Gene Therapy and Cell Therapy Industry Trends in the Second Half of February 2026
Advancements in approvals and applications, the presence of DMD, and a phase where 'manufacturing, funding, and intellectual property' determine competitiveness
Overview of the second half of February: From technical competition to implementation competition
The gene therapy and cell therapy industry in the second half of February 2026 was a period where, while there were successive advancements in product approvals and applications, development cancellations and intellectual property lawsuits were also prominent, highlighting once again the reality that technical capability alone is not enough to win. Topics ranged widely from mRNA vaccines, AAV gene therapy, CAR-T, base editing, exosomes, to non-viral delivery, but looking at the big picture, it seems the axis of competition in the industry is shifting from 'new therapeutic concepts' themselves to the 'integrated power of development, manufacturing, capital, and intellectual property to ensure delivery'.
mRNA field: Approvals and applications progress while IP disputes intensify
Particularly notable was the rush of approvals and applications in the mRNA field. Moderna received marketing authorization from the European Commission for its next-generation COVID-19 vaccine 'mNEXSPIKE (mRNA-1283)' for individuals aged 12 and older, and its BLA for the seasonal influenza vaccine candidate 'mRNA-1010' was accepted by the FDA. In addition, the EMA's CHMP adopted a positive opinion recommending marketing authorization for the influenza and COVID-19 combination vaccine 'mCOMBRIAX (mRNA-1083)'. While mRNA continues to progress in the infectious disease area, BioNTech has filed a patent infringement lawsuit against Moderna's mNEXSPIKE, making it clear that as market formation progresses, intellectual property disputes are also intensifying.
Personalized mRNA therapy: An era where manufacturing speed becomes a competitive advantage
The application of mRNA is not limited to infectious diseases. Southern RNA announced that it will handle the manufacturing of 'PaedNEO-VAX', a personalized mRNA cancer vaccine for pediatric refractory brain tumors sponsored by Providence Therapeutics. The system of manufacturing mRNA vaccines, individually designed according to each patient's tumor biology, within about 10 weeks of registration shows that manufacturing speed is becoming a competitive advantage in the implementation of personalized medicine. Furthermore, with eTheRNA immunotherapies and Almirall's mRNA therapeutic 'LAD116' for non-melanoma skin cancer, and the development of mRNA mucosal vaccines for pandemic influenza in which Ethris is participating, mRNA is expanding its use from vaccines to local immunotherapy and mucosal administration.
AAV gene therapy: Approval progress and development selection proceeding simultaneously
Meanwhile, in AAV gene therapy, 'the eve of approval' and 'development selection' are proceeding simultaneously. The BLA for Ultragenyx's 'DTX401' for glycogen storage disease type Ia was accepted by the FDA and granted priority review. If approved, it could become the first treatment option to address the root cause of the disease. In Japan, Chugai Pharmaceutical launched 'Elevidys intravenous infusion' for DMD, entering clinical practice as the first regenerative medicine product for DMD in the country. Furthermore, Sarepta is presenting long-term evidence for its entire DMD portfolio through 3-year functional assessments and up to 7.5-year safety analyses of delandistrogene moxeparvovec, as well as results from the phase 3 'ESSENCE' trial of its exon-skipping drug. DMD can be said to be a representative area where both gene therapy and nucleic acid medicine are competing to form the market.
The reality shown by development cancellations: The difficulty of gene delivery to the lungs
On the other hand, Boehringer Ingelheim decided to discontinue the development of its inhaled gene therapy 'BI 3720931' for cystic fibrosis. This was because clinical data justifying further development was not obtained in the phase 1/2 trial. CF has long been an area with high expectations in terms of gene transfer to the lungs, but it has once again been shown how difficult it is to simultaneously achieve delivery, expression persistence, safety, and clinically meaningful differences. Gene therapy does not advance just because it is 'theoretically correct'; it is being coldly questioned as to how much it can be reproduced in actual humans.
Rare disease area: New developments in Rett syndrome, SMA, and NKH
In the rare disease area, new movements continued in Rett syndrome, SMA, and NKH. Neurogene's 'NGN-401' for Rett syndrome received breakthrough therapy designation from the FDA, raising expectations for accelerated development. Furthermore, Evox Therapeutics announced a partnership with the Rett Syndrome Research Trust to evaluate an exosome-based gene editing approach using ExoEdit®. In CNS diseases, this suggests that platforms that are non-viral and allow for repeat dosing, rather than just AAV, are becoming the next main battlefield. Gemma Biotherapeutics has also administered the first dose in a phase 1/2 trial of 'GB221' for SMA1, aiming for differentiation through an approach of direct central administration using AAVhu68. In addition, the AAV manufacturing partnership for NKH between the Drake Rayden Foundation and Andelyn Biosciences is also attracting attention as a model for promoting development led by non-profit foundations in rare diseases.
