Biomedicine Weekly · Week 37, 2026 · 09.07 — 09.13
CBA FRONTIER INSIGHTS
This issue's figures
Open a card for the chart; click again to closeGene editing cures, a pelacarsen shock, and ivonescimab's survival win reshape the week
This week paired landmark gene-editing advances with sobering cardiovascular setbacks. An epitope-editing strategy enabled chemotherapy-free haematopoietic replacement in Nature, while a base editing therapy showed cures across ethnic backgrounds and Intellia's in vivo CRISPR therapy won FDA priority review. A gene-edited porcine kidney kept a patient dialysis-free for 271 days. In contrast, Novartis's pelacarsen lowered Lp(a) but missed its cardiovascular endpoint in Lp(a)HORIZON, and Novo Nordisk halted two ziltivekimab trials. Oncology delivered: ivonescimab cut death risk 27% versus pembrolizumab, osimertinib plus chemotherapy doubled progression-free survival in EGFR/TP53 co-mutated NSCLC, and the FDA accelerated approval of camizestrant combinations. Regulators also cleared an mRNA flu vaccine, semaglutide for MASH, and China issued DRG/DIP 3.0 with a 2027 deadline.
Gene editing and xenotransplantation reach new frontiers
On this pageKIT epitope editing enabled non-genotoxic in vivo conditioning and selection of transplanted haematopoietic cells, combined with fetal haemoglobin induction for sickle cell disease and beta-thalassaemia. Correctsequence's tBE-based CS-101/CS206 cured patients across ethnic backgrounds. KNIT editing inserted 0.7 kb to 10 kb DNA fragments without double-strand breaks at up to 89% efficiency, enabling non-viral CAR-T engineering. EpiReg-T silenced PCSK9 in macaques over 90% for 343 days after one dose. Intellia's lonvo-z won FDA priority review with a March 10, 2027 PDUFA date, while a gene-edited porcine kidney maintained dialysis independence for 271 days before a successful human allotransplant.
Non-genotoxic transplantation and in vivo selection through epitope editing.
Epitope editing of KIT lets transplanted haematopoietic stem/progenitor cells escape anti-KIT monoclonal antibodies, enabling non-genotoxic in vivo conditioning and selection. Adenine base editing or prime editing introduced the KIT mutations together with BCL11A erythroid enhancer disruption to induce fetal haemoglobin for sickle cell disease and β-thalassaemia. Edited haematopoietic cells progressively enriched under anti-KIT selective pressure without clonal selection, allowing chemotherapy- and radiotherapy-free haematopoietic replacement.
Base Editing Therapy CS-101/CS-206 Cures Beta-Hemoglobinopathy Patients Across Genetic Backgrounds
A base editing therapy developed with the transformer Base Editor (tBE), CS-101/CS-206 from Correctsequence Therapeutics, achieved clinical cure across patients with different genetic backgrounds, according to results published in Cell Stem Cell on September 7, 2026. Following 100% cure in Chinese beta-thalassemia patients, the study shows tBE is safe and effective in sickle cell disease and in Southeast and South Asian beta-thalassemia patients, supporting broad applicability across ethnicities.
Efficient and precise programmable DNA knock-in without double-strand breaks.
CRISPR kilobase-scale nickase-targeting (KNIT) editing inserts DNA fragments from 0.7 kb to over 10 kb without double-strand DNA cleavage by coupling a Cas9 nickase with a DNA donor recruiting system. It achieves up to 89% efficiency, cuts indel rates, translocations and off-target editing, restores gene expression in mutant cells, and enables non-viral CAR-T engineering with antitumour activity in vitro and in mice. This shifts kilobase-scale knock-in away from double-strand-break editing.
Design of optimized epigenetic regulators for durable gene silencing with application to PCSK9 in nonhuman primates.
An optimized TALE-based epigenetic regulator, EpiReg-T, silenced PCSK9 in macaque liver with over 90% efficiency lasting 343 days after a single lipid nanoparticle dose, lowering LDL cholesterol. In mice, EpiReg-T reached 98% efficiency versus 64% for the initial dCas9-based design. Multiomic analyses showed minimal off-target effects in monkeys, mice and human-derived cells, and the platform can be retargeted to other genes by reengineering the DNA-binding domain.
