Reporting Q4 and full year earnings on Wednesday, J&J executives hailed growth across the healthcare giant’s portfolio while standing fast on its talc lawsuit and tariffs.

Johnson & Johnson is eyeing $100 billion in revenue, setting up a head-to-head competition with Eli Lilly in a race to new heights in the U.S. pharma market. J&J clocked $94.2 billion worldwide for 2025, with expectations of reaching even higher in the year to come.

The 2025 revenue number represents 6% growth in worldwide sales for the company, according to a fourth quarter and year-end earnings release on Wednesday morning. The results show that J&J has easily persisted past loss-of-exclusivity for some of its best-selling drugs. “We came out of really successful 2025,” CEO Joaquin Duato said on a Wednesday conference call, “firmly placing the Stelara LoE in the rearview mirror.”

Stelara, the company’s blockbuster autoimmune drug, has seen dropping revenue since losing patent protection in 2024. Last year’s sales declined 42.7%, still clearing $6.1 billion but a far cry from 2024’s $10.3 billion.

Nevertheless, the mood on the call was rosey, as the continuing loss from Stelara was buffered by growth in almost every other area of the company’s portfolio.

J&J now expects 2026 sales to reach $100.5 billion, 1.6% above consensus of $98.9 billion, according to CFO Joseph Wolk. He pointed to growing sales for franchises like dermatitis treatment Tremfaya, which rose from $3.6 billion in 2024 to $5.2 billion in 2025, as well as depression treatment Spravato, which improved from $1.1 billion to $1.7 billion.

The esketamine spray should be a cornerstone seller for the company, analysts at Jefferies wrote ahead of the call, saying that guidance for its sales out to 2027–2028 are set at $3 billion to $3.5 billion.

The analysts added that they see “a warming regulatory environment” for psychedelic-related treatments like Spravato in other indications, including anxiety and post-traumatic stress disorder.

J&J’s oncology portfolio drove most of the growth, however, going from $20 billion across all drugs to $25 billion year-over-year, driven by sales for anti-tumor antibody Darzalex and CAR T treatment Carvykti, which crossed into blockbuster status for the first time with $1.8 billion in 2025 sales.

J&J mostly breezed past the impacts of regulatory headwinds coming from Washington, D.C. Wolk noted that potential tariffs affecting its medical technologies division amounted to approximately $500 million, significantly above 2025 levies, but said, “our financial strength is a competitive advantage that allows us to both invest in our future and return value to our shareholders.”

Wolk also added that the company expects that tariffs “will be relatively linear in 2026, unlike last year.”

On the subject of the company’s continuing litigation into its talc product, which has been dogging the company for years as plaintiffs say the discontinued product is linked to ovarian and cervical cancers, company executives stood firm.

Yesterday, a court-designated special master would not allow certain expert witnesses to testify in court while also allowing plaintiffs to present evidence. In response, Wolk said that the special master “correctly decided to exclude” those experts, who he said “preponded junk science,” while also bemoaning that “the court did not uphold its proper gatekeeping duty” in allowing plaintiffs to present evidence.

In November, Pfizer was reportedly looking to divest its stake in BioNTech, though the German biotech at the time denied these rumors.

In a vaccines-focused deal, Pfizer is fronting $30 million to partner with Novavax, just a few months after the pharma was rumored to be distancing itself from its other big vaccine collaborator, BioNTech.

Aside from its upfront commitment, Pfizer will put up to $500 million on the line in development and sales milestones, according to a Tuesday news release. Novavax will also be entitled to high-mid-single digit percentage royalties on net sales that might result from the partnership.

In exchange for its investments, Pfizer will receive a non-exclusive license to Novavax’ Matrix-M adjuvant technology, which makes use of naturally occurring compounds from soapbark trees, allowing for a “targeted and efficient way” of boosting the body’s immune response, according to the company’s website.

Pfizer will be able to apply the adjuvant to up to two disease areas, according to the press announcement, though it remains unclear what these indications are or if the company has a set number of programs in mind.

Tuesday’s vaccines pact with Novavax follows reports in November 2025 that Pfizer was considering divesting its stake in vaccine partner BioNTech, with which it owns the COVID-19 shot Comirnaty. Pfizer at the time remained tight-lipped about the rumors, and BioNTech denied the news: “We continue to have a close and strong collaboration,” a spokesperson for the company told Reuters, otherwise refusing to comment on Pfizer’s business dealings.

The Novavax deal also comes amid growing skepticism toward vaccines, driven in no small part by controversial policies and antagonistic rhetoric from the Trump administration. Last month, for instance, Vinay Prasad, director of the FDA’s Center for Biologics Evaluation and Research, claimed, without providing evidence, that “at least” 10 children had died “because of” COVID-19 vaccines. An internal FDA memo released two weeks later stated that the claim was an overestimate.

