Nature Biotechnology, Published online: 26 May 2026; doi:10.1038/s41587-026-03140-1
A wearable ultrasound device is optimized for continuous monitoring of pregnancies.
STAT+: An AI biotech CEO sets the record straight on AI drug development hype
SAN MATEO, Calif. — Peyton Greenside, CEO of BigHat Biosciences, has hot takes.
Her company designs antibody therapies using machine-learning, but she doesn’t like demos in which people open their computers and show off how fast they can design a drug. “If you want me to design you a protein right now in six hours, I’m happy to do it,” she said. In fact, she can do it in 20 minutes. But if you’re actually in the business of making drugs, you are still going to have to do all the downstream tests, which take time and lots of money, Greenside said. “That’s where the hard work is, is making the actual drug.”
BigHat, founded in 2019, has successfully completed a collaboration with Johnson & Johnson and has partnerships with Merck, Amgen, Abbvie, Lilly, and others. In keeping with its name, it hangs a hat on the wall in its office for every completed project. There are a lot of hats.
Parabilis Files for IPO, a Day After Signing Up-to-$2.3B Regeneron Collaboration
Parabilis Medicines, the developer of drugs and antibody-drug conjugates targeting historically undruggable protein targets and based on stabilized helical peptides or Helicons
, has filed for an initial public offering (IPO), joining a growing parade of companies seeking to raise capital by tapping into the improving market for first time biotech stocks.
The company’s IPO filing came a day after Parabilis inked an up-to-$2.3 billion-plus strategic research collaboration with Regeneron Pharmaceuticals to discover and develop an initial five candidates encompassing “antibody-Helicon conjugates,” a new form of antibody-drug conjugates aimed at challenging and historically undruggable targets.
Regeneron has agreed to purchase approximately $75 million of Parabilis common stock in a concurrent private placement, at 90% of the IPO price per share.
It’s too soon to know how much money Parabilis plans to raise through the IPO. The company’s Form S-1 registration statement, filed Tuesday with the U.S. Securities and Exchange Commission (SEC), includes a placeholder “$100 million” figure that will inevitably be revised, and doesn’t say how many shares will be sold. Parabilis has applied to list its shares on The Nasdaq Global Market under the ticker symbol “PBLS.”
It’s also too early to know how much of the proceeds will go toward the four priorities it highlighted in its registration statement. Two of the four priorities relate to Parabilis’ lead Helicon peptide candidate zolucatetide (formerly FOG-001), a first and only direct inhibitor of the elusive β-catenin:TCF interaction, according to the company. Parabilis stated that it plans to continue ongoing clinical development of zolucatetide in desmoid tumors, including continuation of dose expansion and the launch of a Phase III registrational trial to topline data.
Parabilis also plans to continue ongoing clinical development of zolucatetide across several additional indications, including dose escalation and expansion in familial adenomatous polyposis (FAP); hepatocellular carcinoma, the most common type of primary liver cancer; and other rare tumors, with the aim of collecting data to support a registrational trial.
‘Expansive opportunity’
“We believe zolucatetide provides clinical validation of our first-in-industry Helicon approach and represents an expansive opportunity for medical and commercial impact,” Parabilis stated.
In addition, Parabilis plans to use IPO proceeds toward advancing its pipeline of additional programs—including its ERG protein degrader, an allosteric androgen receptor in its active state (ARON), and beta-catenin degraders—to Phase I clinical data; toward continued evolution of the Helicon platform for discovering and developing drug candidates; as well as toward general corporate purposes that include additional development efforts, working capital, and operating expenses.
According to Parabilis, zolucatetide has been evaluated in more than 150 patients to date and has generated positive clinical data in solid tumors characterized by alterations in the Wnt/beta-catenin pathway. In the drug’s lead indication of desmoid tumors, researchers have seen tumor reductions in 100% of patients with a 74% objective response rate (ORR) in patients who have had at least two post-baseline scans.
