BOSTON—The ongoing integration of AI and automation was a central theme at this year’s convening of the Society for Lab Automation and Screening (SLAS) in Boston. And that was evident from several talks, exhibits, and product announcements made at the meeting. One such announcement came from Atinary, a company that claims to have coined the phrase “self-driving labs” and aims to solve scientists’ reproducibility problems. As Atinary co-founder and CEO, Hermann Tribukait, PhD, explained during a press conference, “we augment human [scientists] with these tools to address key bottlenecks that are limiting innovation and progress.”

Atinary announced a new AI-powered laboratory based in Boston that houses two autonomous platforms called Scientific Discovery Factories. These factories integrate the company’s no-code AI platform with robotics and lab instrumentation, and they are built to continuously design, execute, analyze, and learn from real-world experiments. It is a closed loop where AI-designed experiments are physically tested and validated in the lab using robots. Data from the validated experiments is then automatically fed back into Atinary’s machine learning algorithms and foundation models which use the information to inform the next phase of experiments.

During the talk, Tribukait, shared some case studies that demonstrated the value of the company’s technology including projects with the Massachusetts Institute of Technology (MIT) as well as the chemical company DSM-Firmenich. In the MIT project, Atinary worked with researchers to identify an optimal catalyst for converting CO2 into fuels1,000 times faster than conventional methods. Meanwhile, their collaboration with DSM-Firmenich, which focused on optimizing a hydroformylation reaction, reduced the company’s manufacturing costs by 97%.

SPR for small molecule drug discovery

Also at the meeting, Carterra launched a new platform for label-free biomolecular screening and characterization. Dubbed the Carterra VegaTM High-Throughput Surface Plasmon Resonance (HT-SPR) instrument, the company claims that it is the first 48-channel SPR platform, delivery 12-fold higher throughput than competing platforms for small molecule discovery and development workflows. Commenting on the release at the meeting, Tim Germann, Carterra’s chief commercial officer, noted that Vega builds on the success of the company’s Lodestar Array platform.

That device, which was designed for large molecules, “allowed for the characterization of those molecular interactions much earlier in the process and at much greater scale,” he explained. “Where historically you could look at two or four or eight antibodies interacting with their target at a time. This entered the market allowing you … to analyze the characterization of over a thousand antibodies. And so the prospect of characterizing an entire antibody library of antibodies with that very rich SPR data became possible.”

Vega is designed to provide the same capability for small molecules. Specs about the instrument provided at the meeting state that Vega’s software can screen more than 20,000 small molecular interactions per day compared to a few thousand in the same time frame with some current SPR systems. Additionally, the Vega flow cell format includes an internal reference and two binding locations per channel making it possible to run a broad range of label-free SPR applications. It also includes an optional robotic module that when deployed lets the system operate unattended for long periods.

Vega enables “compression” of the drug discovery timeline, Germann explained. Rather than testing subsets of compounds, “testing [an] entire library is now possible” while collecting “very rich SPR binding information” and data on off-target effects, all at the same time. “These tasks, which would once have taken months and weeks of the early discovery process” can now be completed much faster. Reducing weeks and months in early discovery saves millions. But more importantly, avoiding bad choices prior to clinical development increases that likelihood of success in the clinic.

Germann said that the first Vega will ship in the Q1 of this year and then Carterra will ramp up production to meet the needs of its customers in the succeeding quarters. Vega instruments are shipped in large crates and Carterra also provides a wheeled bench for customers’ systems.

Biodegradable labware

When thinking of labware, one image that readily springs to mind, among others, are rows of colorful polypropylene boxes housing pipette tips and sample tubes. PulpFixin, a materials science company, is working to change that by offering paper alternatives to some single use plastic products. At SLAS, the company debuted various automation-ready, compostable lab products for use in liquid handling, sample identification, and cold storage workflows.

The company officially launched two offerings: its AutoSleeveTM tube adapters and its 2D AutoBoxTM sample storage system. The AutoSleeve adapters enable labs assign unique serialized 1D and 2D barcodes to standard 1.5- to 2.0-milliliter conical bottom microcentrifuge tubes. Meanwhile the AutoBox is designed for storing standard microplate freezer racks and automated barcode reading. The company’s portfolio includes a paper-based automation pipet tip rack engineered for use with Agilent’s automated liquid handlers.

The rack, which is manufactured entirely from paper, is a direct replacement for traditional plastic racks and does not require modifications to the liquid handlers to work. Racks are sealed with FeatureFilmTM, the company’s proprietary coating that provides moisture resistance and durability for cold storage conditions, including liquid nitrogen environments without shedding or contaminating samples. PulpFixin also has offers compostable alternative to Styrofoam that it claims can be used for cold chain shipping.