But at the J.P. Morgan Healthcare Conference last week, Sanofi CEO Paul Hudson said these high-level headwinds present a business-making opportunity. “If you’re a short-term thinker, you don’t move,” he said, referring to the high level of uncertainty in the vaccine space right now. “If you’re a long-term thinker—which is what we have to be—then there are less people to compete against to make acquisitions.”

The arrangement will boost AstraZeneca’s cell therapy portfolio as the pharma targets $80 billion in revenue by 2030.

AstraZeneca has doubled down on its CAR T collaboration with Shanghai-based AbelZeta, paying $630 million to scoop up all remaining rights to develop and commercialize the investigational autologous cancer therapy C-CAR301 in China.

With the deal, AstraZeneca now has sole rights over C-CAR031 globally, according to a Sunday news release.

AstraZeneca and AbelZeta first linked up in December 2023, with the pharma paying an undisclosed amount for global ownership of C-CAR031. In China, however, this agreement only gave AstraZeneca co-development and commercialization rights alongside AbelZeta. Now, AstraZeneca is paying the additional $630 million to buy out its Chinese partner.

Sunday’s agreement, according to AbelZeta CEO Tony Liu, will help “maximize C-CAR031’s global reach.” This deal is also AstraZeneca’s second China deal in as many months. The pharma last month put $2 billion on the line to collaborate with Jacobio Pharma on an early-stage pan-KRAS blocker, currently in early development for cancer.

C-CAR031 is an autologous CAR T therapy that targets the glypican 3 protein. Located on the surface of cells, glypican 3 has in recent years been found to be frequently overexpressed in a variety of cancers, particularly hepatocellular carcinoma, an indication in which C-CAR031 is being tested.

First-in-human data published in 2024 pointed to an objective response rate of 56.5%, with efficacy figures reaching as high as 75% for the top dose level. Tumors shrank in more than 90% of patients.

C-CAR031 was designed using AstraZeneca’s proprietary armoring platform, which creates constructs that can withstand the tumor’s microenvironment, which otherwise would deactivate and destroy cell therapies. This armoring involves modifying the CAR cells to disrupt the TGFβ receptor, which would otherwise suppress immune activity around the tumor.

Beyond its armored cell therapies, AstraZeneca is also advancing AZD0120, an investigational CAR T therapy designed to target both CD19 and BCMA for the treatment of multiple myeloma. Phase Ib/II data presented last November showed a complete response and stringent complete response rate of 78.3%, with partial response hitting 17.4%.

The pharma is also advancing the bispecific T cell engager surovatamig, which is currently in late-stage development for diffuse large B-cell lymphoma.

Cell therapies like AZD0120 and C-CAR031, in addition to its antibody-drug conjugate programs, will pave the path for AstraZeneca’s ambitions to hit $80 billion in revenue by 2030, CFO Aradhana Sarin said at a company event during the recently concluded J.P. Morgan Healthcare Conference.

Henry Gosebruch, who has $3.5 billion in capital to deploy, is thinking broad as he steers the decades-old biotech out of years of turmoil. 

Galapagos CEO Henry Gosebruch truly has a clean slate to work with in rebuilding the storied—but chronically unlucky—company.

Brought in last year to replace Paul Stoffels, who made multiple attempts to right the ship, Gosebruch’s mandate is to find a whole new direction. The company has suffered from a series of clinical failures, FDA rejections, restructuring and leadership changes. He has cash—about €3 billion ($3.5 billion)—and the board’s backing to find whatever asset, technology or disease area he wants.

“On the one hand, it’s like, well, what’s it going to be? But on the other hand, that’s the beauty of what we have, that we can be much more broad in our thinking and really just look at the best opportunity,” Gosebruch told BioSpace on the sidelines of the J.P. Morgan Healthcare Conference last week.

This wide-open opportunity is what attracted Gosebruch in the first place—even if the role he was originally offered was quite different. He was brought into the fold in April 2025 to head a proposed spin-out company that would build a brand new pipeline. Barely a month later, the company reversed course on the spin-out idea and announced Stoffels’ departure. Gosebruch instead became CEO of the original Galapagos entity, which would carry on the mission of finding a new pipeline.

Later, Galapagos ditched the cell therapy assets that Stoffels had been carefully curating over his nearly five-year term through a series of acquisitions. Explaining the decision to back off cell therapy, Gosebruch said it was a long time coming. The investment needed would have been too great to advance the technology far enough to be competitive. Gosebruch said it would have cost hundreds of millions of dollars.

