Biomedicine Daily · Friday, 18 September 2026
CBA FRONTIER INSIGHTS
FDA Approves First-Ever Gene Therapy for Children with Sanfilippo Syndrome Type A
The FDA has approved Fayuvi (rebisufligene etisparvovec-hopf), Ultragenyx's one-time intravenous AAV9 gene therapy, as the first treatment for pediatric patients with mucopolysaccharidosis type IIIA, or Sanfilippo syndrome type A. In an open-label, single-arm multicenter study, treated patients aged 2 to 5 maintained or improved cognitive scores versus an untreated historical control. Patients receive corticosteroids from one day before infusion through at least eight weeks after, with warnings including thrombotic microangiopathy. The approval gives families affected by the rare, neurodegenerative condition a disease-directed option for the first time, and marks another advance in a busy regulatory stretch that also included accelerated approval of Bayer's sevapritinib as first-line therapy for HER2-mutant non-squamous non-small cell lung cancer.
Clinical readouts and safety
Chia Tai Tianqing HER2 Bispecific ADC TQB2102 Hits Primary Endpoint in Phase III HER2-Low Breast Cancer Trial
Chia Tai Tianqing's HER2 bispecific ADC TQB2102 has hit its prespecified primary endpoint and key secondary endpoints in the Phase III TQB2102-III-01 trial, per an interim analysis judged by an independent data monitoring committee. The trial tested the drug in HER2-low recurrent or metastatic breast cancer. The company says TQB2102 balances efficacy and safety through three core technical features.
[Articles] Switch to injectable cabotegravir–rilpivirine given every 8 weeks in adolescents living with HIV with virological suppression in sub-Saharan Africa (LATA): a randomised, open-label, multicentre, 96-week non-inferiority trial
Long-acting injectable cabotegravir–rilpivirine given every 8 weeks was superior to oral TLD for maintaining virological suppression in adolescents with HIV in sub-Saharan Africa who were already virally suppressed, with no new safety concerns observed. The LATA randomised, open-label, multicentre, 96-week non-inferiority trial supports the regimen as a maintenance option for this population.
Phase 1 Trial of AAV Gene Therapy BBM-H901 in Chinese Adolescents With Hemophilia B Completed
A Chinese phase 1 clinical trial of BBM-H901 (bopidarbicogene), an AAV liver-targeted gene therapy delivering the high-activity FIX-Padua variant, has been completed in adolescents with hemophilia B. The study was led by Zhang Lei, Hu Qun, Yang Renchi and Xiao Xiao's teams and published in Nature Medicine. BBM-H901 is the first AAV hemophilia B gene therapy product in Asia and one of three such products approved worldwide.
B7-H3 ADC Tam-Peli Cuts Death Risk by 54% in Small Cell Lung Cancer
The B7-H3-targeted antibody-drug conjugate Tam-Peli reduced the risk of death by 54% in patients with small cell lung cancer, according to findings presented at this year's World Conference on Lung Cancer. Tam-Peli is one of two B7-H3-directed ADCs for small cell lung cancer highlighted at the meeting, a cancer described as highly malignant, rapidly progressing and limited in treatment options.
HTD1801 Shows Simultaneous Benefits on Glucose, Kidney and Lipid Measures in CKM at ESC 2026
At the 2026 ESC Congress, HighTide Therapeutics' oral AMPK-NLRP3 dual-mechanism drug HTD1801 showed multi-organ effects across cardiovascular-kidney-metabolic (CKM) disease. In two phase 3 trials in type 2 diabetes it met primary endpoints, with HbA1c changes of -1.3% and -1.2% at week 24, sustained to week 52. It also lowered LDL-C and non-HDL-C, and in a glomerular hyperfiltration subgroup reduced the eGFR slope by 3.00 mL/min/1.73 m2 per year versus placebo.