Concerns about sustainability are certainly top of mind across biotech and biopharma as each year millions of single use plastic components are discarded contributing to lab waste streams and environmental footprints. In a meeting with members of the press, Chad Jenkins, PulpFixin’s CEO, noted that in addition to being better for the environment, his company’s solutions are also more cost effective than the plastic products they replace which removes a potential barrier to adoption.

And the numbers seem to support the company’s cost-effectiveness claims. According to Jenkins, the OEM price per case for filter tips stands at about $849 compared to $659 for PulpFixin’s paper alternative. “The key here is that sustainability is the added benefit, not the added cost.”

PulpFixin is also relying on data to convince the scientific community that its products can withstand the rigors of lab work. Jenkins shared third-party validation results that showed that the company’s racks were DNase and RNase free and could withstand 11 kilograms of force per tip in an automated system. The products also showed no particulate shedding under 600x magnification. These data points are crucial as Jenkins admitted that a major barrier to adoption of PulpFixin’s products is fear. “How can I replace something I’ve been using forever that I know works?” In response, “we have to come back at that with data [that] say[s] yes, it does work. And that’s what we’ve been working on.”

Besides working with Agilent instruments, the company is also developing paper labware options for instruments from companies like Tecan and Hamilton. PulpFixin’s products typically take six to eight months to degrade, and the company is evaluating whether there is any potential harm to the environment from that process.

Detecting protein interactions in living cells

To help scientists seeking assays for studying particularly challenging proteins, Promega launched the TarSeerTM BRETSATM Target Engagement system. This assay is designed specifically to detect ligand-protein interactions in intact cells using protein denaturation.

In a talk at SLAS during which he presented the assay, Matt Robers, PhD, associate director of R&D at Promega, highlighted how the new assay addressed the limitations of an earlier bioluminescence resonance energy transfer (BRET)-based shift assay that the company developed.

That assay requires the use of chemical tracers which excludes proteins in the human proteome that lack high-affinity ligands or well-characterized binding pockets, he explained. That led Promega to explore other methods that could make it possible to target these so-called “undruggable proteins.”

“Thermal shift methods have always been really intriguing for me in this space because you can apply them typically without having to know a lot about your target protein,” Robers said during his talk. “You don’t need to know much about its function, and you can use thermal shift to query binding.” Another “really powerful method … is differential scanning telemetry or DSF” which uses “denaturation sensitive dyes that can kind of decorate the protein as it unfolds.” The BRETSA assay combines principles from thermal shift assays with BRET’s proximity-based energy transfer. Furthermore, instead of a tracer, the assay uses a denaturation sensitive dye combined with a NanoLuc target protein.

Robers described how Promega validated the assay’s quantitative accuracy against the established tracer-based assay using HDAC inhibitors, noting the strong correlation in results. In fact, “the fingerprint of the two assays was almost identical,” he said. And this was proved across multiple protein-ligand pairs throughout the cell. “We’re up to the point now where we’ve shown that we can use this method to measure small molecule engagement to about 130 protein ligand pairings all in isothermal mode” and “there is no cellular compartment that is off limits.”

Biologists often say that structure determines function. In a new study, Northwestern University researchers show that this principle applies not only to proteins but may also to cancer vaccines—where the nanoscale arrangement of a single peptide can dramatically alter therapeutic potency. By re‑engineering the orientation of an HPV‑derived antigen on a spherical nucleic acid (SNA) platform, the team created a vaccine that slowed tumor growth and extended survival in preclinical models of HPV‑driven cancer.

The work, published in Science Advances and titled “E711‑19 Placement and Orientation Dictate CD8⁺ T Cell Response in Structurally Defined Spherical Nucleic Acid Vaccines,” explores how subtle architectural changes can reshape immune activation. HPV‑positive head and neck cancers, which are rising in incidence, often present at advanced stages and are treated with toxic regimens, according to the authors. While prophylactic HPV vaccines prevent infection, they do not help patients with established tumors—leaving a need for therapeutic strategies that can safely drive strong cytotoxic T‑cell responses.

To address this, the researchers designed three SNA‑based vaccines containing identical components: a lipid core, CpG adjuvant, and a short HPV16 E711‑19 peptide. The only difference was how the antigen was displayed. One formulation buried the peptide inside the nanoparticle, while two others attached it to the surface through either the N‑terminus or C‑terminus—a small structural shift with potentially large immunological consequences.

The differences were striking. All three SNAs enhanced dendritic cell activation and CD8⁺ T‑cell cytotoxicity compared to a simple peptide‑adjuvant mixture, but the N‑terminally displayed version, dubbed N‑HSNA, consistently outperformed the others. It induced roughly eight‑fold higher interferon‑γ secretion and about 2.5‑fold greater cytotoxicity in primary human cells, the authors wrote.