Those conversations were happening well before Gosebruch arrived, but they intensified in the past year. On January 5, the board officially announced the wind-down of cell therapy at Galapagos. Galapagos originally intended to find partners for the existing programs, which include the Phase II CD19 CAR T GLPG5101 for blood cancer, but that has not materialized.

“We’re continuing to be open, but at this point, nobody’s come forward, really, with a proposal that makes sense to pursue,” Gosebruch said.

New Outlook

Of his predecessor Stoffels, Gosebruch expressed his admiration and said the board made the decision to part ways.

“Paul for many, many years and I have a lot of respect, but he was very, very focused on cell therapy,” Gosebruch explained. “That was sort of his mission, to bring this set of programs to patients. And I think, given how focused he was on that particular mission, I think he was not focused on opening up to other potential opportunities.”

Gosebruch, on the other hand, has an open mind: “My approach will be that I’m not wedded to one area over another; I’m going to be looking more broadly.”

Galapagos does have a legacy pipeline consisting of one molecule, a TYK2 small molecule for immunology called GLPG3667. It’s “the one program that survived from the sort of olden days,” Gosebruch said. The therapy is currently being tested in Phase II trials for systemic lupus erythematosus and dermatomyositis.

But GLPG3667 may not ultimately stay in Galapagos’ hands either, Gosebruch said.

“Now we are thinking about, given that we don’t have the broad development team anymore, we don’t have the big infrastructure anymore that we had back in the day, does it make sense for us to rebuild around that asset, or does it make sense for us to work with a partner who may already have that?”

No ‘Synergy’ Here

The new CEO was tight lipped on what he wants to bring into Galapagos. He noted that, at the J.P. Morgan conference, he had “a very packed calendar of BD discussions.” He was also meeting with investors to explain the company’s new focus.

Immunology and inflammation and solid tumors are likely candidates and would match Galapagos’ historical focus.

“I’d love to find a way to still see that vision through,” the CEO said. “And I think there are some very exciting opportunities in immunology.” Gosebruch also spent time at I&I king AbbVie, so assets in this area would be complimentary to his experience, while solid tumors would fit in with long-time partner Gilead.

Galapagos still has strong ties with Gilead, which has a major investment in the biotech. Gosebruch said the relationship is strong and that he spent time with the pharma’s leadership even before accepting the role to understand what they want out of the partnership. He doesn’t think Gilead will simply hand off a program to Galapagos—the goal is to spend the capital on hand to find new opportunities, Gosebruch said.

Gosebruch added that the company has a team exploring China for opportunities, just like many peers across the biopharma industry. “I think it’s—I’m going to use the word required—actually,” Gosebruch said. “If you want to do BD these days, you have to be in China, at least to understand what’s the competitive landscape.”

One important piece of leverage that Galapagos has in business development discussions is that the biotech has shrunk down over the past year via layoffs. After the cell therapy restructuring is complete, Gosebruch estimated the remaining workforce will be about 35-40 people. So a whole company could join Galapagos without risk of layoffs—which commonly happen after a merger.

“One of the things we can offer to a potential transaction party is that if there’s a strong R&D team or a strong commercial team, they could, as a full team, come to Galapagos and be our team going forward,” Gosebruch said. “There won’t be any synergy.”

Gosebruch didn’t write off any potential diseases or opportunities and said he plans to be thoughtful—while acknowledging that investors have been very patient amid the restructuring.

“I’d rather take more time, be patient and wait for the right opportunity to come our way than rush into anything.”

Following the hard-won success of early anti-amyloid drugs, a new generation of Alzheimer’s modalities—from tau-targeting gene silencers to blood-brain barrier delivery platforms—is entering the pipeline to anchor future combination therapies.

The development of treatments for Alzheimer’s disease has been marked by consistent disappointment, with a demoralizing 99% candidate failure rate. However, recent market entrants—beginning with Biogen and Eisai’s ill-fated Aduhelm, then their more prosperous Leqembi and Eli Lilly’s Kisunla—have defied these odds.

With proof that progress is possible in this space, a new wave of treatments has emerged in the early-stage pipeline. Many of these candidates are taking novel approaches to the disease, whether through gene silencing, antisense platforms or improved drug-delivery technologies.

Having various modalities with different targets coming through the pipeline could be especially important as the treatment landscape matures, with a combination of approaches likely to be key to managing Alzheimer’s disease, Patrick Trucchio, managing director of Equity Research at H.C. Wainwright, told BioSpace. Several companies, including Eisai, Bristol Myers Squibb and Merck, are working on another common Alzheimer’s target: the tau protein.

