In a CT scan, coronary artery calcium shows up as distinct, bright pixels. It looks like salt in the pepper of the heart. The more calcium, the higher a patient’s risk of a heart attack.
Often, a cardiologist looks for those bright spots on purpose: They’ll grab snapshots of the heart between beats, to get the clearest possible view of the coronary arteries. But calcium is also visible on zoomed-out chest CTs that aren’t synchronized with the heart. Every year, patients receive 19 million of those more general scans — to screen for lung cancer, or investigate a persistent cough — and an eagle-eyed radiologist can report any incidental calcium they spot.
But even as heart disease remains the top cause of death in the United States, an estimated 20% to 40% of that incidental calcium goes unreported. “We need to find more of these patients,” said Ami Bhatt, chair of the Food and Drug Administration’s Digital Health Advisory Committee and chief innovation officer of the American College of Cardiology.
One of the biggest obstacles in targeting CRISPR therapy deliveries directly into the body isn’t the editing chemistry, it’s the size of the editors themselves. The field’s workhorse nucleases, including Cas9 and Cas12a, are considerably large (exceeding 1,300 amino acids) to fit inside adeno‑associated virus (AAV) vectors, the most widely used delivery vehicle for in vivo gene therapy. That size mismatch has forced most clinical applications to rely on ex vivo editing of blood or bone‑marrow‑derived cells, leaving many tissues out of reach. A smaller CRISPR system that can be packaged into AAV without sacrificing efficiency has long been a key missing piece.
A new study published in Nature Structural & Molecular Biology takes a major step toward that goal. Researchers at the University of Texas at Austin and collaborators report the discovery and engineering of a compact Cas12f nuclease that performs robustly in human cells, a notable advance for a class of miniature enzymes that have historically shown lower efficiencies in mammalian cells compared to larger systems. The paper is titled, “Comparative characterization of Cas12f orthologs reveals mechanistic features underlying enhanced genome editing efficiency.”
The team began by mining metagenomic datasets for naturally small CRISPR enzymes and identified a previously uncharacterized ortholog, Alistipes sp. Cas12f (Al3Cas12f). Despite its compact size—roughly one‑third that of Cas9—the nuclease showed unexpectedly strong activity in human cells. In initial screens, Al3Cas12f produced more than 50% editing at many genomic sites and exceeded 90% at several targets. The authors wrote, “Results from a gRNA screen targeting intron 1 of the ALB gene, exon 3 of the APOA1 gene and the AAVS1 site within PPP1R12C intron 1 showed that 27 target sites displayed >10% editing, 19 sites displayed >50% editing and 10 sites displayed >90% editing across AAVS1 and APOA1.”
Cryo‑EM structures revealed why this miniature enzyme punches above its weight. Compared with other Cas12f orthologs, Al3Cas12f forms a more extensive and interlocking dimer interface, creating a stable, preassembled complex that supports efficient R‑loop formation. The guide RNA scaffold also appears naturally streamlined: unlike other Cas12f gRNAs, it lacks an extraneous stem‑loop and adopts a compact conformation that docks cleanly into the protein. As the authors noted, Al3Cas12f achieves “efficient R‑loop formation through a stable dimer interface and a naturally optimized gRNA.”
Using these structural insights, the team engineered an enhanced variant, Al3Cas12f RKK, that dramatically boosts editing efficiency across genomic loci. In human cells, the variant increased editing from below 10% to more than 80% at many targets, with some sites reaching 90%. The researchers tested the system in a leukemia‑derived human cell line, focusing on genes implicated in cancer, atherosclerosis, and ALS.
The mechanistic comparisons were equally revealing. By solving the structures of two additional Cas12f orthologs—Oscillibacter sp. Cas12f and Ruminiclostridiumherbifermentans Cas12f—the team noted “divergent architectures and regulatory features governing protospacer-adjacent motif recognition, gRNA binding, dimerization, and DNA cleavage.” Al3Cas12f’s extended helices and mortise‑and‑tenon‑like interactions appear to be lineage‑specific adaptations that stabilize the nuclease and support high activity.
The next step is to test whether the enzyme maintains its performance when packaged into AAV vectors. If successful, the system could offer a blueprint for engineering future generations of compact CRISPR tools.
Regeneron Pharmaceuticals plans to expand its pipeline into radiopharmaceutical therapies through an up to $4.3 billion collaboration with Telix Pharmaceuticals to co-develop and co-commercialize precision oncology treatments and companion diagnostics.
The companies have agreed to partner on next-generation radiopharmaceutical therapies aimed at up to eight solid tumor targets from Regeneron’s portfolio of antibodies, generated from VelocImmune® technology, which uses the company’s own mouse platform engineered with a genetically humanized immune system.