In tumor‑bearing AAD mice, the N‑HSNA formulation cut tumor burden by more than threefold and extended survival, while also driving a noticeable expansion of CD8⁺ T cells. The same design performed strongly in patient‑derived HPV‑positive head and neck cancer spheroids, where it produced roughly a 2.5‑fold increase in tumor‑cell killing—a sign that the structural tuning may translate across model systems.

The findings highlight the emerging field of “structural nanomedicine,” which emphasizes the deliberate arrangement of vaccine components rather than simply mixing them together—what lead author Chad A. Mirkin, PhD, calls the “blender approach.”

“There are thousands of variables in the large, complex medicines that define vaccines,” said Mirkin, the George B. Rathmann Professor at Northwestern. “The promise of structural nanomedicine is being able to identify the configurations that lead to the greatest efficacy and least toxicity. In other words, we can build better medicines from the bottom up.”

By demonstrating that antigen placement and orientation can dictate immune potency, the study provides a blueprint for revisiting past cancer vaccines that failed not because of their ingredients, but perhaps because of their architecture. The authors suggest that rational design—potentially aided by machine learning—could accelerate the development of more effective therapeutic vaccines for HPV‑driven tumors and beyond.

Moderna will not commit to previous 2028 breakeven guidance as the ripple effects of the FDA’s refusal-to-file decision spread through its pipeline.

Moderna executives didn’t mention the word “breakeven” while reporting fourth quarter earnings—until analysts asked. Having previously said the company would reach that key milestone in 2028, Moderna’s management now admits that the plan has been blown wide open by the FDA’s decision to not even look at an application for the influenza vaccine mRNA-1010.

So will the company make it after a potential $1 billion revenue opportunity has been potentially scuppered? CFO Jamey Mock said there are just too many unknowns at this point, just five days after the FDA’s surprise decision landed with a thud on Moderna’s desk.

“I recognize that it’s on investors’ minds,” Mock started off, speaking on the company’s earnings call Friday morning. “This is a bit of a fresh and fluid situation, and without understanding the resolution of what is next for our flu product, it’s a little bit difficult to comment at this time.”

Without restating the breakeven goal, Mock said that Moderna is expecting 10% topline revenue growth for 2026 thanks to vaccine contracts outside of the U.S.

Mock also pointed to 10 upcoming products in Moderna’s pipeline that are expected to start contributing revenue in the next few years. “We have 10 large shots on goal to increase revenue over the coming years, all with a wide range of potential outcomes,” he said.

But Moderna has to contend with the FDA, which has been showing hostility toward the company’s key mRNA technology and vaccines. Later on the call, an analyst probed Moderna executives for whether the same office that rejected mRNA-1010 will be in charge of the application for personalized cancer vaccine intismeran autogene (INT), which is being developed with Merck.

Moderna President Stephen Hoge, who leads the R&D organization, confirmed that yes, the FDA’s Center for Biologics Evaluation and Research would be in charge of the application for INT. CBER chief Vinay Prasad personally signed off on the refusal-to-file letter regarding mRNA-1010, reportedly overruling other regulators.

Hoge said that while CBER would certainly be the lead office, others would be involved too. “It is an oncology therapy of high import. It gets a lot of attention,” Hoge said.

He stressed that conversations have been going well so far with “highly productive engagement.”

Merck is heavily involved in INT’s development, according to the split laid out in the original licensing deal. The Big Pharma is running the Phase 3 trial for the therapy in melanoma, and a biologics license agreement, “if it goes forward,” will be Merck’s responsibility. Hoge gave no indication that they will change any other leadership aspect of the regulatory process.

The readout for the Phase 3 INTERPATH-001 trial in melanoma is due this year. Other trials in the partnership include Phase 2 tests for adjuvant renal cell carcinoma and adjuvant muscle-invasive bladder cancer.

Moderna does intend to refile the mRNA-1010 vaccine in the U.S., pending feedback from the FDA, CEO Stéphane Bancel said on the call. But for now, the company is eyeing other markets like Europe, Canada and Australia for approvals.

Bancel only delivered prepared remarks during the call and left the analyst questions to his deputies. But he reiterated his disappointment in the FDA’s RTF decision.

“The current uncertainty in the U.S. FDA regulatory environment creates real challenges for businesses, patients and the broader innovation ecosystem,” Bancel said during his opening statement. “When expectations and review timelines are unpredictable, companies face greater risk and can hesitate to invest, slowing the development of breakthrough medicines.”

Bancel then said that this roadblock will mean that companies like Moderna will turn elsewhere for regulatory filings, leaving the U.S. without the next generation of breakthrough medicines.

Going Global

CBER’s decision also imperiled Moderna’s combo flu-COVID vaccine mRNA-1083. The company had been waiting for the flu shot mRNA-1010 to advance in the regulatory process before filing the combo.