FDA Grants Priority Review to Intellia's In Vivo CRISPR Therapy lonvo-z for HAE
The FDA has accepted Intellia Therapeutics' BLA for lonvoguran ziclumeran (lonvo-z), an in vivo CRISPR therapy for hereditary angioedema, with priority review and a PDUFA goal date of March 10, 2027. The agency does not plan to convene an advisory committee. If approved, lonvo-z would be the first approved in vivo CRISPR therapy and the only single-treatment option for HAE.
Porcine kidney xenotransplantation as a bridge to allotransplantation: a first-in-human study.
A gene-edited porcine kidney (EGEN-2784) transplanted into a patient with end-stage kidney disease functioned immediately and maintained dialysis independence for 271 days before failing after immunosuppression reduction, with thrombotic microangiopathy despite negative donor-specific crossmatch. No porcine pathogen transmission or anti-HLA antibody change occurred. 82 days after nephrectomy, the patient received a human kidney allotransplant with immediate function and no sensitisation during 231 days of follow-up.
Cardiovascular disappointments: pelacarsen and ziltivekimab falter
On this pageNovartis reported on September 4 that pelacarsen missed Lp(a)HORIZON's primary endpoint: despite lowering Lp(a) by up to about 80%, cardiovascular events were not significantly reduced among 8323 patients in 42 countries, the first outcome trial testing whether Lp(a) reduction cuts events. The result forces a revaluation of the Lp(a)-lowering field. Novo Nordisk separately halted the HERMES and ATHENA trials of anti-inflammatory ziltivekimab after a monitoring committee judged them unlikely to succeed, following the drug's ZEUS failure despite reducing inflammation markers.
Novartis pelacarsen misses primary endpoint in Lp(a)HORIZON trial
Novartis reported on September 4 that the Lp(a)HORIZON trial of pelacarsen missed its primary endpoint. The trial enrolled 8323 patients across 42 countries and was the first outcome trial designed to test whether lowering Lp(a) reduces cardiovascular events; Lp(a) fell, but cardiovascular events were not significantly reduced, despite earlier studies showing pelacarsen can lower Lp(a) by up to about 80%. The result forces a revaluation of the entire Lp(a)-lowering field, showing that lowering the lipid does not itself translate into fewer events.
Novartis pelacarsen misses Phase III endpoint despite lowering Lp(a)
Novartis's pelacarsen lowered Lp(a) levels but failed to reduce cardiovascular event risk in a Phase III trial of 8323 patients that missed its primary endpoint. The miss forces the Lp(a) field, now a popular global drug development target, to confront whether lowering Lp(a) translates into cardiovascular benefit.
STAT+: Novo Nordisk stops two cardiovascular trials of drug aimed at lowering inflammation
Novo Nordisk has halted two cardiovascular trials of ziltivekimab, the HERMES and ATHENA studies, after a data monitoring committee judged them unlikely to succeed. The decision follows the earlier failure of the same anti-inflammatory drug in the ZEUS trial, where it lowered markers of inflammation but did not reduce the risk of major clinical complications, including deaths and heart attacks. The stop is another setback for targeting inflammation to improve cardiovascular health.
Oncology gains in lung, breast and gastroesophageal cancers
On this pageIvonescimab cut death risk 27% versus pembrolizumab in first-line PD-L1-positive NSCLC (Harmoni-2). Osimertinib plus chemotherapy extended median progression-free survival to 34.0 versus 15.6 months in EGFR/TP53 co-mutated NSCLC (HR=0.44). The FDA accelerated approval of camizestrant plus CDK4/6 inhibitors for ESR1-mutant breast cancer with a ctDNA companion diagnostic, and China's NMPA approved T-DXd plus pertuzumab first-line for HER2-positive metastatic breast cancer. The FDA also cleared zanidatamab first-line for HER2-positive gastroesophageal adenocarcinoma, triggering $250 million in Royalty Pharma financing. A spatial atlas of 520,850 cells mapped metastatic breast cancer progression and resistance.
Akeso, Summit’s ivonescimab tops Keytruda in lung cancer survival, with strong data in PD-L1-high patients
Ivonescimab, from Akeso and Summit, cut the risk of death by 27% versus Keytruda in the phase 3 Harmoni-2 trial, which tested the drug as first-line treatment for PD-L1-positive non-small cell lung cancer. The survival benefit was especially pronounced in patients with high PD-L1 expression, strengthening the case for a challenger to pembrolizumab in this setting.
