Radically different
Nature Medicine, Published online: 20 May 2026; doi:10.1038/s41591-026-04423-5
Analyses of sequencing data from 75,602 children referred for genetic testing from 2016 to 2025 provide insights on the association between pathogenic germline variants and childhood cancer.
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Regeneron Pharmaceuticals will partner with Parabilis Medicines to discover and develop an initial five candidates encompassing a new form of antibody-drug conjugates aimed at challenging and historically undruggable targets, through a strategic research collaboration that could generate up to $2.3 billion-plus for the Cambridge, MA, biotech.
Regeneron will marry its antibody capabilities with Parabilis’ stabilized helical peptide or Helicon
platform, to develop both Antibody-Helicon Conjugates (AHCs) as well as stand-alone therapies based on Helicons—stabilized, cell-penetrant alpha-helical peptides designed to engage intracellular protein targets, including flat surfaces that are not well suited to traditional small molecule binding.
While ADCs traditionally use antibodies to selectively deliver drug payloads into target cells to induce their death from within, the AHCs envisioned by Regeneron and Parabilis would combine antibody-targeted cell access with Helicon payloads designed to selectively modulate specific intracellular proteins, including some long-undruggable proteins.
“In addition to the potential of Helicons to address previously undruggable targets, the collaboration’s intent to couple Helicons to our VelocImmune® derived-antibodies so as to precisely deliver them to cells of interest represents an exciting new approach with the potential to create an entirely new therapeutic class that can span multiple therapeutic areas,” George D. Yancopoulos, MD, PhD, Regeneron’s board co-chair, president, and CSO, said in a statement.
Regeneron has agreed to pay Parabilis $125 million, consisting of a $450 million upfront payment and commitment to invest $75 million in Parabilis’ next equity financing, subject to specified conditions. Regeneron also agreed to pay Parabilis payments tied to achieving development, regulatory, and commercial milestones, as well as tiered royalties up to the low double-digits on future net sales of any approved medicines resulting from the collaboration.
With five initial targets, the collaboration agreement could generate up to approximately $2.2 billion in total milestone payments to Parabilis.
Under the terms of the agreement, additional targets may be pursued upon additional option payments from Regeneron.
Regeneron shares fell nearly 10% Monday, to $629.68, from Friday’s close of $698.25, and plateaued on Tuesday, inching up 0.1% to $630.30. The Monday drop reflected not the Parabilis deal but a clinical setback: Regeneron on Friday evening acknowledged the failure of a Phase III trial (NCT05352672) assessing two dose levels of the lymphocyte-activation gene-3 (LAG-3) inhibitor fianlimab in combination with a PD-1 inhibitor, Regeneron’s marketed drug Libtayo® (cemiplimab), as a first-line treatment for patients with previously-untreated, unresectable locally advanced or metastatic melanoma.
Fianlimab plus cemiplimab failed the trial by not reaching statistical significance for the primary endpoint of improvement in progression-free survival (PFS) compared to monotherapy with another PD-1 inhibitor, Merck & Co.’s Keytruda® (pembrolizumab), the multi-indication cancer immunotherapy, Regeneron said, in an announcement released more than four hours after the close of financial markets.
Parabilis, a privately held company which rebranded from FogPharma in 2024, rang in 2026 by announcing the closing of a $305 million Series F financing on January 8, with proceeds intended to support continued clinical development of its 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—including progression toward a registrational trial in desmoid tumors and continued evaluation across genetically simple and more complex tumor types.
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.
The financing, Parabilis added, will also support advancement of its targeted discovery pipeline, including its prostate cancer franchise, and additional efforts to leverage the company’s Helicon platform to unlock long-undruggable disease targets.
In addition to zolucatetide, Parabilis’ pipeline includes:
“Through our own pipeline, we have demonstrated the potential of Helicon peptides to directly inhibit or degrade several disease-driving proteins in oncology that have long been considered out of reach,” stated Mathai Mammen, MD, PhD, Parabilis’ chairman, CEO, and president. “We are thrilled to enter into a collaboration with Regeneron that builds on this foundation, combining the intracellular access and binding capabilities of our Helicons against challenging targets with antibodies from Regeneron.”
The post Regeneron, Parabilis Ink Up-to-$2.3B Antibody-Peptide Conjugate Collaboration appeared first on GEN – Genetic Engineering and Biotechnology News.
Nature Medicine, Published online: 19 May 2026; doi:10.1038/d41591-026-00026-2
The portable, low-resource MiniDock MTB test, evaluated across multiple countries, shows promise for detecting pulmonary tuberculosis using sputum and tongue swabs.
A new study in Science Signaling identifies a previously overlooked control point in receptor tyrosine kinase (RTK) signaling, one that operates not at the plasma membrane, but at the Golgi. The research, published as “Oncogenic receptor tyrosine kinase signaling is driven by the Golgi protein GOLPH3 and its interaction with MYO18A,” reveals that the Golgi‑localized proteins GOLPH3 and MYO18A act together to route RTKs to the cell surface, thereby setting the strength of growth‑factor signaling across multiple pathways.
The work was led by Kyle Starost and colleagues at Case Western Reserve University School of Medicine and the University of California, San Diego. Their findings help explain why GOLPH3 is frequently amplified in human cancers and why its overexpression correlates with poor prognosis across tumor types.
RTKs such as EGFR, insulin receptor, and PDGFR are central drivers of proliferation and survival in many cancers. Although RTK inhibitors are widely used clinically, resistance often emerges, underscoring the need for alternative strategies that modulate signaling upstream of the receptor. The new study identifies one such upstream node: the delivery of RTKs from the Golgi to the plasma membrane.