While they wait for regulatory clarity from the FDA—a Type A meeting should be made available within 30 days of the rejection—executives say the company will move on with applications for both shots in other parts of the world.

Already mRNA-1083 has been submitted in Europe and Canada, while mRNA-1010 has been submitted in Europe, Canada and Australia. Approvals for the latter could start rolling in by the end of the year. Hoge said that revenue from the products could begin to show up from international markets for the 2027–2028 seasonal flu season.

Moderna’s original COVID-19 vaccine has never had access to the European markets because Pfizer and BioNTech locked down a long-term contract for their joint shot Comirnaty. That contract expires this year, according to Hoge. Moderna plans to move the second-generation COVID vaccine mNEXSPIKE into Europe. That product, along with the flu and combo, will help Moderna tap into a respiratory disease market worth up to $1.8 billion, the R&D chief said.

More Losses

Regulatory trouble notwithstanding, Moderna had a fairly productive quarter, given the company’s challenges over the past few years. The vaccine business beat analyst expectations with $645 million, compared to the $618 million consensus. This was primarily thanks to COVID shots Spikevax and mNEXSPIKE.

Revenue for the full year, which had been pre-announced, was $1.94 billion, or $100 million above the midpoint of the company’s original 2025 guidance, which was $1.6 billion to $2 billion.

Reflecting the beats, Moderna’s shares, which had been battered by the FDA action earlier in the week, rose nearly 4% to $44.03 on Friday morning, erasing the previous losses.

But Moderna is still losing significant amounts of money and has been for five straight quarters. The company lost $826 million for the fourth quarter, which was better than the consensus of a $1.02 billion loss. For the full year, Moderna lost a staggering $2.8 billion.

To help stem the ongoing losses, Moderna has been undergoing an aggressive cost-cutting campaign. Bancel said that AI has “touched every part of our business” to find efficiencies, and new ones are expected to be revealed as that work continues.

Vinay Prasad, the FDA’s Center for Biologics Evaluation and Research head, is accused of interpersonal impropriety as pushback builds against his decision to reject Moderna’s influenza vaccine candidate.

Vinay Prasad is drawing heat for his personal conduct as director of the FDA’s Center for Biologics Evaluation and Research, as scrutiny grows around his decision not to review Moderna’s application for influenza vaccine candidate mRNA-1010.

Prasad is the subject of a number of internal complaints at the FDA, including “sexual harassment, retaliation against subordinates and verbally berating staff,” according to reporting from The Wall Street Journal on Wednesday, citing unnamed sources familiar with the matter.

Formerly a professor at the University of California, San Francisco, Prasad continues to live in the San Francisco Bay area and has commuted for the past year to work at the FDA in Maryland every two weeks, at a cost to taxpayers of about $65,000 per year, according to an estimate viewed by WSJ.

Prasad is also facing pushback internally over his decision to overrule FDA staffers and return a refusal-to-file (RTF) letter to Moderna on Tuesday.

In a January meeting with Prasad, FDA staff argued that a plan to block the application was the wrong approach. A review team was already assembled to review Moderna’s application, according to reporting from STAT News, and David Kaslow, director of the Office of Vaccines Research and Review within CBER, wrote an internal memo arguing against Prasad’s decision. Some staffers at the January meeting specifically pushed back against Prasad’s criticism of Moderna’s trial design, which he said “does not reflect the best-available standard of care.”

There was a “a diverse set of conclusions among the review team” looking at Moderna’s application, Health and Human Services spokesperson Andrew Nixon said in a statement emailed to Fierce Biotech.

Moderna, of course, balked at the squabble over the control arm of its study. “It should not be controversial to conduct a comprehensive review of a flu vaccine submission that uses an FDA-approved vaccine as a comparator in a study that was discussed and agreed on with CBER prior to starting,” Moderna CEO Stéphane Bancel said in a statement on Tuesday in response to the RTF.

Before Prasad took the lead at CBER in May 2025, staff at the agency had agreed to Moderna’s study designs, according to Moderna’s press release. The agency also did not raise objections when a Phase 3 study for mRNA-1010 launched in September 2024, the company noted.

Then in August 2025, a few months after Prasad joined the FDA, Moderna again met with the agency, at which point CBER staff requested additional analyses. Moderna complied, providing data from a separate Phase 3 trial that compared mRNA-1010 against an approved high-dose influenza vaccine.

“At no time in the pre-submission written feedback or meeting did CBER indicate that it would refuse to review the file,” Moderna wrote in its statement.

While Kaslow and other staffers appeared to have been ready to begin their review of mRNA-1010, Prasad apparently had other plans, overruling his team to send the RTF.