Delivery technology: CNS delivery and non-viral approaches become the focus
The evolution of delivery technology was also an important theme during this period. In the partnership between Finding Hope for Frizzle and Apertura Gene Therapy, technology that utilizes the next-generation AAV capsid 'TfR1 CapX' to deliver genes across the blood-brain barrier to the CNS is at the forefront. For brain diseases, the trend of 'how to deliver' becoming the center of value, more than the therapeutic gene itself, is accelerating. Breeze Bio's non-viral delivery platform 'NanoGalaxy' is similar, impressing that the success or failure of mRNA therapeutics depends on new delivery technologies, including those other than LNPs. Including Beam Therapeutics launching the LNP-delivered base editing program 'BEAM-304' for PKU, delivery technology is no longer a peripheral element but a core asset that influences corporate value itself.
Cell therapy: Automation and manufacturing and expansion to solid tumors are the next battlegrounds
In cell therapy, 'automation/manufacturing' and 'expansion to solid tumors' were keywords. Cellares expanded its partnership with the University of Wisconsin and began clinical manufacturing and IND application support for CRISPR-edited GD2 CAR-T for solid tumors. Cell therapy still has major constraints in manufacturing costs and scalability, but the presence of companies with automated manufacturing infrastructure is increasingly growing. Elicera Therapeutics is also planning to update clinical data for its CAR-T 'ELC-301', which incorporates the iTANK platform, and attention is focused on confirming sustained effects and dose-dependency in B-cell lymphoma. Also, the news that Gilead has agreed to acquire Arcellx for approximately $7.8 billion clearly aims to maximize the commercial value of anito-cel, symbolizing the trend of large-scale restructuring where promising assets move toward integration rather than partnership.
Fundraising: Financial design to complete commercialization becomes important
In terms of fundraising, the second half of February 2026 was also impressive. Candel Therapeutics signed a $100 million royalty financing agreement with RTW Investments in anticipation of the market launch of CAN-2409 for prostate cancer. Beam Therapeutics also conducted a strategic fundraising of up to $500 million. Gene therapy and cell therapy remain capital-intensive businesses, and financial design that can carry a company from clinical results through approval application to commercialization is itself an important management issue. A phase continues where, in addition to the quality of R&D, the skill of capital policy significantly influences corporate valuation.
Preclinical/Early clinical: The value of early signals is increasing
The preclinical and early clinical stage pipelines were also diverse. Precision BioSciences plans to present preclinical data for its DMD treatment candidate 'PBGENE-DMD' using ARCUS® at the MDA conference, and Benitec has announced an update on data suggesting the long-term efficacy of 'BB-301' for OPMD. For Opus Genetics' 'OPGx-BEST1,' early signs of improvement in visual function and retinal structure were shown for inherited retinal diseases. While these are still in the early stages, they represent a step beyond mere safety confirmation, demonstrating that 'how to demonstrate early clinical signals' is directly linked to subsequent fundraising and partnerships.
Expansion of Boundary Areas: Spreading into Healthspan and Aging
Furthermore, the expansion of 'boundary areas' cannot be overlooked. Klothea Bio has initiated trials for its alpha-Klotho mRNA therapeutic 'AKL003' in healthy adults, with an eye toward aging-related biomarkers and the extension of healthspan. This differs from the conventional focus on rare diseases and cancer, suggesting the potential for gene and mRNA therapies to expand into the broader healthcare market in the future. However, since the organization of regulations, evaluation metrics, reimbursement, and ethics will be more critical in this type of area, careful assessment of future progress is necessary.
Summary: Three Perspectives for the Second Half of 2026
Overall, the industry trends from late February 2026 reveal the following three points.
First, approvals and applications are steadily advancing in the fields of mRNA, AAV, and cell therapy, and the transition to the commercialization phase is in full swing.
Second, 'peripheral elements' such as delivery, manufacturing, funding, and intellectual property are actually becoming the core of competitiveness.
Third, investment and development continue to be concentrated in areas with clear unmet needs, such as DMD, Rett syndrome, SMA, and rare metabolic diseases.
Future Points of Interest: Approval Reviews, DMD Competition, and Progress in CNS Delivery
Moving forward, simply having a promising modality will not be enough; an integrated strategy that includes which patients to reach, through which route, at what cost structure, and on what timeline will be required. Looking toward the second half of 2026, key points to watch will include cases pending PDUFA, intensifying competition in the DMD space, the outcome of intellectual property litigation in the mRNA field, and progress in non-viral CNS delivery.