Roche’s Lunsumio delivers positive confirmatory trial readout in follicular lymphoma
Regulatory and policy
Bayer’s Kerendia secures FDA nod as first drug in 30 years for Type 1 diabetes-associated kidney disease
FDA Approves First Gene Therapy for Pediatric Patients with Sanfilippo Syndrome Type A
The FDA approved Fayuvi (rebisufligene etisparvovec-hopf), a one-time intravenous AAV9 gene therapy from Ultragenyx Pharmaceutical, as the first treatment for pediatric patients with mucopolysaccharidosis type IIIA, or Sanfilippo syndrome type A. In an open-label, single-arm multicenter study, treated patients aged 2 to 5 maintained or improved cognitive scores versus an untreated historical control. Patients receive corticosteroids from one day before infusion through at least eight weeks after, with warnings including thrombotic microangiopathy.
NMPA Approves T-DXd Plus Pertuzumab for First-Line HER2-Positive Metastatic Breast Cancer
China's NMPA approved on August 11, 2026 the combination of trastuzumab deruxtecan (T-DXd) with pertuzumab as first-line treatment for adults with unresectable or metastatic HER2-positive breast cancer. The approval rests on the global DESTINY-Breast09 trial. The regimen challenges the long-standing escalation model in which patients start on trastuzumab plus pertuzumab with a taxane and switch to an ADC only after progression.
FDA Grants Accelerated Approval to Bayer's Sevapritinib for First-Line HER2-Mutant Non-Squamous NSCLC
Bayer's sevapritinib received FDA accelerated approval as first-line treatment for adults with locally advanced or metastatic non-squamous non-small cell lung cancer harboring HER2 tyrosine kinase domain activating mutations. The approval extends the drug's use from its second-line approval in November 2025, allowing oral targeted therapy from diagnosis. In the SOHO-01 trial, the first-line cohort of 69 untreated patients showed a confirmed ORR of 75%, with 6% complete and 70% partial responses.
FDA Approves Bayer's Kerendia for Chronic Kidney Disease in Type 1 Diabetes
The FDA has approved Bayer's Kerendia (finerenone) for adults with chronic kidney disease associated with type 1 diabetes. Bayer said on September 17 that this is the first fundamentally new treatment option the FDA has approved for T1D-related CKD in more than 30 years. The agency accepted Bayer's supplemental new drug application for the indication in May and granted it priority review.
FDA approves Ultragenyx’s gene therapy Fayuvi for rare neurodegenerative disorder
FDA Approves Bayer's Kerendia for Chronic Kidney Disease in Type 1 Diabetes
The FDA has approved Bayer's Kerendia (finerenone) for adults with chronic kidney disease associated with type 1 diabetes, Bayer announced on September 17, 2026. It is the first drug approved by the FDA for this indication in more than 30 years. The approval fills a long-standing treatment gap for CKD patients with type 1 diabetes.
Industry and deals
Research papers
Levetiracetam therapeutically targets GABAergic synapses in diffuse midline glioma
The antiseizure drug levetiracetam is associated with longer survival and reduced tumor growth in diffuse midline glioma, but not in hemispheric high-grade glioma. In experimental models and patient cohort data, the effect arose from selectively dampening GABAergic synaptic signaling, acting independently of the drug's canonical SV2A-mediated antiseizure mechanism. This points to a tumor-specific therapeutic use for an already available drug.
Targeting LOXL4-driven matrix stiffening with acetyldigoxin restores CD8+ T cell function in lung cancer.
Tumor-derived LOXL4 stiffens the extracellular matrix in lung cancer, activating Piezo1 in CD8+ T cells and triggering Ca2+ influx with FAK1-YAP1 signaling that drives terminal exhaustion via YBX1, ACLY and KAT2A. Conditional knockout of Piezo1, Ybx1, Acly or Kat2a in murine CD8+ T cells abolished stiffness-induced exhaustion and suppressed tumor growth. Acetyldigoxin, identified by molecular docking as a high-affinity LOXL4 inhibitor among FDA-approved agents, softened the ECM, reversed CD8+ T cell exhaustion and synergized with anti-PD-1 blockade for durable tumor regression.