Moderna will share its fourth quarter and full-year 2025 earnings Friday morning, where analysts will likely eagerly await details about Moderna’s plans for mRNA-1010, as well as for mRNA-1083, the company’s combination influenza/COVID-19 vaccine. Last year, Moderna pulled a biologics license application for mRNA-1083 until more data from mRNA-1010 came in, on the assumption that that shot’s progress would improve mRNA-1083’s chances. The RTF for mRNA-1010 now might have the knock-on effect of disrupting mRNA-1083’s future as well.

Moderna’s mRNA-1010 was expected to contribute $1 billion to the company’s coffers by 2028. That plan is now out the window after the FDA refused to even look at the application.

Moderna’s plan to reach breakeven status by 2028 has been imperiled by the FDA’s surprise rejection of the flu vaccine mRNA-1010, which had been expected to contribute to billions in expected revenue along with the combo flu/COVID vaccine mRNA-1083.

“In our view. U.S. sales of these products were expected to be meaningful contributors to 2027 and 2028 revenue,” William Blair wrote in a note to investors Wednesday.

Moderna was stunned by the decision of Center for Biologics Evaluation and Research head Vinay Prasad to refuse to review the application for mRNA-1010. In an unusual step, the regulator personally signed the rejection and appears to have overruled others at the agency who felt the application should be accepted for review.

Prasad, in his letter to Moderna, said that the control vaccines used for the clinical data in the mRNA-1010 package “does not reflect the best-available standard of care.” Health and Human Services spokesperson Andrew Nixon backed Prasad up, saying that Moderna “refused to follow very clear FDA guidance from 2024 to test its product in a clinical trial against a CDC-recommended flu vaccine to compare safety and efficacy.” Moderna maintains that the FDA agreed to its use of already-approved flu vaccines as comparators.

On Wednesday, Moderna’s stock fell 9% after news broke of the refusal-to-file letter and by Thursday morning was trading at $40.08.

Without a near term regulatory path for the flu shot, Moderna may have to find a new path to profitability.

Vaccine Uncertainty

Moderna’s revenues soared in the early years of the COVID-19 pandemic, peaking at $4.9 billion in the fourth quarter of 2021, as the world scrambled to get the company’s mRNA-based vaccine. But as vaccinations rates have dwindled in the U.S. over the past few years, Moderna’s earnings have too. The company has consistently been in the red for the last few quarters, but, through aggressive cost cutting and the addition of new products to the portfolio, Moderna had planned to be back in the black by 2028.

In its 2025 third quarter earnings, Moderna reported a decline of $200 million, an improvement over $800 million lost in the second quarter of the year. MRNA-1010, the company’s next-generation influenza vaccine, was supposed to be a big part of the plan to return to profitability, along with mRNA-1083.

Then came the startling news earlier this week, with Prasad overruling agency staff in denying Moderna the opportunity of even a review for mRNA-1010.

William Blair sounded a troubled note, saying that after the news of the refusal they were reworking their models of Moderna’s future potential earnings on the fly. “This is a substantial hit to the probability of success for mRNA-1010, and in turn mRNA-1083’s (combo flu/COVID vaccine) U.S. approvability,” they wrote in a note to investors on Wednesday.

In the event that the FDA requires a new study, it’s unclear what Moderna will do, as the company last month elected to stop running new vaccine trials, at least for infectious disease. “You cannot make a return on investment if you don’t have access to the U.S. market,” CEO Stéphane Bancel said at the World Economic Forum.

Return to Profitability

Analysts at Jefferies had expected both mRNA-1010 and mRNA-1083, once approved, to each hit sales of $1 billion per year at their peak, which would hypothetically cover Moderna’s losses from recent quarters and put the company back into profitability. Moderna notably paused development of mRNA-1083 last year until mRNA-1010 was approved, in the hopes that approval of the flu vaccine would strengthen the case for the combination shot.

Moderna is planning to market mRNA-1010 in other parts of the world, and the path to approval outside the U.S. seems less obstructed. “We continue to expect approval of mRNA-1010, and potentially a path forward for mRNA-1083, in ex-U.S. geographies, but premium pricing will be difficult here, which further pressures 2028 breakeven guidance,” the William Blair analysts wrote.

Jefferies analysts agreed, expecting to see approvals in Canada, the EU and Australia for both mRNA-1010 and mRNA-1083, according to a Wednesday note. And Moderna has taken the important step of diversifying its pipeline outside of just the approved COVID-19 vaccine Spikevax, with an RSV vaccine, plus pipeline assets for norovirus, a handful of treatments for cancers like solid tumors, lymphoma and melanoma, and even rare diseases like propionic acidemia and methylmalonic acidemia.

In the meantime, Moderna’s 2026 outlook remains the same as before the FDA rejection, since the vaccines were not expected to bring revenues until 2027 and 2028, according to Jefferies. “But we’d imagine it could impact management’s 2028 cash breakeven guidance (still achievable though)—as we viewed mRNA-1010 (and secondarily mRNA-1083) as a core growth driver starting in 2027-28.”