In March, Parabilis presented preliminary clinical data at the 11th Biennial Meeting of the International Society for Gastrointestinal Hereditary Tumors (InSiGHT) showing significant improvement in duodenal polyposis at 60 weeks in a patient with familial adenomatous polyposis (FAP) treated with zolucatetide in the company’s ongoing Phase I/II trial (NCT05919264).
The patient showed a 52.2% reduction in desmoid tumor diameter, as well as “substantial” reductions in polyp number and size compared with a pre-treatment evaluation nearly two years prior, consistent with downstaging from Spigelman stage II to stage I.
Parabilis says its Helicon discovery platform allows it to precisely tune potency, selectivity, and pharmacologic properties by integrating ligands and additional functionalities at multiple positions. The platform integrates artificial intelligence (AI)- and physics-based computational modeling with high-throughput peptide synthesis and experimental screening.
“While our initial programs are focused on disrupting protein-protein interactions and inducing targeted protein degradation, we believe our platform can incorporate other advances in small molecule drug design and extend them to targets that are likely to remain out of reach for other modalities,” Parabilis stated.
Parabilis was founded in 2015 as FogPharma to commercialize technology developed in and inlicensed from the lab of Harvard University researcher and serial entrepreneur Gregory Verdine, PhD. The company, which rebranded itself into Parabilis in 2024, says it has generated proprietary datasets, comprising millions of data points for hundreds of thousands of Helicons across dozens of drug-like properties, following a decade of Helicon drug discovery.
‘Continuous learning loop’
“These data power a continuous learning loop that refines our models from target selection through lead optimization, enhancing our speed, precision, and ability to generate high quality molecules against difficult targets,” Parabilis explained. “As a result, our platform produces unique complex synthetic molecules at scale and a compounding advantage that we believe is difficult to replicate.”
If it carries out the planned IPO, Parabilis would be the 12th biotech to go public this year. The 11 IPO companies to date have raised a combined $3.491 billion, compared to the $1.556 billion raised by 11 companies this time last year. Six of the 11 companies have seen their shares rise since their initial offerings, led by the 520% share price increase of Veradermics, a developer of treatments for dermatology and aesthetic conditions that closed Wednesday at $105.32 a share.
This year’s largest IPO—and the largest of any biotech—was the $625 million offering of Kailera Therapeutics, a developer of therapies for obesity and weight management based on glucagon-like peptide receptor 1 (GLP-1) agonists, alone or in combination with glucose-dependent insulinotropic polypeptide (GIP) receptor agonists.
Kailera last month completed what grew into a $718.75 million IPO last month that generated an estimated $662.1 million in net proceeds through the sale of 44,921,875 shares of common stock—including the exercise in full by underwriters of their option to purchase 5,859,375 additional shares—at the IPO price of $16 per share.
Should Parabilis’ planned IPO raise the placeholder $100 million amount, it would increase by 30% the $329.039 million in cash and cash equivalents that the company reported as of March 31.
Parabilis disclosed in its IPO filing that it ended the first quarter with a $45.316 million net loss, up 18% from its $38.342 million net loss of Q1 2025—as well as a net loss of $145.9 million for last year, up nearly 24% from its $117.9 million net loss for 2024. The company has no reported revenue.
Parabilis’ accumulated deficit rose 8% during Q1, to $586.82 million from $541.504 million at the end of 2025.
To fund its operations, Parabilis reported, it has raised a total $876.8 million as of March 31. That total consisted of $811.8 million from sales of its convertible preferred stock, $15 million in borrowings under a term loan and a $50 million Simple Agreement for Future Equity (SAFE).
The IPO comes just four months after Parabilis completed its last financing, an oversubscribed $305 million Series F round completed in January and co-led by investment firms RA Capital Management, Fidelity Management & Research Co., and Janus Henderson Investors.
Five investment firms were listed in the Form S-1 as underwriters for the IPO: Leerink Partners, BofA Securities, Evercore ISI, Guggenheim Securities, and LifeSci Capital.