Regeneron and Telix also said they plan to develop radio-diagnostics designed to support patient selection and treatment response assessment.
The collaboration is intended to combine the biologics expertise of Tarrytown, NY-based Regeneron, including bispecific antibody discovery, with the radiopharmaceutical development platform, global manufacturing capabilities, and supply chain infrastructure of Telix, which is headquartered in Melbourne, Australia.
“Regeneron is excited to enter the targeted radiopharmaceuticals space and explore the utility of these agents either as monotherapy or rationally combined with our immunotherapy platform, particularly in areas of high unmet patient need such as lung cancer, where our PD-1 inhibitor is a global standard of care,” Israel Lowy, MD, PhD, Regeneron’s senior vice president and clinical development unit head, oncology, said in a statement.
Lowy referred to Libtayo® (cemiplimab-rwlc), a programmed death receptor-1 blocking antibody approved for multiple oncology indications including forms of non-small cell lung cancer (NSCLC), as well as cutaneous squamous cell carcinoma, and basal cell carcinoma. Libtayo finished 2025 with $1.453 billion in worldwide net product sales, up 19% from $1.217 billion in 2024. Figures include $425 million in Q4 2025 global net product sales, up 16% from $367 million in the year-ago quarter.
‘An ideal partner’
“In our view, the deal with Regeneron validates Telix’s differentiated capabilities in radiopharmaceutical development and handling of complex supply chain logistics,” Andy T. Hsieh, PhD, a partner and biotechnology analyst with William Blair, wrote Monday in a research note. “Furthermore, given Regeneron’s track record of developing successful commercial therapeutics, we believe it is an ideal partner in bringing forth antibody-based theranostic assets.”
Telix investors appeared to somewhat agree with that analysis. The company’s ordinary shares traded on the Australian Stock Exchange climbed nearly 8% from A$14.64 ($10.34) to A$15.77 ($11.13). Telix’s American depositary shares traded on NASDAQ rose about 7%, from $10.56 to $11.24.
Hsieh reiterated William Blair’s “Outperform” rating for Telix shares based on several potential value-creating inflection points, including:
Continuing gains in market share gains within the prostate-specific membrane antigen (PSMA) positron emission tomography (PET) diagnostic imaging market, based on rising sales and price stability as payers have offered clarity on reimbursement—factors he said enable Telix to expand its precision medicine franchise “from a position of strength.”
Therapeutic franchise potential, as supported by recent positive preliminary data from part 1 of the ongoing Phase III ProstACT GLOBAL trial (NCT06520345) assessing TLX591 in metastatic androgen pathway modulation resistant prostate cancer.
Potential approvals of two PET imaging agents—TLX250-CDx (Zircaix®, 89Zr-DFO-girentuximab), designed to non-invasively detect and characterize clear cell renal cell carcinoma (ccRCC); and TLX101-Px (Pixclara®, Floretyrosine F 18 or 18F-FET), designed to image glioma. Both could “meaningfully” contribute to Telix’s profit-and-loss statement next year, the analyst predicted.
The FDA rejected both Zircaix and Pixclara last year via separate complete response letters. The agency held in April 2025 that Zircaix required additional confirmatory clinical evidence, which the company agreed to provide. On Friday, Telix said the FDA accepted its resubmitted New Drug Application (NDA) for Pixcara, assigning a target decision date of September 12 under the Prescription Drug User Fee Act (PDUFA).
In August 2025, the FDA rejected Zircaix via complete response letter, alleging deficiencies relating to its chemistry, manufacturing, and controls (CMC) package—deficiencies the company said were “readily addressable.”
“We look forward to additional updates pertaining to efficacy parameters, such as progression-free survival, an approvable endpoint, likely later this year,” Hsieh added.
Growth through acquisitions
Telix has built up its radiopharma infrastructure in recent years through acquisitions, spending $13.6 million to purchase IsoTherapeutics, a contract development and manufacturing organization (CDMO) focused on providing services to Telix and other radiopharmaceutical companies—followed by an up to $82 million buyout of radioisotope production technology firm ARTMS, which stands for alternative radioisotope technologies for medical science.
In September 2024, Telix expanded its manufacturing footprint by acquiring RLS Radiopharmacies for up to $250 million, part of an investment strategy focused around creating vertically integrated supply chain, manufacturing, and distribution.
The global radiopharmaceuticals market is predicted to grow at a compound annual growth rate of 10.1%, more than doubling from $14.2 billion this year to $31 billion in 2032, then soaring again to $54.6 billion by 2040, according to a Roots Analysis report issued in January.
Telix briefly pursued a U.S. initial public offering, which it withdrew in June 2024. The company cited market conditions as biotech IPOs met with chilly receptions on Wall Street and asserted that the offering was not predicated on the need to raise capital.