Moderna will report fourth quarter and full-year 2025 earnings on Friday morning, where the FDA’s actions will likely be the headline.

After receiving a letter directly from CBER Director Vinay Prasad, Moderna said the FDA had previously signed off on the use of a licensed flu vaccine as a comparator for a Phase 3 study of mRNA-1010.

The FDA has declined to even consider Moderna’s application for the investigational mRNA flu vaccine mRNA-1010, a move that comes amid controversial immunization policies from the Trump administration.

The refusal-to-file letter (RTF) was signed by Center for Biologics Evaluation and Research (CBER) Director Vinay Prasad himself, who has been vocal about tightening regulations on vaccines. In the RTF, the regulator claimed that Moderna failed to support mRNA-1010’s application with an “adequate and well-controlled” trial, according to the company’s Tuesday news release. Specifically, Moderna’s comparator group “does not reflect the best-available standard of care,” Prasad said in the letter, which Moderna published in full on its website.

In a statement on Monday, CEO Stéphane Bancel hit back at the FDA’s decision to refuse a review: “This decision by CBER, which did not identify any safety or efficacy concerns with our product, does not further our shared goal of enhancing America’s leadership in developing innovative medicines.”

William Blair analysts said the rebuff represents a major blow to the already struggling vaccine maker’s financial goals. “The RTF letter for mRNA-1010 represents a big hit to the company’s vaccine franchise and its prospect of achieving its breakeven guidance in 2028,” the analysts wrote in a Wednesday morning note.

Moderna’s shares were down 10% $37.76 in premarket trading Wednesday morning.

And it’s not just mRNA-1010 that could be affected by the FDA’s decision, with analysts at Jefferies noting that the RTF could impact the timing of a potential U.S. refiling of mRNA-1083, Moderna’s combination shot for COVID-19 and flu.

In May 2025, the company pulled its approval application for mRNA-1083 and decided to wait for data from mRNA-1010 before resubmitting. The company, Jefferies said on Tuesday, was “planning to seek [additional] FDA guidance” after the mRNA-1010 submission. But after the RTF, the path forward could change, Jefferies speculated in a note to investors.

William Blair agreed. “We will know more after the Type A meeting minutes are issued, but this is a substantial hit to the probability of success for mRNA-1010, and in turn mRNA-1083’s (combo flu/COVID vaccine) U.S. approvability, in our view.”

Moderna backed its application for mRNA-1010 with several late-stage studies, the company said last month. One of which, announced in June 2025, compared the investigational shot against a currently licensed, standard-dose seasonal flu vaccine. Data showed that Moderna’s candidate was 26.6% more effective than the comparator vaccine at preventing flu in adults 50 years and up.

The company pointed out in its news release that the FDA’s own regulation on the use of adequate and well-controlled studies, as well as guidance for flu vaccine development, do not “contain any reference to the use of a comparator reflecting the ‘best-available standard of care.’”

Moderna likewise argued that the refusal “is inconsistent with previous written communications” with CBER, which had found a standard-dose flu shot to be an “acceptable” comparator. “It should not be controversial to conduct a comprehensive review of a flu vaccine that uses an FDA-approved vaccine as a comparator in a study that was discussed and agreed on with CBER prior to starting,” Bancel said in his statement.

The Department of Health and Human Services (HHS) announced in May 2025 that all new vaccines would require placebo-controlled trials, a move that experts widely questioned. “HHS’s judgment appears questionable and risky, in our view,” Leerink Partners wrote at the time, adding that “placebo-controlled trials are unnecessary and unethical for many populations.”

In a statement to STAT News, a spokesperson for the Department of Health and Human Services said that “the FDA generally does not comment on regulatory communications to individual sponsors.”

Tuesday’s rebuff to Moderna compounds the already-mounting worries that vaccine developers face in the U.S., fueled largely by controversial policies under the Trump administration. In addition to the new placebo requirement, Prasad suggested the agency would enact stricter regulations for COVID-19 vaccines, after falsely claiming that at least 10 children had died from the shots.

Over at the CDC, recommendations for vaccine use have also been shifting. Last month, the agency unilaterally scrapped the recommendation that children be immunized against flu and five other diseases. In December 2025, the CDC also pushed back infant hepatitis B vaccination from being given at birth to two months of age.

As a result of these policy headwinds, Moderna announced late last month that it will no longer invest in Phase 3 vaccine studies—though it remains unclear if this directive will only affect its infectious disease programs or will apply broadly to its entire vaccine portfolio.

“You cannot make a return on investment if you don’t have access to the U.S. market,” Bancel told Bloomberg at the time.

A recurring theme Tuesday morning at Phacilitate’s Advanced Therapies Week was the quickly emerging potential of in vivo approaches to cell and gene therapy—a trend also reflected in recent investments by Eli Lilly and Regeneron.