“KOLs . . . expect it will take a combination of anti-amyloid, anti-tau and then some sort of neuroinflammatory drug to address the disease,” he said. “In terms of tau-targeting, I think the next two years will determine which candidates and which type of approach is going to be paired with the amyloid drugs.”

Against this backdrop, attention is turning to the next generation of Alzheimer’s drugs that could form the backbone of future combination therapies.

Biogen’s BIIB080

Antisense oligonucleotide

Biogen was there in the beginning, and the company remains in the thick of the next generation of Alzheimer’s R&D. BIIB080, a tau-targeting antisense oligonucleotide (ASO) granted FDA Fast Track designation last year, is currently being evaluated in Phase II trials. A popular target, the tau protein forms into toxic tangles in the brains of individuals living with Alzheimer’s.

Phase Ib data presented at the International Conference on Alzheimer’s and Parkinson’s Diseases in 2023 showed BIIB080 reduced soluble tau protein in cerebrospinal fluid (CSF) in patients with early-stage Alzheimer’s. The clinical study was the first to show a reduction of this magnitude in tau PET across brain regions, Biogen said in its press release at the time. The ASO therapy reduced biomarkers of tau in all dose groups by approximately 60% compared to baseline. Biogen announced in April 2025 that the Phase II CELIA trial of BIIB080 was fully enrolled. The study’s completion date is set for May, with readouts expected this year.

This readout will be one of the most significant for Alzheimer’s in 2026, Laura Nisenbaum, executive director of drug development at the Alzheimer’s Drug Discovery Foundation (ADDF), told BioSpace.

“So far, the drug appears to be safe and to reduce tau levels in the brain,” she said. “What we’re looking to see next is whether this holds across more individuals, continues to be safe and possibly has an impact on cognitive symptoms.”

Arrowhead Pharmaceuticals’ ARO-MAPT

RNA interference therapy

California-based Arrowhead is also working on a tau-targeting asset, an investigational RNA interference therapeutic called ARO-MAPT. Arrowhead launched a Phase I/IIa trial of the treatment in Alzheimer’s and other tauopathies last month.

Traditional methods of targeting tau, such as antibodies and small molecules, have had difficulty in modulating the protein, which makes it ideal for a siRNA-mediated knockdown approach, James Hamilton, chief medical officer at Arrowhead, told BioSpace in an email.

The primary reason for the other approaches’ struggles, Hamilton said, is that “tau monoclonals do not adequately target intracellular tau neurofibrillary tangles, which are one of the key drivers of disease biology.” ARO-MAPT, on the other hand, “targets tau mRNA expression in neurons and is administered by a subcutaneous injection,” he explained.

Arrowhead is hoping its study will show reductions in CSF translating into lower levels of regional brain tau, Hamilton said. He noted that the study will also include cognitive measures and other disease-relevant rating scales but cautioned that the trial is likely too small to show an effect on cognition, with this set to be assessed in Phase II trials.

The company is anticipating initial data from the first parts of the study in the second half of this year, Hamilton said in the December press release announcing the trial’s launch.

With Arrowhead’s and other significant readouts expected this year, Trucchio referred to 2026 as “the year of tau,” adding that 2027 will also be crucial. The data from these trials will be important because they will help confirm the most appropriate approach, Trucchio said, likening the different methods of tau reduction to either “a chisel or a sledgehammer.”

Alnylam’s Mivelsiran

RNA interference therapy

Alnylam’s mivelsiran, also an siRNA therapy, is being tested in Phase I trials for Alzheimer’s disease. Plans were underway to initiate a Phase II trial during the fourth quarter of 2025, according to the biotech’s third-quarter 2025 earnings report, but as of publication no such trial was listed on ClinicalTrials.gov.

While part of the same class as ARO-MAPT, mivelsiran is delivered via intrathecal rather than subcutaneous injection. Alnylam is targeting amyloid in its Phase I trial, while a further asset, ALN-5288, also a siRNA, is being explored to target tau.

“With RNAi, we can target amyloid-beta and tau at their genetic source, addressing both intracellular and extracellular pools of these problematic proteins,” Tim Mooney, program lead for mivelsiran at Alnylam, told BioSpace in an email. “This upstream approach may be able to more comprehensively address drivers of Alzheimer’s progression compared to other modalities.”

Echoing Trucchio, Mooney said Alnylam expects the future of Alzheimer’s treatment to comprise a variety of approaches and combinations to provide “a more comprehensive effect.” This includes looking at approaches that extend beyond targeting amyloid and/or tau proteins, he added, with Alnylam “exploring multiple mechanisms” to address the disease.

“Looking further into the future, we anticipate that diagnostic screening with blood-based biomarkers may eventually play a role in helping to identify individuals at risk of developing AD who could benefit from treatment with targeted therapeutic approaches like ours to prevent the onset of dementia altogether,” Mooney said.

