As we cross the mid-point of 2025, the momentum behind advanced therapies, particularly cell and gene therapies, has never been more pronounced. From bespoke gene editing to the commercial scaling of landmark treatments, the sector stands on the threshold of transformation. For those working across the pharmaceutical and health markets, these advances present both extraordinary opportunities and fresh complexities, underscoring the need for agile, forward-looking strategies.

Regulatory Developments: Accelerating Access and Innovation

Regulatory bodies on both sides of the Atlantic continue to recalibrate frameworks in response to rapid therapeutic progress. The UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) has implemented a decentralised manufacturing framework, effective from July 2025, supporting the more accessible and point-of-care delivery of advanced therapeutics. This regulatory flexibility not only reduces time-to-patient but also sets a model for other jurisdictions seeking to foster innovation without compromising safety.

Across Europe, recent months have seen expedited assessments for ground-breaking therapies targeting rare and high-burden diseases. In the United States, the FDA’s calendar for the second half of 2025 is crowded with milestone Prescription Drug User Fee Act (PDUFA) dates, including the highly anticipated verdict on deramiocel for DMD-related cardiomyopathy, expected 31 August 2025 and pivotal decisions on gene therapies for mucopolysaccharidosis type 2 and recurrent respiratory papillomatosis. This pace is mirrored globally, as agencies work closely with industry to ensure that scientific progress translates swiftly into patient benefit.

Market Growth: Commercial Milestones and Confidence in Scale

The commercial story of advanced therapies continues to impress. For the first time, celebrated products such as Zolgensma (AAV gene therapy for spinal muscular atrophy) and Yescarta (CAR-T cell therapy for lymphoma) have surpassed US$1 billion in annual sales, cementing advanced therapies as pivotal pillars of the biopharmaceutical market. Major fundraising rounds such as Capstan Therapeutics’ US$175 million for in vivo CAR-T and ArsenalBio’s US$325 million for solid tumour platforms further underscore investor confidence in the next generation of precision cell therapies.

Corporate strategies are evolving in response, with new approaches to pricing, reimbursement, and infrastructure investment. Performance-based reimbursement schemes such as Germany’s programme for HEMGENIX in haemophilia B, which ties payment to individual patient outcomes, represent a first-of-its-kind model in the country. This signals a broader willingness among payers to embrace innovative value propositions, provided that the therapeutic benefit can be robustly demonstrated. In parallel, the US Centres for Medicare & Medicaid Services (CMS) is piloting a Cell and Gene Therapy Access Model, aiming to negotiate outcomes-based agreements for Medicaid patients, potentially reshaping access across public systems.

Therapeutic Breakthroughs: From Tailored Gene Editing to Broadening Indications

2025 has marked a turning point for personalised gene therapies. Notably, clinicians at the Children’s Hospital of Philadelphia achieved the first successful bespoke CRISPR-based therapy for carbamoyl phosphate synthetase 1 (CPS1) deficiency, leveraging an “N-of-1” regulatory pathway. Designed, approved and delivered within eight months, this paradigm exemplifies what is now possible when science, regulation and clinical care converge with a focus on individual patients. Such regulatory creativity opens new doors for rare and ultra-rare indications, shifting perceptions of what constitutes a feasible development timeline.

Elsewhere, late-stage trials continue to deliver for broader populations. Ocugen’s OCU410 for geographic atrophy has completed Phase 2 enrolment, while Nanoscope Therapeutics has advanced a first-of-its-kind, gene-agnostic BLA submission for retinitis pigmentosa. CAR-T and stem cell therapies are expanding in both their range of indications and commercial reach, moving into outpatient care models and accelerating timelines for diseases previously considered intractable.

Meanwhile, AI and robotics are reshaping the operational model, from accelerating target discovery to enabling automated, decentralised production. These technologies are not only driving clinical innovation but are also anticipated to usher in the next wave of cost containment and operational scalability, which is vital for market sustainability as volumes increase.

Major Players & Role of CDMOs as Powerhouses of Advanced Therapies Market

Leading companies, such as Bluebird Bio, Novartis, Spark Therapeutics, and Sarepta Therapeutics, continue to drive progress across gene and cell therapies, particularly in areas such as neuromuscular disorders, ophthalmology, and oncology. In regenerative medicine, firms including Vericel Corporation and Organogenesis are advancing the commercial case for tissue-engineered products, thereby expanding the therapeutic potential of cell-based interventions.

At the same time, CDMOs like Lonza, WuXi Advanced Therapies, and AGC Biologics are playing an increasingly central role in scaling production, accelerating timelines, and ensuring compliance. As decentralised and point-of-care manufacturing models gain traction, these partners are not just enabling delivery—they are powering the infrastructure behind the next wave of advanced therapies.

Navigating Challenges: Cost, Complexity, and Access

Despite remarkable progress, persistent challenges demand attention. Prime among these is the cost of personalised and ultra-orphan therapies. While the NHS’s rollout of gene-editing treatments for sickle cell disease (US$£2.2 million per patient) demonstrates commitment, such initiatives also place urgent focus on sustainability, equity, and long-term health system integration.

Furthermore, regulatory agencies and industry are collaborating to address manufacturing bottlenecks and Chemistry, Manufacturing, and Controls (CMC) hurdles, as evidenced by recent Complete Response Letters (CRLs) from the FDA. These instances, although disappointing in the short term, reflect the maturing regulatory dialogue and provide clarity to support robust and scalable manufacturing and supply.

Why It Matters: A Field at an Inflection Point

For professionals in pharma and health, advanced therapies now constitute a dynamic, high-stakes arena where scientific prowess must be matched by commercial acumen and operational excellence. The current climate rewards those who can navigate complex regulatory landscapes, anticipate value-based care trends, and forge collaborations that unite academia, industry, and healthcare systems.

As advanced therapies transition from promise to practice, their impact will be measured not only in scientific firsts, but also in the number of lives changed. The field’s maturation demands a holistic, systems-level approach ensuring that innovation remains tethered to the needs of patients and that markets are ready to support the therapies of tomorrow.

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