Osimertinib plus chemotherapy doubles progression-free survival in EGFR/TP53 co-mutated advanced NSCLC
First-line osimertinib plus chemotherapy extended median progression-free survival to 34.0 months versus 15.6 months with osimertinib alone in treatment-naive advanced NSCLC patients with EGFR-sensitizing mutations and concurrent TP53 mutations, cutting the risk of progression or death by 56% (HR=0.44). The randomized phase 3 trial, led by researchers at Sun Yat-sen University Cancer Center and published in JAMA, is described as the first prospective trial dedicated to this co-mutated population, giving clinicians a biomarker-defined basis for adding chemotherapy to osimertinib.
FDA Grants Accelerated Approval to AstraZeneca's camizestrant Plus CDK4/6 Inhibitors for ESR1-Mutant Breast Cancer
AstraZeneca's camizestrant, combined with a CDK4/6 inhibitor (palbociclib, ribociclib or abemaciclib), received FDA accelerated approval on September 4 for adults with HR-positive, HER2-negative locally advanced or metastatic breast cancer who developed ESR1 mutations during aromatase inhibitor and CDK4/6 inhibitor treatment. The FDA also approved a companion diagnostic that detects emerging ESR1 resistance mutations in circulating tumor DNA. The approval rests on the pivotal Phase III SERENA-6 trial.
FDA grants accelerated approval to AstraZeneca's camizestrant combinations for ESR1-mutant breast cancer
The FDA granted accelerated approval to AstraZeneca's Etcamah (camizestrant) combined with a CDK4/6 inhibitor (palbociclib, ribociclib or abemaciclib) for adults with HR-positive, HER2-negative locally advanced or metastatic breast cancer whose ESR1 mutations were detected during aromatase inhibitor and CDK4/6 inhibitor therapy. The FDA also approved a companion diagnostic for detecting emerging ESR1 resistance mutations in ctDNA. The approval rests on positive results from the pivotal Phase III SERENA-6 study.
NMPA Approves T-DXd Plus Pertuzumab as First-Line Treatment for HER2-Positive Metastatic Breast Cancer
On August 11, 2026, China's NMPA approved trastuzumab deruxtecan (T-DXd) combined with pertuzumab for first-line treatment of adults with unresectable or metastatic HER2-positive breast cancer. The approval rests on the global DESTINY-Breast09 trial, which showed efficacy exceeding standard treatment with a safety profile validated in clinical testing. Professor Liao Ning offers a comprehensive assessment of the regimen, citing durable survival and manageable safety.
A spatial single-cell transcriptomic atlas of metastatic breast cancer progression
A single-cell spatial transcriptomic atlas of metastatic breast cancer profiles 520,850 cells from 126 tumor samples of 44 patients, covering 171,766 cancer cells and 349,084 tumor microenvironment cells across primary and metastatic lesions. Multiple time points per patient revealed treatment-induced transcriptional changes driving therapy resistance, immune subpopulations predictive of hormone receptor loss, and transcriptomic features of subclones selected during progression that hold future metastatic potential.
FDA approves Ziihera for first-line HER2-positive gastroesophageal adenocarcinoma, triggering milestones and $250 million Royalty Pharma financing
The FDA on August 25, 2026 approved Ziihera (zanidatamab-hrii) for first-line unresectable locally advanced or metastatic HER2-positive gastroesophageal adenocarcinoma: IHC 3+ and IHC 2+/ISH+ patients can receive it with tislelizumab plus chemotherapy, and IHC 3+ patients with chemotherapy alone. Under the BeOne Medicines collaboration, Zymeworks has received $81 million, with up to $144 million in further milestones and tiered royalties of up to 19.5%. The approval triggers the structure set up on March 2, 2026, when Zymeworks BC moved 30% of future Ziihera royalties into Zymeworks Royalty Limited Partnership, which borrowed $250 million in non-recourse notes from Royalty Pharma secured solely on those royalties.
Metabolic and liver disease: semaglutide and oral GLP-1 advances
On this pageNovo Nordisk's semaglutide injection was approved for MASH with moderate to severe liver fibrosis, based on part 1 of the ESSENCE trial in 1,197 patients with biopsy-confirmed F2 or F3 fibrosis, where both liver histology primary endpoints were met versus placebo. The oral small-molecule GLP-1 agonist Foundayo produced mean weight loss of 12.4% (27.3 pounds) at 72 weeks at the highest dose versus 0.9% for placebo on an on-treatment estimand, and 11.1% versus 2.1% under a treatment-policy estimand, in the ATTAIN program published in NEJM and The Lancet.