Regeneron has agreed to pay Telix $40 million in upfront cash for access to its radiopharmaceutical manufacturing platform for four initial therapeutic programs, with Regeneron holding an option to expand the collaboration to include four additional programs with additional upfront payments.
Telix and Regeneron have agreed to share equally their global commercialization costs and potential profits, with Telix retaining the option to co-promote certain potential products. However, if Telix were instead to opt out of the co-funding model for any of the original four programs, it would then be eligible to receive up to $535 million in development and commercial milestone payments, plus low double-digit royalties on future net sales, for that program.
If Telix opts out of co-funding for all four, company could achieve $2.14 billion in payments tied to achieving milestones.
For the diagnostics to be covered by the collaboration, Telix and Regeneron have agreed to jointly develop diagnostic assets, with Telix leading commercialization and Regeneron receiving a set percentage of profits.
“The collaboration with Regeneron reflects a highly complementary set of capabilities and a unique opportunity to explore what true ‘next gen’ biologics-based radiopharmaceuticals can potentially do for patients,” added Christian Behrenbruch, DPhil, managing director and group CEO at Telix. “We are well positioned to work toward the shared goal of advancing next-generation precision radiopharmaceuticals for patients with hard-to-treat cancers.”
Background: The use of online physician rating platforms has significantly increased and has been shown to influence physician selection. There are limited data on the use of these platforms for rating surgeons. Objective: In this study, we sought to assess the geographic distribution of and patterns in rating scores of surgeons in the United States. Additionally, we examined rating volumes across different surgical specialties and the association between peer-nominated and patient-initiated ratings on online rating platforms in the United States. Methods: We conducted a cross-sectional study by identifying 201,154 surgeons in the United States via the National Plan and Provider Enumeration System records and Doctors and Clinicians downloadable file. We assessed surgeon coverage on 3 online rating platforms and their geographic use patterns. We described the rating scores and volumes across different surgical specialties and assessed the relationship between rating platforms by comparing peer-nominated and patient-initiated online ratings. Results: A total of 78.86% (158,630/201,154) of the surgeons had ratings on at least 1 of the 3 patient-initiated websites across 11 specialties. Plastic surgeons, neurosurgeons, and orthopedic surgeons had the highest mean number of patient-initiated ratings. Surgeons with “Top Doctor” recognition from peers (23,171/201,154, 11.52%) were associated with an increased median patient-initiated rating (Healthgrades: 4.36, IQR 3.88-4.71 vs 4.20, IQR 3.64-4.64, <.001, and =0.09; Vitals: 4.30, IQR 4.00-4.60 vs 4.20, IQR 3.80-4.50, <.001, and =0.09; RateMDs: 4.20, IQR 3.80-4.50 vs 3.80, IQR 3.60-4.60, <.001, and =0.16). Geographic analysis indicated that 91.06% (295,816,471/324,870,510) of the US population lives in a county with a surgeon rated 10 times or more. Conclusions: Both patient-initiated and peer-nominated rating platforms have a comprehensive coverage of surgeons in the United States, but this coverage differs significantly between surgical specialties. Further work should assess how publicly available online ratings drive surgeon selection and their association with patient experience and postoperative outcomes.
Cancer researchers in the United States are once again bracing for a high-stakes funding battle in Washington, as a proposed $6 billion cut to the National Institutes of Health (NIH) for fiscal year 2027 threatens to derail years of scientific progress.
For advocates like Jon Retzlaff, Chief Policy Officer and Vice President for Science Policy and Government Affairs at the American Association for Cancer Research (AACR), the situation feels strikingly familiar and deeply consequential. That sense of déjà vu is shaping the response from the cancer research community, which is now urging Congress to once again reject the administration’s proposal just as it did last year.
To understand the urgency of the current moment, Retzlaff points back to the turmoil of the previous budget cycle. “A year ago, the president had proposed a 40% cut to NIH,” Retzlaff told Inside Precision Medicine. “Things looked pretty bleak.” The consequences were immediate and unsettling: grants were stuck and there were cutbacks on committees and staff.
But Congress ultimately intervened decisively. “We engaged with Congress, who has the power of the purse,” Retzlaff said. “They summarily rejected the president’s proposal for the 40% cuts and instead provided a $450 million increase for NIH.” Lawmakers also delivered a significant boost to the National Cancer Institute (NCI), reinforcing what Retzlaff described as a clear signal of bipartisan support for biomedical research. “What we saw for the current fiscal year… is they summarily rejected the president’s proposal,” Retzlaff said. “So now we are going through the exercise all over again.”
Despite the renewed threat, Retzlaff sees reasons for hope rooted in last year’s outcome. “People asked, ‘How can you be so optimistic?’” He recalled the earlier funding fight. “At least this year, I’m going to be able to tell them why I can be optimistic,” he said. “Because it was Congress that stood up.”