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Human Gut Organoids with Functional Nerves Developed that Can Be Mass Produced
Researchers at Cincinnati Children’s Hospital Medical Center and Nantes Université in France have designed 3D-printed scaffolding trays that will reportedly allow scientists to produce larger versions of functional human gut organoids twice as fast as previous methods—and these organoids grow their own nerve cells.
This improved technology could help accelerate production of human mini-organ tissues that are large enough to be useful in patching damage or restoring diminished functions of a person’s small intestine, stomach, or colon. Such tissues also would be valuable for future disease studies and to more accurately evaluate organ damage risks linked to oral medications, according to the investigators.
Details of the study “Large-scale and innervated functional human gut tissues for transplantation via transient spheroid confinement” appear in Nature Biomedical Engineering.
Using a confined culture system (CCS), the team grew small intestine, colon, and stomach organoids from tiny spherical forms into centimeter-scale tubular forms nearly 10 times larger than previous methods. Also, unlike methods that require a complex effort to introduce nerve cells, these organoids develop a nervous system on their own.
“By reaching transplantation maturity twice as fast and developing their own functional nerves, these organoids demonstrate how engineering principles can drive biological innovation,” said staff investigator Holly Poling, PhD. “Our confined culture system is more than a production method; it’s a scalable, flexible platform for building complex human tissues.”
New production system prompts faster growth
Experts at Cincinnati Children’s Center for Stem Cell & Organoid Medicine (CuSTOM) have been making miniature versions of digestive system organs for more than 15 years, working on improving the sophistication of the lab-grown tissues. More recently, the team has been developing methods to make enough customized tissue to transplant into patients to help patch organ damage or restore diminished specialized functions.
The new technique uses 3D printing technology to make tray-like scaffolding molds from surgical resin, then filling the molds with degassed polydimethylsiloxane—a flexible rubber-like type of silicone.
The new trays contain grooves designed to confine a collection of sphere-shaped organoids into a row, which encourages the spheroids to fuse together and mature. The fusions occur within a special mix of nutrients and other ingredients that support initial growth from induced pluripotent stem cells (iPSCs) into more complex organoids.
By day six, the discrete spheroids develop into unified constructs along the grooves of the trays. These are moved into another hydrogel medium for continued growth for another eight days.
By day 14, the organoid constructs have produced all the cell types and structures that previously required 28 days to achieve. These tissues are then transplanted into rodents that are genetically modified to minimize rejection risk.
All of the transplanted tissues engrafted in rodents, the co-authors state. After growing in the rodents, the team produced as much as eight cm of functioning small intestine tissue, compared to approximately one cm of tissue using previous protocols. Not only were the structures much larger than previous methods, but now their neuromuscular function was also similar to native human tissue, representing a major advance.
“We are now able not only to generate complex gastrointestinal organoids at scale, but also to guide their differentiation into functional tissues with integrated enteric neuronal networks,” noted senior author Maxime Mahe, PhD. “By leveraging a defined growth environment, the intrinsic self-organization capacity of the cells drives the formation of tissue structures that closely resemble the human gastrointestinal tract.”
Jim Wells, PhD, a study co-author and chief scientific director at CuSTOM says the new technology overcomes key barriers to scale and function in organoid research and biomanufacturing.
“This platform’s simplicity, reproducibility, and versatility make it accessible for widespread adoption,” said Wells. “In addition, the emergence of a self-organized nervous system within these organoids is particularly important for further studies of neurodevelopmental disorders.”
Another step closer to human clinical trials
Michael Helmrath, MD, a surgeon-scientist at Cincinnati Children’s who co-directs CuSTOM, has been working for more than a decade to develop intestine organoids sophisticated enough for transplantation in human patients.
In 2017, Helmrath and colleagues demonstrated how to combine neural crest cells with intestinal tissue cells in a layered process to make the first human organoids with nerve function. His team also showed how intestine organoids could be grown larger by implanting them in a mouse to provide a blood supply. Ever since, intestine organoids have been getting more sophisticated, including versions with immune cells in addition to the specialized organ cells and nerves.
