Preclinical Pulse: The research catching our eye in September 2026

Preclinical Pulse: The research catching our eye in September 2026 By Jules Adam Add Labiotech as your Google Preferred Source 10 minutesmins September 9, 2026 10 minutesmins Share WhatsApp Twitter Linkedin Email Photo credits: Ousa Chea Add Labiotech as your Google Preferred Source Newsletter Signup - Under Article / In Page"*" indicates required fieldsInstagramThis field is for validation purposes and should be left unchanged.Subscribe to our newsletter to get the latest biotech news!By clicking this I agree to receive Labiotech's newsletter and understand that my personal data will be processed according to the Privacy Policy.*Company name*Job title*Business email* Preclinical Pulse is Labiotech’s new monthly selection of early-stage therapies and technologies that stand out from recent biotech research. The series focuses on preclinical work backed by experimental data, from new therapeutic candidates to drug delivery systems and research platforms. In this first edition of the Preclinical Pulse, we selected seven technologies catching our eye in September 2026, from RNA interference to gene editing and protein degradation. Table of contentsCSPC Pharmaceutical Group: a long-lasting siRNA takes aim at complement in IgA nephropathy At a glance: C5-siRNA is a preclinical GalNAc-conjugated RNA interference therapy developed by CSPC Pharmaceutical Group for IgA nephropathy (IgAN). CSPC Pharmaceutical Group has developed a small interfering RNA (siRNA) designed to provide long-lasting suppression of complement component 5 (C5), a protein involved in the kidney inflammation and damage seen in IgA nephropathy (IgAN). The preclinical candidate is conjugated to N-acetylgalactosamine (GalNAc), which directs the siRNA to liver cells, where it silences the RNA encoding C5 and reduces production of the circulating protein. C5 is already a clinically validated target in IgAN, including through RNAi drug cemdisiran, but CSPC’s approach stands out for the duration of suppression reported after a single dose. In a study published in August 2026 in Molecular Therapy, t the siRNA showed high potency against C5 in vitro. A single subcutaneous injection in cynomolgus monkeys suppressed C5 by more than 80% for two months and by more than 50% for up to four months. The researchers also tested the treatment in a cynomolgus monkey model of IgAN against Nefecon, an approved targeted-release formulation of budesonide. Both improved measures of kidney function, while the C5 siRNA produced greater improvements in renal pathology and almost completely blocked complement activation in the glomeruli, the kidney structures that filter blood. The results remain preclinical, but suggest that prolonged RNAi-mediated C5 suppression could be another way of targeting complement-driven kidney damage in IgAN. The next question is whether that long duration of C5 suppression can be maintained safely as the program moves toward clinical development. Dana-Farber Cancer Institute: Slide-GoTags maps the T cells that recognize cancer At a glance: Slide-GoTags is a preclinical spatial transcriptomics platform developed by researchers at Dana-Farber Cancer Institute to identify neoantigen-specific T-cell receptors directly within patient tumors. One of the biggest challenges in personalized cancer immunotherapy is identifying which of the thousands of T cells inside a tumor are actually attacking cancer cells. Slide-GoTags looks into that problem by combining spatial transcriptomics, tumor mutation profiling, and T-cell receptor (TCR) sequencing in a single assay, for researchers to map where neoantigen-expressing tumor cells sit and which nearby T cells recognize them. More commonly, this is done by studying mutations or immune cells separately; the platform reconstructs their interactions directly within intact tumor tissue. In a Nature Biotechnology study published in July, the researchers applied Slide-GoTags to mouse models and human samples from colorectal cancer, melanoma, renal cell carcinoma, glioblastoma and ovarian cancer. The platform identified clonally expanded T cells colocalized with their cognate neoantigens, distinguished spatial immune landscapes shaped by anti-PD-1 and anti-CTLA-4 therapies, and uncovered interferon-rich “immunogenicity niches” in tumors with active immune responses. In practical terms, this could help researchers narrow down which T cells are actually involved in an anti-tumor response. While Slide-GoTags could accelerate the discovery of tumor-reactive TCRs for next-generation personalized T-cell therapies, broader validation will be needed to determine how reliably the platform can identify therapeutically relevant TCRs across different tumors. Evox Therapeutics / Karolinska Institutet: extracellular vesicles offer a new route to brain gene editing At a glance: Evox Therapeutics’ platform is a preclinical gene-editing approach that uses engineered extracellular vesicles to deliver CRISPR machinery to the brain, with Huntington’s disease as one of its potential applications. Getting gene editors beyond the liver and into organs, notably the brain, remains a major challenge for in vivo CRISPR therapies. Evox and researchers at Karolinska Institutet are exploring extracellular vesicles (EVs), naturally occurring membrane-bound particles used by cells to transport biological material, as an alternative delivery vehicle. The researchers engineered both the CRISPR components and the vesicles carrying them, and reported a roughly 300-fold increase in editing potency compared with their earlier system. They then programmed the EVs to disrupt MSH3, a DNA-repair gene involved in the expansion of the CAG repeats that contribute to Huntington’s disease progression. Suggested Articles The biggest biotech funding rounds in August 2026 Top biotech deals in August 2026 Beyond fundraising: how do venture capitals build early-stage biotechs? In the preprint published on BioRxiv in August 2026, the researchers injected the MSH3-targeting EVs directly into the caudate and putamen of two cynomolgus monkeys. Four or seven weeks later, CRISPR editing was detected across the targeted brain regions, reaching 60 to 75% in some sampled areas of the striatum. The study isn’t peer-reviewed yet and did not test whether this slowed Huntington’s disease, and the non-human-primate experiment was small, but it is early evidence that a non-viral delivery system can achieve genome editing in the primate brain. Chinese research team: blood stem cell editing in vivo with targeted LNPs At a glance: CD34/LNP^DP is a preclinical lipid nanoparticle platform developed by researchers at the Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Sciences, and collaborating institutions to deliver CRISPR