Still, he cautioned against complacency. “We can’t rest on our laurels. We can’t take it for granted,” Retzlaff said. “We will be continuing to press the issue.”
Holding down the precision oncology fort
For AACR, the renewed funding fight underscores a central truth: cancer research depends on long-term, uninterrupted investment. “You need this sustained funding over time,” Retzlaff said. “You go where the science is showing opportunities and also where there might not be opportunities right now.”
He emphasized that scientific progress is rarely linear or predictable. “Even though people can’t necessarily say it’s clear-cut that if we do research in this, we’re going to make some progress,” he said, “for some of the cancers, we just need to do research to try to have that knowledge discovery going on.”
Retzlaff added, “Basic biology is so important,” stressing that foundational science underpins every future breakthrough and the continued growth of precision medicine as the new standard of healthcare. “It’s about identifying the biomarkers that are important,” Retzlaff explained.
Meanwhile, emerging areas such as cancer vaccines are generating both excitement and urgency. “Cancer vaccines are now a big issue,” he said. “AACR is very interested in pushing that kind of research forward.”
Yet all of this progress depends on stable funding. Without it, Retzlaff warned, research priorities could narrow dangerously. “If you start cutting back, the next thing you know, we’re just funding breast cancer and lung cancer,” he said, “whereas the rare cancers need to be investigated. We need to give those people hope.”
Sustained national commitment to health
Funding cuts would also ripple through the clinical research pipeline. Retzlaff, who has become more involved in clinical trials in recent years, noted their complexity and cost.
While pharmaceutical companies often support later-stage development, early and exploratory studies depend heavily on NIH funding. “We rely on pharmaceutical companies… once you get into the translational part,” he explained, but without federal investment at the front end, fewer discoveries will ever reach that stage.
For AACR, protecting NIH funding is about more than preserving scientific momentum; it’s about sustaining a national commitment to health. “We’ve got 50,000 members,” Retzlaff said. “Two-thirds of them are from the U.S., and probably two-thirds of them are completely reliant in many ways on NIH funding.” That dependence drives the organization’s advocacy efforts. “Our number one priority is inspiring excitement on Capitol Hill and from lawmakers for robust, sustained and predictable funding for the NIH,” he said.
AACR’s outreach spans everything from congressional briefings to large-scale advocacy events. “It’s working with the entire community,” Retzlaff said, noting collaborations with hundreds of organizations and initiatives, such as Medical Research Hill Day. “We’re constantly looking at drum[ming] up conversations with the media,” he added. “It’s things like that—briefings, reports, letters—you name it.”
At the same time, AACR is navigating broader policy and public health challenges. Retzlaff highlighted ongoing engagement with the Food and Drug Administration (FDA) on issues ranging from clinical trial efficiency to tobacco regulation.
Backing cancer vaccines
Prevention, too, remains a critical priority. “HPV prevention is very important,” he said, though he acknowledged that misinformation has slowed progress. “The anti-vaccine movement is a huge concern.”
Retzlaff said that the cancer vaccine issue is rooted in communication and not the regulators. According to Retzlaff, the director of the National Cancer Institute has had some meetings with Secretary Kennedy, who was supportive of moving cancer vaccines forward. “We have to figure out what it is that people will accept about cancer vaccines that they’re not accepting about vaccines overall,” Retzlaff said. “That’s a communication issue… trying to combat the misinformation out there.”
AACR has even debated trying new names for the modality. Retzlaff elaborated, “There was some discussion about whether we can change the name of this from ‘cancer vaccines’ to something else.”
As Congress weighs the proposed cuts, AACR is calling on researchers, patients, and advocates to speak out once again. The message, Retzlaff said, is simple but urgent: “We definitely want to get the information out… about the importance of NIH medical research… and inspire people to take action.”
The outcome will determine not only the trajectory of cancer research but also the pace at which new discoveries can translate into treatments and, ultimately, save lives.
While developing an ultrasensitive test for the detection of Mycobacterium tuberculosis DNA (TB-DNA), researchers from Boston University have unexpectedly found a high prevalence of the molecular marker in U.S.-born patients hospitalized in Boston.
“We began this research with the intent of sourcing respiratory samples to support the ongoing development of a new molecular assay for TB,” said Guillermo Madico, MD, PhD, scientist at Boston University’s National Emerging Infectious Diseases Laboratories (NEIDL) and co-inventor of the TOP TB assay. “What we found was completely unexpected. Our ultrasensitive test is detecting Mycobacterium tuberculosis DNA in patients who are unlikely to be diagnosed with TB using current methods. This opens the possibility that there could be thousands of Americans infected with forms of tuberculosis disease that remain hidden from our current diagnostic tools—putting them at risk of developing more serious complications or potentially transmitting the disease to others.”