Now the new process—involving rats instead of mice—produces larger amounts of tissue.
“It is still not possible to grow complete, full-sized human organs in some sort of tank, but research like this has produced significant amounts of tissue that can be matched directly to individual patients,” explains Helmrath. “We believe such tissues, once transplanted, would further grow and multiply as part of the patient’s own organ to restore functions.”
More research and development is needed before “CCS organoids” will be ready for human clinical trials, according to Helmrath. But if successes continue, organoid medicine may allow more infants and children with dysfunctional organs to be treated without ever needing a full organ transplant.
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Enzymes Involved in Cholesterol Transport May Point to New Cancer Therapies
Some types of cancer have a relentless appetite for the metabolite cholesterol, using as much as they can access to accelerate their growth beyond the capabilities of normal cells. Research by scientists at Sanford Burnham Prebys Medical Discovery Institute and collaborators at the University of Illinois Chicago have now unveiled a potential method for turning the table on these tumors by subverting their cholesterol cravings.
The researchers’ studies, in mice and in human cancer cells, revealed new insights into enzymes known as phosphatidylinositol 5-phosphate 4-kinases (PI5P4Ks) that help move cholesterol around cells. The researchers showed that without the help of these enzymes, a cholesterol traffic jam occurs, blocking the cancer cell’s ability to fuel tumor growth.
Headed by Brooke Emerling, PhD, the director of and associate professor in the Cancer Metabolism and Microenvironment Program at the Sanford Burnham Prebys NCI-Designated Cancer Center, the team reported on its findings in Science Advances, in a paper titled “Noncanonical PI(4,5)P2 coordinates lysosome positioning through cholesterol trafficking.”
The TP53 gene is mutated in roughly half of all cancers. Emerling and first author Ryan Loughran, PhD, a postdoctoral associate in the Emerling lab, focus on difficult-to-treat forms of breast cancer, where TP53 mutations are found in more than 84% of triple-negative breast cancers and three of every four HER2-amplified breast cancers.
Cancer cells with a mutation in the tumor-suppressing TP53 gene are known to produce extra cholesterol. This may make them more vulnerable to starvation if scientists can put a stop to the steady supply of the lipid. “We need more ways to treat cancers with this common mutation,” said Emerling. “One of our main goals with this work was to find new treatment possibilities for the large subset of breast cancers harboring TP53 mutations,” said Loughran. “We recognized a real opportunity in targeting the enzymes that control cholesterol transport, especially since cancer cells depend on this process far more than normal cells do.”
To better understand how to turn these cancers’ cholesterol consumption into a weakness, the research team turned to a family of cell membrane lipids known as phosphoinositides and the kinase enzymes that regulate them. The investigators had shown that a branch of the lipid enzyme family known as PI5P4Ks were required for the growth of cancers with TP53 mutations in mice, and they suspected that this tumor prevention was due to the enzymes’ role relocating cholesterol in the cell. “Our group has shown that suppression of the most catalytically active PI5P4K isoforms (α and β) in TP53-deficient cancer cells inhibits proliferation, and the deletion of these enzymes in Trp53-knockout mice confers protection from tumorigenesis,” the investigators wrote.
“Normally, when mice lose TP53 as the guardian of their genomes, they are fated to die from cancer in four-to-eight months,” said Emerling. “When you delete these kinases, the animals are 100% protected and never develop a tumor—and cholesterol turned out to be one of the missing pieces in this puzzle.”
The scientists conducted experiments in mouse and human cancer cells showing that PI5P4Ks influenced the movement and behavior of organelles that carry cholesterol around our cells. In cancer cells with TP53 mutations and PI5P4Ks, cholesterol-laden lysosomes were found near the exterior cell membrane. Without PI5P4Ks, lysosomes remained in the interior of the cells, near the nucleus.