Alector Therapeutics’ AL137

Next-gen antibody paired with brain carrier technology

Alector suffered two setbacks to its neurodegenerative pipeline, experiencing clinical trial failures in its partnerships with both AbbVie and GSK in a 13-month span. The biotech is still developing AL101 alongside GSK for Alzheimer’s but has pivoted its approach in its earlier-stage pipeline.

AL137, a preclinical anti-amyloid beta antibody, is being paired with Alector’s “brain carrier” (ABC) delivery technology to target Alzheimer’s disease. This design is intended to counter one of the major barriers to administering treatments for Alzheimer’s by navigating the blood-brain barrier (BBB).

Alector’s ABC technology uses receptor-mediated transcytosis to cross the BBB, targeting specific receptors of endothelial cells to facilitate the delivery of therapeutics. This delivery technology is designed to improve drug delivery and also to reduce dose levels whilst maintaining efficacy, according to the company’s website.

Navigating the BBB could help to prevent one of the key known side effects of the anti-amyloid antibody class: amyloid-related imaging abnormalities (ARIA), Trucchio said. Last July, Lilly’s Kisunla received a label update for its dosing schedule to reduce the likelihood of ARIA.

ARIA prevention is now becoming a key differentiator that companies in this space hope to achieve, Trucchio said, adding that analysts will be looking to the next-generation anti-amyloid therapies or delivery platforms to deliver this.

As the potential for improved delivery of Alzheimer’s treatments becomes clear, companies emerging later into the space—or those on the rebound, such as Roche with its antibody trontinemab—are leveraging this as a means to catch up to the early frontrunners.

The progress currently being seen in the Alzheimer’s space is lifting the negativity that existed in the field five years ago, Nisenbaum said.

“With the success of anti-amyloid therapies and the development and approval of blood-based biomarker tests, we’ve seen renewed excitement and a greater willingness from drug developers to enter this space.”

Many natural compounds that act on the human body provide active ingredients for medicines or clues for developing them, and they play a crucial role in pharmaceutical research.

Among these natural compounds, many molecules classified as alkaloids—such as caffeine, nicotine and morphine—are highly complex, and often available only in very small quantities. To understand how alkaloids act in the body, it is important to grow large crystals of the molecules and determine their structures by X-ray diffraction. However, for molecules like alkaloids, crystal growth itself is difficult.

The crystalline sponge method explained

A method that addresses this challenge is the crystalline sponge method. In this approach, molecules are absorbed into a crystalline material with regularly arranged, sponge-like pores, and researchers observe how the molecules are fixed within those pores.

The crystalline sponge used for this purpose is a metal–organic framework (MOF), a class of materials recognized by the Nobel Prize in Chemistry in 2025. Because MOFs have orderly, lattice-like pores, they are well suited to the crystalline sponge method. However, a longstanding drawback has been that when highly reactive molecules such as alkaloids are introduced, the MOF itself can collapse.

Development of the APF-80 MOF

A research team led by Professor Masaki Kawano and Assistant Professor Yuki Wada at Institute of Science Tokyo (Science Tokyo) has created APF-80, a new MOF that resolves the long-standing issue of MOF fragility, which has hindered wider use of the crystalline sponge method.

The work is published in the Journal of the American Chemical Society.

APF-80 is a MOF composed of metal ions (cobalt) and organic molecules. The team identified and incorporated an organic molecule that strengthens the crystal itself more effectively than in conventional MOFs. In addition, they introduced a design that prevents the crystal from degrading by shielding the parts of the structure where reactions are likely to occur, even when highly reactive molecules are introduced.

They also implemented a structural design in which the absorbed molecules cooperate with the MOF and function like an adhesive. Through these improvements, molecules no longer wobble inside the crystal; instead, they become firmly immobilized—as if they hold perfectly still just before a photograph is taken.

Breakthroughs in molecular visualization

Using this approach, the team was able to visualize the three-dimensional structures of a wide range of compounds—including caffeine, nicotine, and omeprazole (an active ingredient in stomach medicine)—which would previously have caused MOF crystal degradation. Thanks to the high stability of APF-80, the structures of the compounds could be observed with clarity.

The method also succeeded in directly revealing the shapes of compounds whose structures had not been determined before. Remarkably, it can even distinguish between two closely similar molecules, quinine (a component used to treat malaria) and quinidine (a component used to treat arrhythmia). In other words, it has become possible to capture subtle molecular differences, much like identifying fingerprints.