In 2022, there were over 8000 reported cases of TB in the United States, over 600 TB-related deaths, and an estimated 13 million people with Mycobacterium tuberculosis infection. Although incidence has steadily decreased in the U.S., the rate of decline is too slow to meet the ambitious World Health Organization strategy to end the global TB epidemic by 2035.
One threat to the global elimination goal is a gap in the detection of paucibacillary TB disease—a type of TB characterized by a low concentration of M. tuberculosis bacilli in samples that often results in false negative test results.
To improve detection, Madico and colleagues developed an ultrasensitive molecular assay developed at Boston University called the Totally Optimized PCR (TOP) TB assay, which targets a gene involved in M. tuberculosis cell wall assembly.
During the development process, the researchers conducted three separate clinical studies involving 297 patients from Boston hospitals.
Across the studies, the TOP TB assay detected TB DNA in 12–16% of samples—a rate far higher than expected given Boston’s low TB incidence rate. Of note, most TB DNA-positive patients tested negative on standard TB infection tests (tuberculin skin tests or interferon-gamma release assays), and the researchers hypothesize that the findings “indicate the existence of a paucibacillary form of TB that remains unrecognized and is not detectable using current diagnostic tools.”
During the study, there were three patients diagnosed with acute chest syndrome, a life-threatening complication of sickle cell disease, all of whom tested positive for TB DNA.
The researchers point out in Nature Communicationsthat this “previously unrecognized association” has potential implications for clinical care in the U.S. and many other settings.
“These findings suggest we may be missing a significant burden of TB disease, particularly in older Americans and in patients with certain underlying conditions,” said Edward Jones-López, MD, who co-led the study while at Boston Medical Center and Boston University Chobanian & Avedisian School of Medicine. “Most concerning is the potential association with acute chest syndrome in sickle cell patients. If confirmed and expanded upon in larger studies, this finding could lead to better health outcomes for patients with this potentially life-threatening condition.”
The researchers emphasize that their preliminary findings require confirmation in larger, prospective multicenter studies that include comprehensive clinical, radiological, immunological, and microbiological correlation. However, they argue the evidence warrants immediate dissemination given potential implications for medical care and public health.
AWS has now unveiled Amazon Bio Discovery, an AI platform that grants researchers direct access to a broad library of biological foundation models that can be fine-tuned for specific use cases in drug discovery. Announced at the AWS Life Sciences Symposium at the Javits Center in New York, the platform is supported by an AI agent that can select models for research goals, and evaluate candidates for synthesis and testing to enable a rapid lab-in-the-loop experimentation cycle.
While rising AI models show promise, they require coding skills and the ability to manage computing infrastructure. Additionally, diverse models face benchmarking challenges and moving candidates from computational design to physical synthesis remains a multi-step process. Given that data live in disconnected systems, scientists must manage multiple lab partners and manually coordinate timelines and execution.
Amazon Bio Discovery addresses these challenges with three capabilities: a benchmarked library of AI models and analysis packages, an AI agent that supports experimental lab, and integrated lab partners that test top antibody candidates and route results back to the researchers. This feedback loop improves the next round of design.
“AI agents make powerful scientific capabilities accessible to all drug researchers, not just those with computational expertise,” said Rajiv Chopra, PhD, vice president of AWS Healthcare AI and Life Sciences.
Currently, 19 of the top 20 global pharmaceutical companies use AWS to power research workloads. Amazon Bio Discovery will bring enterprise-grade scale, privacy, and security to researchers across pharmaceutical, biotech, and academic research organizations. MSK, Bayer, the Broad Institute, and Voyager Therapeutics are among early adopters of Amazon Bio Discovery.
Among the Amazon Bio Discovery broad catalog includes open-source and commercial models from Apheris and Boltz. Biohub and Profluent are expected to join the platform.
Amazon Bio Discovery enables scientists to fine-tune the model by feeding prior experimental data from their organization’s lab results into the application without complex training pipelines or custom code. In-house models can also easily be deployed and hosted within Amazon Bio Discovery.
To support model selection, an antibody benchmark dataset is available to evaluate the likelihood of a drug candidate to have favorable biological properties, such as manufacturability and stability.
Candidates selected for experimental validation can be directly sent to Amazon Bio Discovery’s integrated network of laboratory partners, including Twist Bioscience, Ginkgo Bioworks. A-Alpha Bio is also anticipated to join the network.
Twenty years ago, Sven Bocklandt, PhD, sought to create a hypoallergenic cat. He had the genetic engineering chops to do it, but the embryology was beyond his capabilities. At a small animal genetic engineering conference, known as TARC (Transgenic Animal Research Conference), held near Lake Tahoe, he met James Kehler, VMD, PhD, whose research at that time was to make transgenic and knockout cats as models of human disease.
