Drug design, drug delivery and technologies

Preclinical data support Evaxion’s platform behind CMV vaccine candidate

Tue, 14 Jul 2026 09:00:00 -0400

Human cytomegalovirus (CMV) is a clinical challenge in infants and immunocompromised individuals, since no licensed vaccine exists. Vaccine development is complex due to several factors, including viral latency, genomic complexity and immune evasion, among others. Evaxion A/S recently presented data on its proprietary AI-immunology platform used to create the EVX-V1 vaccine for CMV infection.

AI co-scientist performs biomedical research ‘at expert level’ in less time

Fri, 10 Jul 2026 09:00:00 -0400

While biomedical resources in the form of specialized tools, hundreds of thousands of published papers and huge repositories of ‘omics, health records and other data, are growing exponentially, discovery is getting slower and more expensive. That is the perspective from which scientists at Stanford University approached the development of their artificial intelligence (AI) research assistant Biomni.

AI co-scientist performs biomedical research ‘at expert level’ in less time

Thu, 09 Jul 2026 14:00:00 -0400

Insilico Medicine and Takeda collaborate on drug discovery

Fri, 03 Jul 2026 09:00:00 -0400

Insilico will lead AI-driven discovery to identify molecules meeting predefined scientific and early development criteria, while Takeda will apply its global development capabilities to advance selected candidates through clinical validation.

Artan closes seed round to support tRNA platform

Thu, 02 Jul 2026 09:00:00 -0400

Artan Biotechnologies LLC has closed a $1 million seed round to support progression of the company’s proprietary mutation-specific codon suppression platform toward first-in-human development.

CINP 2026: organoids reveal autism and addiction mechanisms

Wed, 01 Jul 2026 09:00:00 -0400

At the 2026 World Congress of Neuropsychopharmacology (CINP), held in Glasgow June 26-29, 2026, researchers from Japan’s National Center of Neurology and Psychiatry (NCNP) showcased how human organoid technologies are reshaping the study of neurodevelopmental vulnerability, addiction and psychiatric disorders.

CDW reports in vivo results for LNP-LANFA

Tue, 30 Jun 2026 09:00:00 -0400

A Biotech Co. Ltd., a subsidiary of CDW Holding Ltd., and Neoregen Biotech Co. Ltd. have obtained promising results from in vivo murine testing of LNP-LANFA, a next-generation lipid nanoparticle (LNP) technology under joint development.

ENCALS 2026: From genetics to advancing strategies against ALS

Fri, 26 Jun 2026 09:00:00 -0400

Amyotrophic lateral sclerosis (ALS)-associated genes provide direct therapeutic targets and reveal pathways that can be used to develop treatments that counteract their harmful molecular effects. Because the underlying causes of most ALS cases remain unknown, identifying disease-associated variants is essential to uncover the mechanisms that drive the disease, as shown at the European Network to Cure ALS (ENCALS) meeting, held in Madrid from June 24 to 26, 2026.

AI uncovers hidden antibiotic potential in prion proteins

Thu, 25 Jun 2026 09:00:00 -0400

Some proteins embedded in the structure of prions may have antimicrobial activity, according to a study led by scientists at the University of Pennsylvania. An AI analysis of millions of fragments from prion-related proteins has revealed more than a thousand peptides that disrupt bacterial membranes and reduce infection in animal models, suggesting these proteins could be an unexpected source of new antibiotics.

Going after gonorrhea with artificial intelligence

Tue, 23 Jun 2026 09:00:00 -0400

Researchers at the Wyss Institute of Harvard University have used a mix of machine learning and classical screening techniques to identify small molecules that were effective against Neisseria gonorrhoeae in vagina-on-a-chip and mouse models of infection. Their findings were published in the June 17, 2026, issue of Science Translational Medicine.

Going after gonorrhea with artificial intelligence

Fri, 19 Jun 2026 09:00:00 -0400

Signal rewiring turns ovarian cancer against itself

Tue, 16 Jun 2026 09:00:00 -0400

Harnessing an oncolytic signal and redirecting it against the tumor itself could be developed as a selective strategy for certain cancer types, as occurs with ErbB hyperactivity, a form of signaling that drives many carcinomas. Inspired by this idea, scientists at Stanford University have engineered a virus that replicates only in ErbB-hyperactive ovarian cancer cells. This allowed them to precisely kill this specific tumor population, achieving greater efficacy and safety than previous oncolytic viruses.

Skape Bio unlocks GPCR targets with de novo-designed miniproteins

Mon, 08 Jun 2026 09:00:00 -0400

Modulating G protein-coupled receptors (GPCRs) is one of the major challenges in biomedicine. These are flexible proteins with small, deep binding pockets. The scientific community has explored small molecules, antibodies and nanobodies to develop ligands. Skape Bio Inc. is betting on creating miniproteins, a strategy that brings precise solutions for different functions.