Implications for research and industry

Advancing this technology will significantly accelerate pharmaceutical research. Because it enables structural analysis from only minute amounts of material, it can support faster and more accurate characterization of natural products and novel drug candidates. Moreover, “molecule-encapsulating crystals” like APF-80 are expected to find applications beyond pharmaceuticals, including fragrance compounds, catalysts, and even energy-related materials.

As a new method for more clearly depicting molecular structures that were previously out of reach, this achievement greatly expands future possibilities in the molecular world.

“A crystalline sponge is like building a small photography studio in the molecular world—one that has been difficult to observe until now. With APF-80, even challenging molecules that could not previously be analyzed can now be captured more clearly. Through further research, we will elucidate the structures of many more compounds and contribute to a wide range of research,” says Yuki Wada.

“Seeing is believing—the crystalline sponge method is a way to make that saying real. APF-80 has made it possible to observe compounds that could not be analyzed before. I believe it will grow into a powerful crystalline tool that supports emerging drug discovery and materials research. Through TEKMOF, a spin-off from Science Tokyo, we are exploring this research with the goal of social implementation across multiple industries,” says Kawano.

AI is moving from a supporting role into the core of drug discovery. By 2026, it is expected to shape how targets are chosen, how biology is analysed and how development decisions are made.

Artificial intelligence (AI) is moving from isolated applications into the core of drug discovery. By 2026, it is expected to influence how targets are identified, how biological data are analysed and how clinical development decisions are made.

As AI tools are applied earlier in discovery, computational prediction is increasingly used alongside experimental validation rather than after it. This allows teams to test biological hypotheses earlier, build confidence sooner and reduce late-stage failure.

AI-guided target identification becomes the starting point

In early discovery, target identification has traditionally involved reviewing published evidence, developing biological hypotheses and validating them through repeated laboratory experiments. This process can be slow and often relies on partial views of disease biology. By 2026, early target selection is expected to depend far more on computational analysis, enabling scientists to interrogate large biological datasets before committing to wet-lab work.

“In 2026, identifying disease targets will rely on in silico exploration before any wet-lab validation begins,” says Veronica DeFelice, Director of Biologics at Sapio Sciences. She explains that AI-guided platforms connected to laboratory information management systems will integrate genomic, proteomic and transcriptomic datasets to reveal molecular patterns and disease mechanisms that were previously hidden when data were analysed in isolation.

By combining these datasets, scientists can define more precise starting points for biologics discovery. DeFelice notes that researchers will use these insights to select targets “with stronger biological rationale and a clearer therapeutic pathway based on disease association and molecular verification.” This, she says, will reduce the number of programmes that stall during preclinical development. Earlier confidence in target biology also allows teams to focus resources on candidates with a clearer line of sight to mechanism, modality selection and downstream development decisions, rather than revisiting foundational assumptions later in the pipeline.

Biological modelling moves into everyday workflows

Alongside AI-guided target identification, biological modelling is set to become foundational in early discovery rather than a specialist activity. DeFelice points to the growing availability of structure prediction and binding simulation tools embedded directly within AI-native laboratory systems and digital lab notebooks.

By 2026, scientists working on complex biologic modalities such as multispecific antibodies and fusion proteins will routinely evaluate affinity and specificity computationally before committing resources to experimental work. Making modelling part of daily scientific practice reduces trial and error and improves candidate selection at earlier stages.

This closer integration of computational prediction and experimental validation is changing how discovery teams work in practice. Rather than operating in sequence, modelling and wet-lab experiments increasingly inform one another, allowing data generated in one step to guide the next. This shortens feedback cycles and helps teams move from biological insight to candidate selection more efficiently.

Genomics data reaches unprecedented scale

The growing reliance on AI in discovery is closely linked to the scale of biological data now being generated, particularly in genomics. Advances in sequencing technologies continue to accelerate data generation, but analysis remains a major bottleneck.

“In 2026, we will see stunning examples of how AI can interpret genomics data to understand complex biology,” says Neil Ward, Vice President and General Manager for EMEA at PacBio. He highlights the scale of the challenge, noting that analysing a single genome involves tens of thousands of lines of code. At population scale, genomics studies “could generate up to 15× more data than YouTube over the next decade.”

At this scale, traditional bioinformatics approaches alone are no longer sufficient. Ward points to a growing move towards natural language interfaces that allow scientists to interrogate genomics data without relying exclusively on specialist code.

“We are already seeing major AI players partnering with science firms to analyse genomics data in natural language rather than relying on specialised bioinformatics code,” he says. These collaborations are intended to make complex genomic analysis easier to use without removing the underlying analytical complexity.

Digital twins move from pilots to practice

AI is also increasingly being used in later stages of development, particularly through digital twins. According to Dr Gen Li, Founder and President at Phesi, 2026 will mark a turning point for their adoption.