The two men bonded, agreed the hypoallergenic cat idea was “crazy enough,” and decided to move forward with it. They worked together, completely unfunded, for years—FedEx’ing samples back and forth as Bocklandt was on the west coast and Kehler on the east coast—trying to make their “garage cat” while each one worked different day jobs.
Bocklandt, passionate about animal genome engineering, continued to develop different ideas for genome engineering in animals. Around the same time that he started sharing his ideas with scientists like George Church, PhD, a start-up focused on animal genome engineering was taking shape—Colossal Biosciences, co-founded by Church. Introductions were made, and Bocklandt joined in 2022 as species director to work on the dire wolf project. Kehler joined a short time later as VP. And everyone knows the rest of that story (there was no shortage of media coverage).
The pair eventually succeeded with the cat project: his name is Archie, and he is, Kehler noted, only partially hypoallergenic. But the generation of Archie and the dire wolves may not be the successes of this story. The real success may be what Bocklandt and Kehler learned along the way—and what they are going to do next.
Chickens as the next biologic factory
Neion Bio, co-founded by Dimi Kellari and Sam Levin, PhD, and located on the Rockefeller University campus on the east side of Manhattan, is aiming to re-engineer eggs to produce drugs in chickens. The team uses genetic engineering to integrate therapeutic proteins into native egg proteins, creating a new manufacturing platform for drugs that runs on grain and water.
Bocklandt joined the team at Neion Bio as CSO after leaving Colossal in 2024; Kehler joined more recently, as head of avian sciences.
When thinking about producing complex proteins, using the chicken “makes a lot of sense,” Bocklandt told GEN. Breeding and genetic engineering are all established in the chicken. And the vaccine industry has established an existing infrastructure to grow eggs under disease-free conditions. Purifying proteins out of an egg, Bocklandt added, is easier than purifying them out of a Chinese hamster ovary (CHO) culture (the traditional cell choice for drug production) because there are fewer host proteins.
Sven Bocklandt, PhD [Marco Figueroa]
It makes “far more sense” than what we’re doing right now, Bocklandt noted, which is using CHO cells. “Everyone is doing that because everyone has been doing it that way,” he asserted.
“The fact that we’re now seriously questioning whether CHO cells should remain the default manufacturing platform for biologics is long overdue,” noted Ola Wlodek, PhD, CEO of Constructive Bio. “Any credible new approach that breaks this decades-old lock-in is ultimately good for patients and for the field.”
For Kehler, who did his graduate work in the lab of stem cell pioneer Hans Schöler, PhD, the chicken is a clear choice because it is the only species, besides the mouse, where the primordial germ cells have been used to transmit genetically modified gametes to the next generation.
Mike McGrew, PhD, group leader at the Roslin Institute in the U.K., and an advisor to Neion Bio, demonstrated years ago that modifying chicken primordial germ cells is a reliable way of making gene-edited chickens. This background is comforting to Kehler, who noted that, “unlike at Colossal, where everything was bleeding edge, we are able to focus on a single species and capitalize on some pretty tried and true technology.”
Drugs in eggs meet biomanufacturing reality
The lab space on the Rockefeller University campus can support research and even house chickens. But it cannot support the production of a drug. When asked about turning their egg-borne proteins into drugs, the company leans on the existing infrastructure that supports vaccines in specific pathogen free (SPF) eggs. The idea is that the egg whites will be frozen in giant batches and then processed in a CDMO.
When asked about potential challenges, Bocklandt noted that, “technically, there’s not much to worry about. I have no concerns about Neion Bio being able to do what we want to do or what we need to do.”
But there may be hurdles ahead. Rahul Dhanda, co-founder, president, and CEO of Syntis Bio, told GEN that “at the beginning, everything can look like it has infinite potential—it’s when you actually build and operate the system that the real challenges show up.”
More specifically, Dhanda pointed out that biomanufacturing “ultimately comes down to reliable, consistent, and cost-efficient production.” Leveraging animal biology for drug manufacturing is exciting, he noted, “but scalability and cost are still open questions, especially at this early stage. Biological variability between animals and individual outputs, like eggs, introduces additional risk compared to more controlled cell-based systems,” Dhanda added.
Wlodek agreed: “because egg-based production is inherently a biological supply chain, it will face avian flu risks, batch-to-batch variability from seasonal and flock effects, animal-welfare/regulatory overhead, and practical limits on how fast you can expand output compared with stainless-steel or single-use fermenters.”
Microbial and yeast systems still “win decisively on GMP containment, land/water footprint,” she noted, and “the ability to go from a few liters to tens of thousands of liters in weeks rather than months.”