Using an unbiased signaling analysis, the team found that siRNA knockdown of GOLPH3 or MYO18A impaired phosphorylation of EGFR at Tyr1068 and Tyr1086, as well as downstream AKT and ERK signaling. These defects persisted even when PI3K/AKT/mTOR signaling was pharmacologically blocked, demonstrating that GOLPH3 acts directly at the receptor level rather than through mTOR modulation.
To pinpoint the mechanism, the researchers turned to trafficking assays. Imaging of endogenous EGFR showed that loss of GOLPH3 or MYO18A caused the receptor to accumulate in intracellular puncta rather than at the plasma membrane. A quantitative PDGFR‑GFP surface‑delivery assay confirmed that both proteins are required for Golgi‑to‑surface transport. Treatment with brefeldin A or golgicide A, which disrupt Golgi structure, produced similar reductions in surface receptor levels, reinforcing the conclusion that the GOLPH3–MYO18A complex is essential for RTK delivery.
Overexpression experiments completed the mechanistic picture. Increasing GOLPH3 or MYO18A levels enhanced EGF‑stimulated phosphorylation of EGFR and AKT, while a GOLPH3 mutant unable to bind PI4P failed to do so. These results position the GOLPH3–MYO18A complex as a central determinant of RTK availability at the cell surface.
The authors wrote, “The GOLPH3-MYO18A complex at the Golgi apparatus was required and rate-limiting for RTK signaling across the cell types and receptors assessed.” The findings suggest that targeting Golgi‑based trafficking machinery could offer a new therapeutic angle for tumors that rely on hyperactive RTK signaling or have developed resistance to RTK inhibitors.
The post Oncogenic Signaling Shaped by a Golgi Trafficking Protein Pair appeared first on GEN – Genetic Engineering and Biotechnology News.
Virginia Gov. Abigail Spanberger has vetoed closely watched legislation to create an advisory panel to lower prescription drug costs, a setback to attempts by lawmakers across the United States to address the rising cost of medicines.
Unlike affordability boards in other states, the Virginia panel would have used Medicare as a benchmark. Rather than start from scratch to identify drugs considered expensive, each year the panel would have targeted the same drugs chosen by Medicare for price negotiations. The board would have also set upper payment limits to create a ceiling on what would be paid.
By doing so, Virginia would have leapfrogged plans by other states that are at varying stages of establishing affordability boards. Of the other nine states that have boards, none is eyeing all of the same drugs chosen annually by Medicare and only four have the authority to set upper payment limits.
Background: Traditional anatomy teaching relies on cadaveric dissection and 2D resources, which often require in-person attendance and may limit spatial understanding. Virtual reality (VR) provides an immersive, remote alternative that supports 3D visualization from home. Recent evidence suggests that while VR may yield comparable factual knowledge gains to 2D methods, its primary value lies in enhancing learner engagement, motivation, and perceived educational value. Objective: This pilot randomized controlled trial compares remote synchronized VR with didactic animated anatomy lectures for the teaching of tracheostomy anatomy. Methods: Participants were recruited via convenience sampling through the VRiMS (Virtual Reality in Medicine and Surgery) Surgical Society network. All participants first attended a synchronous 20-minute online lecture delivered by a consultant surgeon. They were then individually randomized to one of 2 groups using a computer-generated sequence. Allocation was concealed until the intervention; however, participants and researchers were unblinded. The intervention group completed a 10-minute metaverse-based VR session, delivered by a consultant surgeon via 3D Organon’s Medverse platform on the PICO 4 Ultra headset. The control group completed a 10-minute prerecorded 2D animated lecture, accessed on their personal device. Participants then swapped to the other modality. Data were collected via Google Forms at 3 intervals (baseline, postintervention, and postcrossover) to assess confidence, spatial understanding, and knowledge (10-item multiple-choice questions). The analysis of nonparametric data utilized Wilcoxon signed-rank tests for within-group changes and Mann–Whitney tests for between-group differences. Results: Twenty-four medical students from 11 United Kingdom and Irish medical schools participated. Adherence was 100%, with all participants completing their assigned 10-minute intervention and all assessment points. Ninety-two percent of participants (n=22) reported no prior tracheostomy anatomy teaching. Additionally, 83% (n=20) had no prior remote-synchronized VR anatomy teaching experience. Anatomical confidence improved significantly in the VR group compared with animation (mean change 1.58, SD 1.00 vs mean 0.50, SD 0.80, =.01). Knowledge scores improved significantly in both groups (VR: mean 1.75, SD 1.54, =.007; animation: mean 2.83, SD 1.70, =.003), with no significant postintervention difference between groups (=.46). VR participants reported significantly superior spatial understanding across all measured domains (all ≤.009). These included depth perception (3.75 vs 2.58, =.009), appreciation of anatomy from different viewpoints (4.25 vs 2.33, =.001), mental reconstruction from varying angles (3.83 vs 2.08, =.002), and spatial depth supporting anatomical understanding (4.08 vs 2.08, =.001). Following the completion of both modalities, participants rated VR as more engaging (mean 4.54, SD 0.78) and more educationally effective (mean 4.29, SD 0.95) than animation. Conclusions: Remote VR teaching is feasible and engaging and enhances spatial understanding compared to animation. While knowledge gains were comparable between modalities, VR improved learner confidence and perceived 3D comprehension. Hence, VR may represent a scalable adjunct or alternative to traditional anatomy teaching.
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