“After years of experimentation, 2026 will mark the year digital twins move from pilot to practice in clinical development,” Li says. Sponsors have increasingly explored digital twins to optimise protocol design, reduce amendments and accelerate timelines, but uncertainty around regulation has limited wider use.

“That’s now changing,” Li explains. Regulators including the FDA are expanding AI frameworks and finalising risk-based guidance to support safe and effective use of these technologies in clinical development. As regulatory clarity improves, opportunities to integrate digital twins into trial design and execution are expected to grow.

Li emphasises that regulatory trust is central to adoption. “To unlock the full value of digital twins, sponsors must earn regulatory trust through rigorous validation, ethical data governance and clear documentation,” he says. Continued collaboration between regulators, sponsors and technology partners will be essential.

If implemented responsibly, digital twins offer the potential for faster, more patient-centric and more equitable clinical trials.

Towards a more integrated discovery process

Taken together, these developments point to a more integrated and computationally driven approach to drug discovery and development. AI-guided target identification, embedded biological modelling, scalable genomics analysis and digital twins are increasingly used together rather than as isolated tools. As AI becomes more widely embedded, discovery teams are moving from experimentation to routine use, with the focus now on where these tools sit in existing workflows and how their outputs inform early scientific decisions.

An international research group led by scientists at Pompeu Fabra University has reported new discoveries that help to better understand the nanomachine that controls a process known as constitutive exocytosis, which is the uninterrupted delivery of spherical molecular packages to and fusion with the cell membrane. This is an essential activity present in virtually all organisms to preserve cell fitness and other vital functions such as communication with the cell’s exterior, cell growth and division.

Studying the yeast Saccharomyces cerevisiae the scientists resolved the dynamic architecture of the tiny machine that delivers essential molecular packages to the cell surface. Discovery of this flexible and transient ‘nanocourier’ required the combined power of multiple microscopes and artificial intelligence, yielding unprecedented information of a key process that occurs billions of times per day in our bodies.

Better understanding of exocytosis may have profound implications for the treatment of some infections and rare diseases. Research lead Oriol Gallego, PhD, leader of the Biophysics in Cell Biology Group at the UPF Department of Medicine and Life Sciences (MELIS), said, “despite being one of the largest nanomachines in the cell, its short lifespan and dynamism made it very challenging to capture.”

Gallego and colleagues reported on their findings in Cell, in a paper titled “Continuum architecture dynamics of vesicle tethering in exocytosis.”

“Constitutive exocytosis (hereafter exocytosis), the uninterrupted transport of secretory vesicles to and subsequent fusion with the plasma membrane (PM), is an essential cellular process for nearly all eukaryotes,” the team wrote. “Exocytosis is critical for the preservation of PM homeostasis, cell growth, and cell division.”

Every day, every cell of our body transports between 10,000–100,000 of these spherical packages to the cell surface to fulfill cellular processes that require the release or display of any molecule on the outside of the cell, such as the secretion of enzymes and hormones, repairing wounds on the cell surface or simply because the cell needs to grow, move or change its shape. Therefore, the delivery of packages to the surface is essential because it is linked to many vital processes that the cell undergoes daily. Central to this intricate process is tethering, which is the finely tuned docking of cargo-loaded vesicles with the plasma membrane, the team further explained.

Despite being vital for the cell, studying exocytosis hasn’t previously been possible in detail. Gallego’s lab, in collaboration with Carlo Manzo, PhD, at the Universitat de Vic, Daniel Castaño, PhD, at the Instituto Biofisika, and Jonas Ries, PhD, at Max Perutz Labs, combined advanced light and electron microscopes with image analysis using artificial intelligence, to resolve the 3D organization of this nanomachine, and filmed how it quickly changes its structure during the delivery of spherical packages. Gallego added, “The function of this nanocourier is so important that it is very rare to find it mutated in patients as its alteration would normally impair the viability of the embryo.”

At the core of this nanomachine, the concerted motion of seven protein assemblies known as exocysts builds a flexible ring that holds the spherical packages in place upon their arrival at their destination: the cell surface. “The exocyst, a conserved heterooctameric protein complex, is the main component of tethering,” the authors noted. “We found that seven exocysts form a flexible ring-shaped ExHOS that tethers vesicles at <45 nm from the PM.”

Co-senior Marta Puig-Tintó, PhD, one of the main authors of the study, further explained, “We have named this nanocourier ExHOS, standing for exocyst higher-order structure. The ExHOS features three checkpoints and a mechanism of disassembly that ensures that the delivery of molecular packages continues at the required speed.”