Dhanda agreed that “getting it to work in principle is far different from getting it to work at scale, and that seems far off.”
If these challenges can be addressed at scale, safely and humanely, Dhanda noted, the approach could deliver meaningful health benefits—”but there are still significant logistical and technical hurdles to work through.”
Engineering the chicken genome
Creating dire wolves at Colossal started with deriving wolf cells, editing them, and cloning them back into a live animal. But cloning doesn’t exist in birds. To genetically engineer chickens, the Neion Bio team edits the germline, starting the process with a fertilized egg.
Neion Bio [Marco Figueroa]
The egg is incubated for 65 hours, at which point germ cells float in the blood because the ovaries and testes don’t exist yet. A microliter of the blood is removed, put into cell culture media, and the germ cells grow out. The transgene that codes for the therapeutic protein is inserted using CRISPR-Cas enzymes, in the coding region of a gene that codes for Ovalbumin—which makes up a bit over 50% of the egg white protein. This protein is made “on a massive scale” by the oviduct, the company noted.
The genome is screened for correct integration and potential off-target edits. Once the clone is approved, several thousand cells are injected back into another embryo (also at 65 hours old). After incubation, the egg hatches and becomes a chicken.
Kanuma set the precedent—but not the scale
In 2015, the U.S. Food and Drug Administration approved Kanuma (sebelipase alfa) to treat Lysosomal Acid Lipase (LAL) deficiency, also known as Wolman disease. The drug, an enzyme replacement therapy, was the first treatment for patients with the rare disease and the first drug to be made in chickens. Kanuma is produced by Alexion Pharmaceuticals, which was acquired by AstraZeneca in 2021.
This historical precedent may provide a proof of concept for Neion Bio. That said, “the scale required for Kanuma is very different from what would be needed for large biosimilars,” explained Wlodek.
An Odyssean journey
For both Bocklandt and Kehler, the move to Neion Bio feels like their careers are coming full circle. When Bocklandt first left Colossal, he was not sure how he would surpass that level of excitement. But the move came at an interesting time for him; the call to join Neion Bio came just weeks after he learned that his sister had been diagnosed with leukemia.
He thought, “Well, maybe this is not such a bad use of my skills.”
Earlier in his career, he didn’t think that he had anything special to add to a field like cancer research. But now Bocklandt sees it differently: throughout his career, he has pushed the state-of-the-art of genetic engineering. Now, he said, “I bring something to the field. And the fact that I can do my passion, animal genetic engineering, and apply that to make drugs better, cheaper, and more accessible, is really exciting.”
As for Kehler, Neion’s goal was his goal all along. He went to the University of Pennsylvania to make better animal models to test drugs for humans. “It never really dawned on me that we could use animals to make the drugs for humans. But taking everything I know about stem cell biology, germ cell biology, and gene editing, and bringing that to bear to make what should be a disruptive, transformational approach to making drugs—it feels like the culmination of my career.”
Neion (pronounced Neon) Bio is named after the birthplace of Odysseus; Mount Neion is a mountain mentioned in Homer’s The Odyssey as a landmark on Ithaca—Odysseus’ island home. As described by the company, the name is a testament to the shared qualities between the Greek hero and the company’s goals: relying on intelligence and resourcefulness over strength. And yes, Odysseus was successful in his return home to reclaim his throne. But it was a bittersweet success given the enormous cost and hardship.
Neion Bio’s name may mirror the resilience and ingenuity required to undertake the journey, but time will tell how long the similarities in the namesake are shared between the two.
Rise and shine, everyone, another busy day is on the way. And it is getting off to a good start here on the Pharmalot campus, where clear blue skies and unusually balmy breezes are greeting us. Who could ask for anything more? Actually, we could — it is time to reheat the kettle for another cuppa stimulation. Our choice today is elderberry-infused green tea. And here is a helpful tip — a teaspoon of honey enhances the flavors splendidly. Of course, you are invited to join us. For the full experience, we are now hawking replicas — take a look. Meanwhile, here are a few tidbits to help you along. As always, do keep in touch. We appreciate feedback, suggestions, criticism, and tips. …
In a bid toward greater transparency, the U.S. Food and Drug Administration sent reminder letters to more than 2,200 companies and researchers that they are required to report clinical trial results to a federal government database or they may face fines, STAT says. FDA officials disclosed that an internal analysis found results were not submitted for nearly 30% of studies that were “highly likely” to fall under mandatory reporting requirements. The agency also noted that the letters were sent to companies and researchers associated with more than 3,000 registered trials, some of which were publicly funded. In explaining its move, the regulator acknowledged a long-standing complaint from researchers who have argued that without access to specific data, trial results cannot be easily duplicated, which inhibits greater understanding of how medicines might work.
