The Discipline of Differentiation: Winning Strategies in a Crowded Cell and Gene Therapy Market

The cell and gene therapy industry has achieved breakthroughs once thought impossible, translating novel concepts into real therapeutic options for patients. The FDA has now approved 43 cell and gene therapies, and with these milestones, what was once a frontier space is now increasingly crowded. 

With over 1,800 active clinical trials and more than $11.7 billion in recent investment activity, we’re seeing this momentum sustained across platforms and indications. At the same time, the field is entering a period of recalibration. 

Investor and regulatory expectations have shifted, now demanding clinical viability, operational readiness, and scalable platforms. We’re seeing both favor programs that demonstrate near-term data and manufacturing feasibility over speculative science. With competition increasing, the path to success in this space now demands disciplined portfolio management, and a deeper understanding of where commercialization is heading. A strong program now goes beyond winning approval and must be able to withstand the realities of commercialization and delivery at scale. 

Differentiating in a crowded market 

In today’s increasingly competitive cell and gene therapy landscape, breakthrough innovations alone will not garner investor interest or ensure long-term sustainability. As leaders reassess pipelines and work to stand out in this crowded sector, platform differentiation and regulatory viability are the priority. These are the levers that will separate durable programs from stranded innovation.

Clinical maturity and indication strategy must also be weighed against market saturation and program development risks. As noted in recent investor analyses, early-stage or preclinical cell and gene platforms without differentiated biology or delivery are at risk of falling into a promising but un-fundable category. Programs that can deliver de-risking data early, and do so in an indication with established regulatory precedent, will remain appealing to investors.

Operating in a new regulatory landscape

The regulatory environment is also a leading factor in program viability. The regulatory landscape has further evolved in recent months and we’re seeing shifts in where the FDA is modernizing and willing to adapt. Under the current CBER leadership, we’ve seen openness to adaptive trial designs in rare and ultra-rare disease, as highlighted in discussions at the June FDA roundtable about the Baby KJ gene therapy case and discussions around “parachute trials”. But for higher-burden conditions like cardiovascular or autoimmune disease, assume the need for more rigorous comparator data.

Industry leaders must prioritize programs where the regulatory framework either already exists or is actively being shaped. Rare diseases with clear precedent, or broader diseases that can be part of the data-generation conversation.

Diversification, scalability, and manufacturing feasibility 

Following the 2021-2022 sector boom and valuation excitement, investors are now emphasizing early human data and strong IP positions. As an industry, we’re operating in a post-hype reality where the commercial viability of a therapeutic platform is judged by how well science can scale, diversify, and be manufactured with consistency and cost-efficiency.

Platform-based strategies

The past five years saw an influx of companies built around single lead assets or narrow, high-risk indications, but that model is being re-evaluated. As recent investor commentary and market movements have shown, platform-based strategies with diversified indications and shared infrastructure are more resilient in the wake of market volatility and regulatory shifts.

The deal between Sirius Therapeutics and CRISPR Therapeutics earlier this year is a prime example, expanding beyond gene editing into siRNA and broaden its reach into cardiovascular indications reflect a deliberate move to de-risk. In this environment, investors are favoring companies that can build platform velocity, leveraging one technology across multiple programs with shared infrastructure. 

Manufacturing feasibility is essential 

Manufacturing feasibility and the ability to scale at speed is a major gatekeeper. Delayed milestones are frequently tied to CMC complexity, vector shortages, and regulatory bottlenecks.

The FDA’s cell and gene therapy stakeholder roundtable highlighted this point, with multiple speakers emphasizing the need for national infrastructure, modular manufacturing platforms, and faster CMC development, especially for rare and ultra-rare disease programs. 

FDA platform designations and modular CMC strategies are beginning to reward repeatable frameworks. Earlier this summer, Sarepta won the first publicly documented Platform Technology Designation, signaling that the FDA is beginning to support modular approaches for cell and gene. This is particularly critical where viral vectors or delivery mechanisms are conserved across indications. Often, diversification and feasibility are added in response to late-stage friction, the industry must begin to build these strategies into early-stage decision-making. 

