in vivo gene editing, where therapies are administered directly into the body to edit cells. Intellia Therapeutics presented compelling data for its CRISPR-based treatments, showing durable, multi-year efficacy. A single dose of its therapy for hereditary angioedema (HAE) resulted in patients being attack-free for a median of nearly two years, a 98% reduction in the attack rate. Its therapy for ATTR amyloidosis showed a 90% reduction in harmful protein levels, sustained for at least 24 months. This clinical success provides significant validation for platforms that could offer a more scalable and less complex alternative to traditional ex vivo cell therapies.
Venture capital cools, forcing a “Flight to Quality”
The investment climate for cell and gene therapy transitioned in H1 2025, as venture capital funding cooled considerably in the second quarter. The downturn reflects macroeconomic pressures and sector-specific challenges. Despite the overall slowdown, large “megarounds” of $100 million or more continued for a select group of companies with compelling human data. This indicates that discerning investors are concentrating larger bets on fewer, de-risked assets. Notable funding rounds included:
- Dispatch Bio (US): $216 million for a universal immunotherapy platform.
- Tune Therapeutics (US): $175 million for its epigenetic editing platform.
- Strand Therapeutics (US): $153 million for programmable mRNA therapy for solid tumors.
- Atsena Therapeutics (US): $150 million for an AAV gene therapy for an inherited retinal disease.
- SpliceBio (Spain): $135 million for an AAV gene therapy to treat Stargardt disease.
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A watertight IP strategy is what separates promising science from a high-value, fundable enterprise. Protecting your core technology is paramount to attracting investment, enabling partnerships, and securing a successful exit.
At Evolve, we are experts in IP strategy for cell and gene therapies. We have extensive experience developing and coordinating IP strategy to align with your critical regulatory and manufacturing functions.
Do you need advice on how to protect your cell therapy innovation? We would love to hear from you.