Location is critical for lysosomes transporting cholesterol. While positioned near the edge of the cell, lysosomes and their cargo are in proximity with many receptor proteins, enzymes and signaling molecules that exist around the cell membrane. This includes mechanistic target of rapamycin complex 1 (mTORC1), an enzyme that governs cell growth and runs amok in cancer. “When lysosome positioning is biased towards the cell nucleus, mTORC1 activation is suppressed,” said Loughran. “This connects directly to our previous work, where we found that the loss of these kinases triggers starvation-like states in cancer cells. “When PI5P4Ks are absent, the link between lysosomal cholesterol and mTORC1 is compromised, a bit like two ships passing in the night.”
The change in lysosome position towards the cell’s interior that occurs without PI5P4Ks reduced interaction with mTORC1 and prevented it from sending signals associated with tumor growth. “The mTOR activation pathway is really what drives tumorigenesis, and so mTOR is an important target for cancer drug development,” said Emerling. “If we can target mTOR activity in aggressive cancers by blocking the sensing of cholesterol, that would be a promising treatment strategy.”
In their report the authors noted in summary, “The dependence of p53-deficient tumor cells on PI5P4Ks has been previously attributed to their roles as critical modulators of cellular stress responses, including protection from oxidative stress, maintenance of mitochondrial health, and regulation of autophagy. We now identify a previously undescribed role for PI5P4Ks in maintaining lysosomal cholesterol homeostasis and mTORC1 signaling.”
Previous research has looked at the use of statins as cancer drugs due to their ubiquity and safety as treatments for patients with high cholesterol. While more research is needed, studies so far suggest that tumors eventually acquire resistance to statins. “While cholesterol synthesis inhibitors such as statins have shown initial success, their efficacy is often compromised by the development of acquire resistance,” the team noted in the paper. “Consequently, strategies are being explored to disrupt cholesterol homeostasis more comprehensively by inhibiting its synthesis and intracellular transport.”
Loughran added, “It is important for us to find other ways to more comprehensively cut cancer cells off from cholesterol to impede their growth.” Emerling further stated, “We’ll continue to explore blocking PI5P4Ks as a more targeted approach tailored to how tumors operate.”
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AI Scientists, Stock Tumble, Patent Lawsuit, and New Partnerships
Agentic AI is growing in its applications. Google DeepMind and Edison are leveraging the growing capabilities by developing AI Scientists. These platforms are poised to streamline the scientific process, aiding human scientists with a variety of tasks. Meanwhile, despite positive data in its Phase III DMD therapy trial, Regenxbio’s stock fell for a variety of reasons. 10X Genomics and Harvard University are suing Element Biosciences over patents for a multiomics platform. Finally, Bristol Meyers Squibb is partnering with Hengrui Pharma to develop 13 early-stage programs with the potential to grow their investment to a predicted $15 billion in sales.
Listed below are links to the GEN stories referenced in this episode of Touching Base:
The State of Precision Medicine Summit
Join us June 3, 2026
Google DeepMind and Edison Are Building the AI Scientist
By Fay Lin, PhD, GEN Edge, May 19, 2026
StockWatch: Regenxbio Tumbles Despite Positive Pivotal Data for DMD Gene Therapy Candidate
By Alex Philippidis, GEN Edge, May 17, 2026
10x Genomics, Harvard Target Element’s Multiomics Platform in Patent Lawsuit
By Alex Philippidis, GEN Edge, May 12, 2026
BMS, Hengrui Pharma Partner on 13 Programs in Up-to-$15.2B Collaboration
By Alex Philippidis, GEN Edge, May 13, 2026
Touching Base Podcast
Hosted by Corinna Singleman, PhD
Behind the Breakthroughs
Hosted by Jonathan D. Grinstein, PhD
The post AI Scientists, Stock Tumble, Patent Lawsuit, and New Partnerships appeared first on GEN – Genetic Engineering and Biotechnology News.