Co-senior author Sasha Meek, PhD, added, “It is as if every time the cell needs to deliver a heavy package, a team of seven strong couriers work together to do so. Because the package is so heavy, they can’t just drop it all at once and have to lower it in three steps. And when they’re finished, they need confirmation of receipt so that the team of couriers can break up and go on to make other deliveries.”

Increasing what is understood about exocytosis goes far beyond the mere desire to know, and could one day affect many fields of applied science. Plants, for example, need the ExHOS to defend cells against microbial invasion. Hence, many phytopathogens have developed mechanisms to attenuate plant immunity by attacking the ExHOS. A good example is Magnaporthe oryzae, also known as rice blast fungus, which causes the loss of up to a third of the world’s rice production.

In humans, several viruses such as SARS-CoV-2, HIV, or pathogenic bacteria, such as Salmonella, behave similarly and hijack exocytosis during infection. “Beyond its canonical role in exocytosis, the exocyst is involved in the secretion of exosomes and herpesviruses,” the researchers stated. “Not limited to secretory processes, it is a key player in autophagy, host invasion by pathogens (e.g., Salmonella typhimurium), and it is both a pathogen target and an immune receptor in plants. Together, these roles highlight the exocyst’s central importance across diverse biological pathways in both biomedical and agricultural contexts.

Even mild alterations of ExHOS components are linked to human diseases. Though infrequent, mutations in components of the nanocourier cause rare diseases related to neurodevelopmental disorders. In other cases the ExHOS participates in cell invasion in metastatic cancers.

“Despite being small in size, the cell interior is a vast space full of enigmatic nanomachines that have never been observed because of the limitations of current microscopy tools,” Marta Puig-Tintó commented. ”But I think that the future lies in integrating various imaging technologies with the power of new computational tools like AI to “make the invisible visible.”

In their report the authors concluded, “Overall, this study provides quantitative insights into the biophysical principles that drive tethering of secretory vesicles. Given the exocyst’s central role across multiple cellular processes and diseases, these insights could help advance our understanding of the exocyst’s mechanism of action across biology more generally.”

Gallego added, “With these new opportunities, we have unveiled a fundamental and vital cellular process. It’s like explaining how oxygen is exchanged during breathing or how the periodicity of the heartbeat is maintained. It might not have an immediate application, but the discovery of this nanomachine will facilitate future research to find solutions to severe biomedical and biotechnological problems.”

Incoming PhRMA Chair Paul Hudson, a day before the White House announcement, pledged to work with the administration as the president turns to insurers as a source of cost savings for prescription medicines.

President Donald Trump unveiled “The Great Healthcare Plan” on Thursday, calling on Congress to enact changes to lower drug prices and insurance premiums and stretch Most Favored Nation drug pricing beyond the 17 pharma companies that were targeted last year.

Speaking with reporters on Wednesday in San Francisco, Sanofi CEO—and PhRMA’s next board chair—Paul Hudson, had pledged to work with the administration on any drug pricing action that targeted insurers. Trump’s actions seem to confirm what Hudson and other leaders suspected would be next from the administration on the drug pricing issue.

The plan outline calls on Congress to codify the Most Favored Nation deal, although details were not provided. On lowering insurance premiums, the plan suggests that money will be sent directly to Americans while insurance companies will be required to disclose rate and coverage comparisons in “Plain English.”

“Instead of putting the needs of big corporations and special interests first our plan finally puts you first and puts more money in your pocket,” Trump said in a statement. “The government is going to pay the money directly to you. It goes to you, and then you take the money and buy your own healthcare… the big insurance companies lose and the people of our country win.”

The day before the announcement, Hudson stressed that the industry wants to be involved in advising on future drug pricing policy, preferably through organizations like PhRMA that can represent the interests of the entire industry.

“I did say this to some of [Trump’s] advisors, when we were in the White House, that I prefer for them to deal with PhRMA, because there’s a large group of companies that don’t have a deal exactly, and what are they left with?” Hudson said.

Over the fourth quarter of 2025, a handful of companies—kicked off by Pfizer—signed individual deals with the Trump administration to lower the cost of certain therapies in their portfolios. The White House also proposed TrumpRx, a direct-to-consumer platform that promises to offer the lowest prescription drug prices in America. The site has yet to launch.

Hudson suspected that there may be a “catch all” policy effort to come that would collect the remaining drug companies that have not signed deals.

Whether The Great Healthcare Plan will do that is unclear. The provisions will require codification from a divided Congress.

Hudson says his goal as incoming chair of PhRMA is to make sure that Most Favored Nation drug pricing negotiations come to an end.

“The things I’m interested in as incoming chair is trying to make sure that this round of MFN is the round, and that we’re vigilant enough to know that we we’ve all paid a price, and hopefully that will be the conclusion.”