Novo Nordisk and ChatGPT maker OpenAI agreed to work together on how to leverage artificial intelligence to discover new drugs, the latest AI partnership in the medical field as health care companies seek to harness the technology to get ahead of the competition, The Wall Street Journal tells us. The drugmaker said it would integrate OpenAI’s models across its operations to help its workforce analyze complex datasets and reduce the time it takes to move from research to delivering treatments to patients. The group said the partnership would boost efficiency across the organization, with pilot programs initially launching in research and development, manufacturing, and commercial operations ahead of a full AI integration by the end of the year.
This is today’s edition of The Download, our weekday newsletter that provides a daily dose of what’s going on in the world of technology.
Want to understand the current state of AI? Check out these charts.
If you’re following AI news, you’re probably getting whiplash. AI is a gold rush. AI is a bubble. AI is taking your job. AI can’t even read a clock. Stanford’s 2026 AI Index—the field’s annual report card—cuts through the noise.
Stanford’s 2026 AI Index is full of striking stats. It also reveals a field riddled with inconsistencies, most notably in the gap between experts and non-experts.
On jobs, 73% of US experts view AI’s impact positively, compared to just 23% of the public. Similar divides emerged on the economy and healthcare. What’s driving this disconnect?
Part of the answer may lie in their diverging experiences. Those using AI for coding and technical work see it at its best, while everyone else gets a more mixed bag. The result is two very different realities. Read the full story on what they are—and why they matter.
This story is from The Algorithm, our weekly newsletter on AI. Sign up to receive it in your inbox every Monday.
—Will Douglas Heaven
Job titles of the future: Wildlife first responder
Grizzly bears have made such a comeback across eastern Montana that in 2017, the state hired its first-ever prairie-based grizzly manager: wildlife biologist Wesley Sarmento.
For seven years, Sarmento worked to keep both bears and humans out of trouble. He acted like a first responder, trying to defuse potentially dangerous situations. He even got caught in some himself, which led him to a new wildlife safety tool: drones. Find out the results of his experiments in digital ecology.
—Emily Senkosky
This article is from the next issue of our print magazine, which is all about nature. Subscribe now to read it when it lands on Wednesday, April 22.
The must-reads
I’ve combed the internet to find you today’s most fun/important/scary/fascinating stories about technology.
1 Human scientists still trounce the top AI agents at complex tasks The best agents perform only half as well as experts with PhDs.(Nature) + Can AI really help us discover new materials? (MIT Technology Review)
2 OpenAI is escalating its fight with Anthropic while pulling away from Microsoft A leaked memo exposes plans to attack Anthropic. (Axios) + And says Microsoft “limited our ability” to reach clients. (The Information $) + While touting a budding alliance with Amazon. (CNBC)
3 Carbon removal technology is stalling—and that may be good news Better solutions could now emerge.(New Scientist) + Here are three that are set to break through. (MIT Technology Review)
4 AI is finding bugs faster than we can fix them—and hackers will benefit Welcome to the bug armageddon. (WSJ $) + AI may soon be capable of fully automated attacks. (MIT Technology Review)
5 A Texas man has been charged with the attempted murder of Sam Altman He allegedly threw a Molotov cocktail at the OpenAI CEO’s home last Friday. (NPR) + The suspect reportedly had a list of other AI leaders. (NYT $)
6 AI is beginning to transform mathematics It’s proving new results at a rapid pace. (Quanta) + One AI startup plans to unearth new mathematical patterns. (MIT Technology Review)
7 Students are turning away from computer science It’s had a massive drop in enrollments. (WP $) + AI coding tools have diminished the degree’s value. (NYT $)
8 India’s bid to become a data center hub is sparking a fierce backlash Farmers are protesting Delhi’s courtship of hyperscalers. (Rest of World)
9 Meta is set to overtake Google in advertising revenue this year And become the world’s largest digital ad platform for the first time. (WSJ)
10 AI influencers are taking over Coachella Synthetic content creators are “everywhere” at the festival. (The Verge)
Quote of the day
“These people are almost nothing like you. They are most likely sociopathic/psychopathic and, in the case of Altman, consistently reported to be a pathological liar.”
—The alleged firebomber of Sam Altman’s home shares his distrust of AI leaders in a blog post.
One More Thing
We’ve never understood how hunger works. That might be about to change.
A few years ago, Brad Lowell, a Harvard University neuroscientist, figured out how to crank the food drive to the maximum. He did it by stimulating neurons in mice. Now, he’s following known parts of the neural hunger circuits into uncharted parts of the brain.
The work could have important implications for public health. More than 1.9 billion adults worldwide are overweight, and more than 650 million are obese. Understanding the circuits involved could shed new light on why these numbers are skyrocketing.