STAT+: EU and US advisers split over AstraZeneca breast cancer drug
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Merck’s bet on an antibody-drug conjugate developed in China has paid off in a major Phase 3 lung cancer study, where sacituzumab tirumotecan paired with Keytruda sharply outperformed Keytruda alone in a subset of lung cancer patients.
Also, the NIH is down another leader, and Sam Altman-backed longevity startup Retro Biosciences raises more funds.
STAT+: Longevity startup Retro Biosciences says latest fundraising values it at $1.8 billion
Retro Biosciences, the longevity startup backed by OpenAI CEO Sam Altman, has raised more money at a $1.8 billion valuation, it announced Friday.
Retro has a big mission: Add 10 healthy years to the human lifespan. It is seeking to do that by using a variety of technologies, including in vivo gene therapies, cell replacement therapies, and other approaches to spur younger, healthier cells into aging tissues.
The company is currently running its first clinical trial — testing a pill designed to enhance the body’s ability to better clear out protein aggregates in patients with Alzheimer’s disease. Retro CEO Joe Betts-LaCroix told the audience at STAT’s Breakthrough Summit West on Tuesday that the trial is going “super good” and that researchers haven’t seen any dose-limiting toxicities. He said he anticipates releasing some data from the trial around August.
STAT+: Merck-Kelun lung cancer drug cut risk of tumor progression by 65%, ASCO abstract shows
A type of targeted chemotherapy developed by China-based Kelun-Biotech and licensed to Merck cut the risk of tumor progression by 65% in patients with lung cancer, according to Phase 3 study results reported Thursday.
A preliminary survival benefit favoring the Kelun-Merck drug, called sacituzumab tirumotecan, or sac-TMT, was also seen in the study, but will require longer follow-up to confirm.
The study, conducted in China, is the first successful combination of an antibody-drug conjugate with a PD-1-targeted immunotherapy in patients with advanced but previously untreated non-small cell lung cancer.
Wacker Expands Service Offerings with Launch of Contract Research for Nucleic Acid-Based Therapies
Wacker reports that it is launching contract research services (CRS) at its biotech center in Munich for R&D-grade pDNA, RNA, and LNPs for preclinical studies. The new offering complements services of subsidiary CDMO Wacker Biotech, which has sites in Germany, the Netherlands, and the U.S.
In addition to producing pDNA, RNA, and LNP formulations, Wacker’s CRS team says it offers construct design services, including plasmid and RNA construct design as well as RNA engineering and optimization via partners, e.g., UTR, poly(A) and cap optimization. Company scientists also conduct lipid library screening and lipid nanoparticle formulation and provide functional assays and analytical services.
By integrating early-stage R&D support with a globally interconnected GMP manufacturing network, CRS helps customers streamline development and reduce supply-chain fragmentation, maintains a Wacker spokesperson. The approach enables an accelerated path from design to delivery in the field of advanced therapies, while lowering risk and cost through resourcing in early phases and a scalable transfer to Wacker Biotech for clinical material, continued the company official.
“Every RNA or LNP project is unique. Our goal is to provide flexible, customizable services that adapt to our clients’ specific needs in a rapidly evolving landscape,” explained Christian Dubiella, the CRS global program manager. “Too often, innovative, potentially life-saving therapeutic concepts die on the vine due to the high cost of developing even small amounts of drug substance for R&D studies. Our CRS enable us to serve highly specialized customers, especially small startups.”
One of CRS’ first customers is SRTD Biotech, an emerging biotech in Germany, which is starting small scale on novel therapeutic approaches.
“Our platform technology based on seRNAs (selectively expressed RNAs) can easily be adapted to numerous therapeutic areas by utilizing the transcriptome for selective cell targeting and fusogenic LNPs for organ-specific targeting,” said Bernd Hoffmann, CEO/CSO and cofounder of SRTD. “Wacker is an ideal partner on the road to realizing our vision of delivering seRNAs to patients to improve their lives.”
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