In vivo cell and gene therapy is en vogue at Phacilitate’s Advanced Therapies Week in San Diego this week, a theme that reflects a recent rush of activity in the space, including Eli Lilly’s $2.4 billion acquisition of Orna Therapeutics on Monday.

“In vivo is the new allogeneic,” Susan Nichols, CEO of Propel Bio, a commercialization acceleration partner, said during Tuesday’s kickoff session, which convened Phacilitate’s advisory board to discuss 2025: What Worked. What Didn’t. What Comes Next?

While ex vivo cell therapy involves extracting patient cells, modifying or engineering them outside the body and then reinfusing them into the patient, in vivo approaches deliver the therapeutic payload directly into the patient’s body, bypassing this step.

In vivo cell and gene therapies have “huge promise [and] heavy investment,” Nichols continued, though she added that human data is limited for now. “As one advisor put it, in vivo approaches are full of promise, but the investment momentum is outpacing the clinical data.”

While in vivo cell and gene therapy (CGT) approaches might be early-stage, Big Pharma—including obesity darling Eli Lilly—is buying in. In the past, issues around accessibility had limited Lilly’s interest in the modality.

“The data is amazing,” Lilly CEO David Ricks told Reuters in 2019, “but practically, it’s not reaching many people.”

Lilly appears to believe that Orna’s technology, which uses circular RNA paired with novel lipid nanoparticles to enable a patient’s own body to generate cell therapies, can solve this challenge.

“We look forward to working with Orna colleagues to potentially unlock an entirely new class of genetic medicines and cell therapies for patients who today have limited or no treatment options,” Francisco Ramírez-Valle, head of Immunology Research and Early Clinical Development at Lilly, said in a statement.

Negating the Need for Chemotherapy

Victoria Gray, a patient advocate who was the first person with sickle cell disease (SCD) to be treated with Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene editing treatment Casgevy, told BioSpace that in vivo gene therapy could be available to more people because it negates the need for immunosuppressive chemotherapy.

“I would love to see the science advance for patients like myself” who are currently required to undergo a conditioning regimen with a chemotherapeutic agent, Gray said during a patient advocacy session at Advanced Therapies Week on Tuesday. That agent, busulfan, is used to destroy the defective blood stem cells in the bone marrow to make way for the edited cells. Both Casgevy and Genetix Bio’s (formerly bluebird bio) Lyfgenia come with this caveat. “That was the hardest part for me,” Gray said.

Kristen Hege, former senior vice president of Early Clinical Development, Hematology/Oncology & Cell Therapy at Bristol Myers Squibb, emphasized the long-term impact of lymphodepletion treatment.

“When we think about in vivo gene therapy, I think a lot of people talk about patient access, cost of goods, but there’s more than that,” she said during a session titled: We’ve solved for science. What now? The chemotherapy that’s required before the infusion of ex vivo CAR T cells can cause immune suppression that can last for weeks, she continued. This can “increase the risk of infection, and we don’t really talk about it, but [can also] cause leukemia. Eliminating the need for lymphodepleting chemotherapy, if it pans out with in vivo, could be another real advantage.”

Indeed, the approach is catching on. In addition to Lilly, Regeneron also recently bought into the in vivo approach, linking up with Cambridge, Mass.–based Tessera Therapeutics late last year. The partnership is aimed at advancing the in vivo editor TSRA-196 for alpha-1 antitrypsin deficiency (AATD). Tessera is also developing a gene writer for SCD, Cell & Gene.com reported in January 2025, aiming to directly correct the mutation that causes cells to sickle through a one-time IV treatment.

But the potential advantages don’t stop with access and tolerability, according to Hege.

“Regulations are often built around what you can measure, and if you can measure it, then you’re required to measure it. Or if you can control it, then you’re required to control it,” she said. “I think that’s been one of the challenges for ex vivo manufacturing, because there’s a lot of things that you can measure and control.”

With in vivo approaches, Hege continued, “What happens in the patient is beyond your control, and therefore you’re not held to a standard” in terms of pharmacodynamic, phenotypic and gene expression endpoints as part of the product characterization.

“Belief is currently leading data, not following it,” Nichols said of in vivo treatments during her presentation.

Ultragenyx Pharmaceutical has long stood behind in vivo gene therapy. The rare disease–focused biotech presented new data last week from two trials of its novel in vivo AAV9 gene therapy UX111 at the WORLDSymposium 2026. The candidate, in development for Sanfilippo A syndrome, sustained cognitive improvements over up to eight years of follow-up, according to Ultragenyx’s Feb. 3 press release.

UX111 was rejected by the FDA in July 2025, but clinical data was not the issue. Rather, the agency wanted to see “improvements” in “certain aspects” of the therapy’s chemistry, manufacturing and controls,” Ultragenyx said in its announcement of the complete response letter.

