One-time base editing lowers cholesterol in phase 1 study
Verve Therapeutics has reported phase 1 data in The New England Journal of Medicine for VERVE-102, an investigational in vivo adenine base-editing therapy designed to durably inactivate PCSK9 in the liver. The one-time treatment consists of mRNA encoding an adenine base editor and a guide RNA targeting PCSK9, delivered in a GalNAc-containing lipid nanoparticle, and is being developed for adults with heterozygous familial hypercholesterolemia or premature coronary artery disease.
In the open-label, single-ascending-dose study, 35 participants received VERVE-102 across six dose cohorts. No dose-limiting toxicities were reported, while mild-to-moderate infusion reactions and transient liver enzyme elevations were observed. The therapy produced dose-dependent mean reductions in PCSK9 of up to 88 percent and LDL-C reductions of up to 62 percent at the highest dose, with effects appearing durable through follow-up of at least one year in 15 participants. Source
Experimental gene therapy protects neurons from TDP-43 damage
UC San Diego researchers have reported preclinical data for SynCav1, an experimental systemically delivered gene therapy designed to protect neurons from TDP-43-related neurodegeneration. The therapy uses a modified viral vector to deliver the SynCav1 gene, increasing production of caveolin-1, a neuroprotective protein involved in organizing key neuronal signaling pathways.
In mouse models, SynCav1 crossed the blood-brain barrier, increased caveolin-1 expression across the brain and spinal cord, and preserved learning, memory, and fear extinction. The treatment also reduced pathological TDP-43 levels in the cortex and hippocampus and protected cellular structures involved in neuronal communication and mitochondrial function. The researchers say the approach could have potential across TDP-43-related diseases, including frontotemporal dementia, Alzheimer’s disease, and ALS. Source
Lilly acquires Engage Bio to expand non-viral genetic medicines
Eli Lilly has acquired Engage Bio, a preclinical biotechnology company developing non-viral DNA delivery technologies for genetic medicines. Engage’s Tethosome platform is designed to improve the potency, tolerability, and redosability of DNA-based therapies by combining engineered DNA payloads with lipid nanoparticle delivery and mRNA-encoded technology intended to enhance localization and expression.
The deal brings Engage’s non-viral DNA delivery platform into Lilly’s broader genetic medicines portfolio. Unlike viral vectors, the approach is designed to retain the durability and programmability of DNA-based therapies while addressing challenges such as nuclear localization and innate immune sensing. Lilly acquired Engage for up to $202 million in cash, including upfront and development milestone payments. Source
King’s spinout advances tumour-activated CAR-T platform
King’s College London has spun out Korecyte Bio with investment and support from Cell and Gene Therapy Catapult to develop HypoxiCAR, a tumour-activated CAR-T cell therapy platform for solid cancers. The modality is an engineered CAR-T approach designed to activate only in low-oxygen environments, a hallmark of many solid tumours, while remaining inactive in healthy tissues.
The platform aims to reduce off-target toxicity, improve anti-cancer potency, and open up new CAR-T targets in solid tumours, where cell therapies have so far had limited success. CGT Catapult is supporting Korecyte through seed investment from the Innovate UK Cross-Catapult Investment Pilot, as well as development and commercialisation expertise to help prepare the company’s lead candidate for clinical translation. Source
United Immunity adds macrophage assets for in vivo CAR-M platform
United Immunity has acquired macrophage-related assets from Carisma Therapeutics to support development of in vivo engineered macrophage therapies for cancer and liver fibrosis. The company plans to combine the assets with its PEG-free, pullulan-coated lipid nanoparticle delivery system, known as P-LNP, to generate in vivo CAR-M and other genetically engineered macrophage approaches.
The platform is designed to target myeloid cells directly in the body, potentially enabling next-generation macrophage-based therapies without ex vivo cell manufacturing. United Immunity says the integrated approach will be used to advance programs for solid tumors and fibrotic diseases, including liver fibrosis. Source
Cartesian secures financing ahead of phase 3 CAR-T readout
Cartesian Therapeutics has secured up to $150 million in non-dilutive financing from K2 HealthVentures to support development and potential launch preparation for Descartes-08, an autologous anti-BCMA mRNA CAR-T cell therapy for autoimmune diseases. The initial $50 million tranche is expected to extend Cartesian’s cash runway into 2028.
Descartes-08 is being developed as an outpatient-administered CAR-T therapy without preconditioning chemotherapy. Topline data from the phase 3 AURORA trial in myasthenia gravis are expected in Q1 2027, with a BLA filing planned for mid-2027. Cartesian also expects data in 2027 from phase 2 TRITON in myositis and phase 1/2 HELIOS in juvenile dermatomyositis. Source
Scribe cleared to start first-in-human PCSK9 silencing study
Scribe Therapeutics has received clearance from Australia’s Therapeutic Goods Administration to begin a first-in-human study of STX-1150, an investigational in vivo CRISPR-based epigenetic silencing therapy for LDL-C reduction. The liver-targeted therapy is designed to durably silence PCSK9 without permanently altering DNA, using Scribe’s ELXR platform delivered by a lipid nanoparticle.
The planned phase 1 trial will evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics in up to 64 adults with elevated LDL-C and increased cardiovascular risk across sites in Australia and New Zealand. STX-1150 consists of mRNA encoding an engineered ELXR and a guide RNA targeting PCSK9, with the aim of delivering long-lasting LDL-C lowering after a single dose. Source
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