Population-Scale Precision Safety in Oncology Reveals Clinical and Genetic Determinants of Systemic Therapy Toxicity
A large language model-based resource, MSK-Tox, captured the incidence, timing, and grade of anti-cancer therapy toxicity across more than 50,000 patients. Analysis of six representative adverse events showed toxicity risk varies by cancer type, regimen, and clinical context, is predictable from pretreatment clinical features, and has two germline modes: an organ-intrinsic mode, exemplified by a regulatory variant near FOXE1 linked to hypothyroidism, and an immune-mediated mode in which HLA-DRB1*15 drives adrenal insufficiency after immune checkpoint inhibition. The work supports pretreatment risk assessment for harm on the same principles used to predict benefit.
Myeloid reprogramming generates myeloid-derived activating cells to potentiate radiotherapy and suppress metastasis
Radiotherapy was found to drive a TET2-dependent epitranscriptomic program in myeloid cells that promotes differentiation into immunosuppressive M-MDSCs. Loss of TET2 preserved caRNA m5C-MBD6 signaling, restored IRF3-IFN-I activation, and generated myeloid-derived activating cells that strengthen radiotherapy-induced antitumor immunity. The work identifies a myeloid reprogramming strategy to potentiate radiotherapy and suppress metastasis.
Beyond genes and hotspots: Protein-informed interpretation of cancer mutations
A framework for interpreting cancer mutations maps significantly mutated regions onto protein domains and structural features, extending analysis beyond single genes and recurrent hotspots. The work by Hyeon et al., highlighted in Cancer Cell, establishes proteo-genomics as a way to assign biological and clinical meaning to somatic mutations across cancer genomes. It offers clinicians and researchers a structural lens for reading variants that gene-level interpretation misses.
De novo-engineered guide RNA-directed transposition with TnpB-family proteins
TnpB-family proteins have been engineered into minimal de novo RNA-guided transposition systems. Nuclease-deficient TnpB-family proteins mediate three-component DNA integration with high efficiency, target-site immunity, orientation control, and programmable insertion into the bacterial chromosome. Mutations at the TniQ-TnsC interface in wild-type V-K CAST also attenuate off-site transposition.
STAT+: Why do ‘never-smokers’ get lung cancer? In some cases, rare genetic variant may be a factor
A very rare genetic variant is associated with 25-fold higher odds of lung cancer in people who never smoked, researchers reported in Science. The variant is far more common in Southern Appalachia than elsewhere in the U.S. The finding, drawn from genetic data including 23andMe records, points to distinct risk factors that could shape screening and treatment for never-smokers.
Immune checkpoint knockout screen reveals TIGIT-CD155 interaction as a significant modulator of adapter CAR-T cell function in acute myeloid leukemia
A CRISPR/Cas9 knockout screen of immune checkpoint receptors in adapter CAR (AdCAR)-T cells identifies TIGIT disruption as a potent enhancer of cytotoxicity against AML. TIGIT knockout boosted AdCAR-T killing across five leukemia and lymphoma cell lines, whereas PD-1 disruption did not significantly improve killing of MOLM-13 wildtype cells. Pharmacological TIGIT blockade matched the knockout effect, and PD-1 blockade outperformed PD-1 gene disruption. The work points to the TIGIT-CD155 interaction as a key modulator of AdCAR-T function in AML.
Methods and resources
Validating LLM judges for automated oversight of patient communication
LLM judges match clinician ratings for safety screening and factual verification of patient communication about trial informed consent (Spearman ρ > 0.80), but agree less on communication quality (ρ < 0.60). Using a seven-criterion rubric validated on ICON-Bench, 19 LLM judges were benchmarked; safety-specialized guard models underperformed general-purpose models, and patient advocates rated communication quality lower than both clinicians and LLM judges. The results support LLM judges for scalable oversight, with recalibration needed to patient-centered standards.