Novo Nordisk's Semaglutide Approved for MASH with Moderate to Severe Liver Fibrosis
Novo Nordisk's semaglutide injection (Wegovy/Novo Nordisk's brand Nuvo Ying) has been approved for MASH in patients with moderate to severe liver fibrosis. The approval is based on the primary results of part 1 of the ESSENCE outcomes trial, which enrolled 1,197 MASH patients with biopsy-confirmed F2 or F3 fibrosis. Semaglutide met both liver histology primary endpoints versus placebo.
Oral GLP-1 Agonist Foundayo Cuts Weight Up to 12.4% at 72 Weeks in ATTAIN Trials
The oral non-peptide small-molecule GLP-1 receptor agonist Foundayo produced mean weight loss of 12.4% (27.3 pounds) at 72 weeks at the highest dose versus 0.9% (2.2 pounds) with placebo, in an on-treatment estimand counting only those who stayed on the drug, within the ATTAIN global clinical program covering both diabetic and non-diabetic populations, with results published in the New England Journal of Medicine and The Lancet. Under a treatment-policy estimand including discontinuations, the highest dose gave 11.1% (25 pounds) versus 2.1% (5.3 pounds) for placebo.
Approvals and policy: vaccines, orphan drugs and DRG reform
On this pageThe FDA approved Moderna's mFlusiva, the first mRNA seasonal flu vaccine, for ages 50-64, beating GSK's Fluarix in two trials. Scholar Rock's Isembyld became the first therapy targeting SMA muscle loss for patients aged 2 and older. Priovant's dual TYK2/JAK1 inhibitor Lisraya was approved for adult dermatomyositis, the only approved targeted oral therapy for the disease. China's NMPA approved Tainomab's anti-RSV antibody Ruiwei Badan, with a single 120 mg injection protecting infants for up to 9 months (69.9% efficacy at day 270; 90.2% against severe cases). China's NHSA issued the DRG/DIP 3.0 grouping scheme, requiring practical application by end of March 2027.
Moderna flu vaccine wins FDA approval
The FDA has approved Moderna's mFlusiva, an mRNA-based seasonal flu vaccine, for people aged 50–64. It is the first seasonal influenza vaccine to use mRNA production, encoding full-length hemagglutinin from three WHO-recommended strains delivered in lipid nanoparticles. In two clinical trials, mFlusiva outperformed GlaxoSmithKline's four-strain vaccine Fluarix in preventing influenza infection or eliciting antibody responses.
STAT+: Scholar Rock wins FDA approval for first drug to target SMA muscle loss
The FDA has approved Scholar Rock's Isembyld, the first therapy to target muscle loss in spinal muscular atrophy, for adults and children aged 2 and older already receiving SMA therapies that target SMN2. In a late-stage trial, Isembyld combined with an SMN2-targeting drug improved young patients' motor skills over a year while placebo recipients declined, a statistically significant difference. Approval was granted on Friday.
FDA Approves Priovant's TYK2/JAK1 Inhibitor Lisraya for Adult Dermatomyositis
The FDA approved Priovant Therapeutics' Lisraya (brepocitinib), a first-in-class selective dual TYK2/JAK1 inhibitor, for adult dermatomyositis on August 27, 2026. Priovant was set up by Roivant and Pfizer, and the drug, originally coded PF-06700841, was discovered and taken through early-phase I/II trials by Pfizer. It is the only approved targeted oral therapy for dermatomyositis, received orphan drug designation and priority review, and is now available in the US.
NMPA Approves Tainomab's RSV Antibody Ruiwei Badan for 9-Month Infant Protection
China's NMPA has approved Zhuhai Tainomab's fully human anti-RSV monoclonal antibody Ruiwei Badan (formerly TNM001) for preventing RSV lower respiratory tract infections in neonates and infants entering or born during their first RSV season. A single 120 mg intramuscular injection protects for up to 9 months, with phase 3 efficacy at day 270 of 69.9% against medically attended RSV LRTI and 90.2% against severe cases, versus roughly 5 to 6 months for the imported nirsevimab. This is the company's second self-developed new drug after its tetanus antibody approved in 2025.
China's NHSA Issues DRG/DIP 3.0 Grouping Scheme With 2027 Rollout Deadline
China's National Healthcare Security Administration issued the DRG/DIP 3.0 grouping scheme for case-based and diagnosis-intervention-packet payment on September 2. Regions and medical institutions using case-based payment should complete preparation for switching to version 3.0 by December 31, 2026, and apply it in practice by the end of March 2027.
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