Aligning R&D spend and corporate strategy

In the current environment, breakthrough science must be matched by operational discipline. Organizations that can align R&D direction, capital deployment and corporate strategy will be best positioned for long-term success. As the regulatory and investment landscapes continue to evolve, decisions must be backed by data and plans must be flexible. 

Investor sentiment is shifting away from aspirational forecasts toward evidence-driven development plans. Capital decisions are increasingly influenced by clinical maturity, regulatory viability, and a program’s ability to scale. Across the board, we’re seeing a more deliberate approach to portfolio strategy, favoring fewer, high-conviction assets with clear near-term data potential and infrastructure leverage. This disciplined mindset emphasizes three guiding principles:

• Prioritizing programs with compelling clinical data and regulatory clarity
• Staging manufacturing investment based on scalability and supply chain readiness
• Remaining adaptable to changes in FDA guidance, payer expectations, and market signals
  

Program selection now hinges on near-term data, regulatory momentum, and the potential for shared infrastructure. Strategic focus has shifted toward fewer, high-conviction assets that are well positioned for both clinical success and operational execution. Maintaining alignment requires close collaboration between R&D, regulatory, and finance teams from the outset to ensure each program reinforces the platform’s long-term value.

Leveraging partnerships, emerging technologies, and flexible leadership

Market volatility, shifting regulations, and the challenges of delivering cell and gene therapies to patients are reshaping industry leadership and growth expectations.

Key partnerships can de-risk program execution

While building a solid infrastructure is critical, the current funding climate means companies have to determine whether they can or should build everything in-house. Academic and CDMO partnerships are critical in the face of capacity constraints and CMC delays, especially in rare disease programs where time-to-patient is paramount. Partnerships should be core to a cell and gene therapy business model, especially in areas like vector supply and clinical trial infrastructure. Partnerships have always been key in this sector, but as funding remains tight the right collaboration can unlock capabilities that may be impractical, or impossible, to build internally. At the same time, targeted outsourcing allows companies to focus internal resources where they’re most needed.

Shifting toward modular scalable platforms

Future ready leaders will be those who integrate flexible manufacturing strategies and innovative delivery technologies into development from the outset. At the June 2025 FDA roundtable, experts emphasized the importance of building modular, scalable infrastructure, including plug-and-play approaches like CRISPR paired with lipid nanoparticles (LNPs), to expand access and accelerate timelines.

David Liu, who leads several genome editing trials, called for the establishment of small-scale, rapid-turnaround CMC capabilities and shared platforms that can support multiple programs. These investments are intended to reduce complexity, enable repeatable regulatory and manufacturing pathways, and ensure development can scale with clinical and commercial demands. As the field matures, the ability to apply flexible platforms across indications will be a key differentiator.

Flexible management to remain strategic, not reactive

In today’s complex and competitive cell and gene therapy environment, leadership teams must take a proactive, integrated approach to decision-making. The most resilient organizations are building strategies that align funding, development, and operational planning early in the lifecycle, ensuring risks are identified and addressed up front. Cross-functional collaboration and end-to-end planning reduce the chance of costly delays and increase the likelihood of securing necessary funding. Partnerships, alternative financing strategies, and access to public-private resources are becoming essential to sustain progress and meet clinical and commercial milestones.

The cell and gene therapy sector has hit incredible milestones in recent years. As the sector continues to evolve and the climate becomes increasingly crowded and volatile, leaders must remain flexible in constantly refining the pipeline. 

While the bar is higher, there is still massive opportunity across the cell and gene therapy sector. As industry leaders evaluate their portfolios and work to maintain a competitive edge in an increasingly crowded sector, programs built on diversification, scalability, and manufacturing feasibility will lead the sector.

Photo: Gerasimov174, Getty Images

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