“Regulation is no longer the gating issue,” Nichols concluded.

The newly public Evommune shared data showing that EVO301, an IL-18 targeting protein, cleared symptoms comparably to Regeneron and Sanofi’s mega-blockbuster in a mid-stage atopic dermatitis clinical trial.

Shares of Evommune, a member of the late-2025 biotech IPO class, rose 75% on Tuesday after the therapeutic protein EVO301 eased the severity of atopic dermatitis by 33% in a mid-stage trial.

Those numbers position Evommune to complete with the likes of Regeneron and Sanofi, which market one of the biggest drugs in the world for dermatitis, Dupixent, analysts at William Blair wrote on Tuesday.

Dupixent earned $17.8 billion in 2025, a 26% increase from 2024, according to Regeneron’s full-year 2025 earnings report.

Shares of Evommune climbed 75% to $29.87 in Tuesday morning trading.

In a Phase 2a trial that the company described in a Tuesday release as “proof-of-concept,” Evommune tracked 70 patients for 12 weeks after they were given an intravenous dose of EVO301 on the first day and the 28th day of the trial, both of the same dose level. There were 48 patients in the treatment arm and 22 in the placebo group.

After 12 weeks, patients in the treatment arm saw a 55% reduction in eczema severity versus 22% for patients in the placebo arm, a 33% placebo-adjusted rate.

EVO301’s results are “highly encouraging,” the William Blair analysts said, especially given that Dupixent showed a 35% to 36% placebo-adjusted improvement after 16 weeks in a Phase 3 trial, and that EVO301 was only tested at one dose level. “We believe greater efficacy could be achieved with a more optimized dosing regimen,” the analysts added.

Evommune plans to test just that in a Phase 2b dose-ranging trial using a subcutaneous formulation of EVO301. More data from the Phase 2a trial will be presented at an upcoming medical conference, according to Tuesday’s statement. Evommune is also evaluating the candidate in other indications like ulcerative colitis.

EVO301 is a fusion protein designed to bind IL-18, an inflammatory cytokine implicated in a number of immune conditions, including dermatitis.

In late October 2025, Evommune joined a sudden rush of biotechs attempting IPOs, raising $172.5 million to back its immunology and inflammation pipeline. The biotech’s lead molecule is EVO756, which blocks the MRGPRX2 receptor and is being tested for chronic spontaneous urticaria and atopic dermatitis.

The agency flagged several violations at a compounding pharmacy owned by Hims & Hers, including “infestation by rodents, birds insects, and other vermin.”

In a warning letter issued in June last year to MedisourceRx, a compounding pharmacy owned by Hims & Hers, the FDA cited clear violations of good manufacturing practices. The revelations, first reported Monday by STAT News, add to the troubles of the telehealth company, which has come under fire from both regulators and Novo Nordisk for selling compounded semaglutide.

An FDA inspector found that facilities for drug manufacturing, processing, packaging or storage “are not free of infestation by rodents, birds insects [sic], and other vermin,” according to the warning letter. The inspector also spotted a live spider in the area where MedisourceRx produced active pharmaceutical ingredients for human drugs.

Among these products, the warning letter specified, was Hims & Hers’ compounded semaglutide injection.

The FDA also flagged a dead cricket which “was observed in the middle of the incubator room.” MedisourceRx uses these incubators for fill vials as part of the manufacturing of drugs for human use.

Aside from the infestation, the FDA inspector also noted that one patient who had taken a dose of injectable compounded semaglutide experienced severe gastrointestinal issues that necessitated “three nights of hospital stay.” MedisourceRx “has not submitted an adverse event report” as required, the letter claimed.

“Patient safety and regulatory compliance are foundational to how we operate,” a spokesperson for Hims & Hers told STAT News, which reported that the FDA sent a second letter to MedisourceRx in December 2025, which has yet to be released publicly.

The spokesperson also told STAT that the company is working on addressing the FDA’s concerns about reporting of toxicities, but insisted that the issues of infestation enumerated in the June letter no longer appear in the December communique. The spokesperson did not provide STAT a copy of this latter letter.

The last few days have been rough for Hims. The company last week launched a compounded version of oral semaglutide, triggering a firestorm of backlash from regulatory authorities and Novo Nordisk. FDA Commissioner Marty Makary said in an X post that the agency will “take swift action against companies mass-marketing illegal copycat drugs.”

Mike Stuart, general counsel for the Department of Health and Human Services, also blasted Hims’ knockoff pill, saying in a Feb. 7 X post that he has referred the matter “to the Department of Justice for investigation.”

Novo has also sued Hims for allegedly infringing on key patents protecting semaglutide. John Kuckelman, the pharma’s general counsel, said that the company could seek trebled damages that could reach “hundreds of millions” of dollars.