gene-editing directly to blood-forming stem and progenitor cells. There is already clinical proof that editing hematopoietic stem cells can treat genetic blood disorders, but today’s approaches generally require cells to be removed from the patient, genetically modified outside the body, and then reinfused after conditioning. The researchers behind CD34/LNP^DP are exploring whether some of that process could eventually be moved directly into the body. After screening 15 lipid nanoparticle formulations, they attached an antibody targeting CD34, a marker found on hematopoietic stem and progenitor cells (HSPCs), to a lead LNP and showed that it could deliver mRNA and CRISPR/Cas components to human HSPCs. In humanized mice, the team injected the targeted LNP directly into the femur and edited an enhancer controlling BCL11A, a gene whose disruption can reactivate fetal hemoglobin. Targeting this gene is an established strategy for treating disorders such as sickle cell disease and beta-thalassemia. The blood cells maintained increased fetal hemoglobin expression over long-term follow-up. In a separate humanized mouse model carrying an ELANE mutation associated with severe congenital neutropenia, the same platform edited ELANE in human HSPCs and partially restored neutrophil development. The major translational challenge lies in delivery: the particles were injected directly into the femur, and systemic administration still largely sent the LNPs to the liver. Making bone-marrow targeting practical without direct injection will be an important next step. Penn State: biomimetic islet encapsulation could shield transplanted cells from immune attack At a glance: Biomimetic zona pellucida, or BZP, is a preclinical hydrogel encapsulation technology developed at Penn State to protect transplanted insulin-producing islets from immune rejection in type 1 diabetes. Islet transplantation can restore insulin production in people with type 1 diabetes, but donor cells are vulnerable to immune attack and generally require long-term immunosuppression. Researchers at Penn State developed BZP as an ultrathin protective coating inspired by the zona pellucida, the natural protein layer surrounding mammalian egg cells. The coating forms directly around cell clusters through aptamer-guided molecular recognition and crosslinking, producing a hydrogel capsule about 20 micrometers thick. The aim is to keep immune cells out while still allowing nutrients and insulin to diffuse across the barrier. The team encapsulated donor islets and transplanted them into immunocompetent diabetic mice without immunosuppressive treatment. Most mice receiving the encapsulated allogeneic islets maintained normal blood glucose levels for more than 100 days, while the coating preserved cell viability and function. The team also reported 100% encapsulation efficiency under physiological conditions, without the droplet-based processing used in many conventional cell-encapsulation methods. UCL Great Ormond Street Institute of Child Health: targeted Angpt1 mRNA slows WT1 kidney disease in mice At a glance: This preclinical platform, developed at UCL Great Ormond Street Institute of Child Health, uses targeted lipid nanocomplexes and renal-artery delivery to carry Angpt1 mRNA into the kidney in WT1-associated glomerular disease. WT1 mutations can cause severe childhood glomerular disease, in which damage to the kidney’s filtering structures leads to heavy protein loss in the urine and progressive scarring. With no disease-modifying treatments available, researchers at UCL Great Ormond Street Institute of Child Health developed lipid nanocomplexes designed to deliver messenger RNA specifically to podocytes and glomerular endothelial cells. The nanocomplexes target integrin αvβ3 and carry mRNA encoding angiopoietin-1, a vascular growth factor that helps maintain the glomerular filtration barrier that is reduced in the WT1 disease model. The team published a peer-reviewed study in Science Translational Medicine in August 2026. The therapy was delivered directly into the renal artery of Wt1+/R394W mice using an ultrasound-guided procedure. Three doses over four weeks reduced albuminuria, preserved glomerular endothelial cells and podocytes, and alleviated glomerular scarring. The approach also kept nanocomplex-derived protein localized in the glomeruli for up to seven days. A future challenge is whether repeated kidney-targeted delivery can be made practical enough for a chronic pediatric disease. University of Wisconsin-Madison: recyclable degraders target proteins outside the cell At a glance: McR-TAC is a preclinical targeted protein degradation platform developed at the University of Wisconsin-Madison to eliminate membrane-bound and extracellular proteins without relying on a specific lysosome-shuttling receptor. Targeted protein degradation has largely focused on proteins inside cells, while lysosome-targeting chimeras (LYTACs) have opened the door to degrading proteins on the cell surface or outside the cell. But conventional LYTACs depend on specific cell-surface receptors to pull their targets into the lysosome, and the degrader itself is generally not designed for repeated use. Researchers at the University of Wisconsin-Madison developed McR-TACs to address both limitations. The chimeras combine a target-binding ligand with a polyzwitterion that triggers macropinocytosis, allowing the target to enter the cell without relying on a particular lysosome-shuttling receptor. In a study published in Nature Biotechnology, the researchers showed that once inside the cell, McR-TACs release their target in acidic compartments before being transported back outside through an endoplasmic reticulum-Golgi pathway, allowing them to begin another degradation cycle. The platform degraded both the membrane protein PD-L1 and the extracellular protein MIF, and McR-TACs directed against either target produced durable protein degradation and inhibited tumor growth in mouse models of triple-negative breast cancer. What should we cover next month? Preclinical Pulse will return next month with a new selection of preclinical therapies, technologies and platforms that have caught our attention. If you are working on, or have come across, preclinical research that is worth featuring in a future edition, let us know. Published studies, preprints and other supporting data are particularly welcome. This article is reserved for subscribers Subscribe for free to continue reading.Enter your details to log in or subscribe. Email Company name Job title Continue Readingor Continue with Microsoft Continue with LinkedIn By continuing, I agree to receive Labiotech's newsletter and understand that my personal data will be processed according to the Privacy Policy. 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