Lipopolyplex-delivered, DISE-inducing small RNAs as broad-spectrum anticancer therapy

Fri, 05 Jun 2026 09:00:00 -0400

Previous work found that certain short RNAs can induce cell death in a RISC-dependent fashion by targeting several networks of survival genes simultaneously, therefore triggering multiple cell death pathways. This form of cell death was named death induced by survival gene elimination, or DISE, an effect that depends on a toxic 6-mer seed.

AxS007 penetrates BBB for breast cancer brain metastases

Fri, 05 Jun 2026 09:00:00 -0400

About 90% of brain metastases are often limited therapeutically speaking due to the impermeable blood-brain barrier (BBB). Nanocarry Therapeutics Ltd. has presented AxS007, a novel insulin-mediated nanocarrier that delivers multiple copies of trastuzumab and pertuzumab across the BBB, using native insulin as a brain transporter and increasing brain exposure.

Conexeu Sciences launches preclinical program for bioregenerative breast matrix

Thu, 28 May 2026 09:00:00 -0400

Conexeu Sciences Inc. has launched a preclinical development program for B.R.E.A.S.T. (Bio-Regenerative Ergonomically Architected Smart Tissue), its 3D-printed bioregenerative breast matrix, at the Wake Forest Institute for Regenerative Medicine (WFIRM), a leading regenerative medicine research organization.

Protuoso Biosciences building multifunctional protein engineering platform

Thu, 28 May 2026 09:00:00 -0400

Protuoso Biosciences has announced the close of an oversubscribed $9.5 million seed financing round, the proceeds of which will be used to advance its multifunctional protein engineering platform and broad pipeline across cardiometabolic, oncology and autoimmune diseases.

Researchers identify innate immune barrier against melanoma

Tue, 26 May 2026 09:00:00 -0400

Australian researchers have identified a previously overlooked population of immune cells in the skin that physically restrain melanoma growth by engulfing live melanoma cells, and the discovery could reshape thinking around macrophage-targeted cancer therapies and innate immunity in oncology.

‘Detargeted’ targeted gene therapy improves activity in Pompe

Wed, 20 May 2026 09:00:00 -0400

A new strategy aims to improve gene therapy for Pompe disease by optimizing both the genetic component that restores the function of a deficient lysosomal enzyme and the vector that delivers it to the target tissue while avoiding the liver. The findings suggest that combining an optimized transgene with a targeted capsid could significantly enhance the effectiveness of gene therapy for Pompe disease.

Launch of CGTxchange to reactivate cell and gene therapy programs

Fri, 15 May 2026 09:00:00 -0400

The American Society of Gene & Cell Therapy (ASGCT) and Orphan Therapeutics Accelerator (OTXL) have announced the public launch of CGTxchange, an AI-enhanced clearinghouse and marketplace built to help reactivate cell and gene therapy programs that have been shelved despite strong scientific and clinical evidence.

ASGCT 2026: Directed evolution in gene therapy

Fri, 15 May 2026 09:00:00 -0400

Directed evolution has become a central pillar in gene therapy. This engineering strategy enables the generation of more efficient variants of genetic editors and delivery vectors. Molecular diversification methods are increasingly sophisticated and are now accelerated by machine learning and AI tools, as showcased at the 29th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) held in Boston this week.

ASGCT 2026: Uncovering the mechanisms of AAV toxicity

Thu, 14 May 2026 09:00:00 -0400

Gene therapies rely on vectors to reach the target tissue where they act, such as adeno-associated viruses (AAVs) or lipid nanoparticles (LNPs), among other delivery strategies. Each combination is optimized for a specific cell type and indication, aiming to overcome challenges such as efficacy, specificity and toxicity. On May 13, 2026, two sessions included in the scientific symposia of the 29th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), being held in Boston this week, addressed AAV-related toxicities, which have led to fatal cases in clinical trials and remain an area for improvement in approved therapies.

ASGCT 2026: Circular RNA, the new beast in gene and cell therapy

Wed, 13 May 2026 09:00:00 -0400

Circular RNA (circRNA) is not a new concept, but it is a novel strategy in the field of gene and cell therapy. While mRNA vaccines have revolutionized medicine, this RNA fragment without free ends surpasses their performance in both efficacy and durability, bringing it to the attention of several pioneering companies. The latest advances in circRNA presented at the 29th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) clearly surpass the performance achieved with linear mRNA.

Two-step HIV vaccine induces broadly neutralizing antibodies

Tue, 12 May 2026 09:00:00 -0400

A designed chimeric virus induced broadly neutralizing antibodies against the macaque equivalent of HIV. The strategy works in two steps: first it uses an envelope protein with a mutation that reduces the glycan shield that makes it invisible to the immune system, and then it exposes the part of the protein most likely to generate these antibodies capable of blocking many variants of the virus. The macaques developed potent and diverse antibodies with this approach, which pave the way for the development of an HIV-1 vaccine.

New assay for drug discovery against chemo-induced peripheral neuropathy

Fri, 08 May 2026 09:00:00 -0400

A group led by researchers at Boston Children’s Hospital established a scalable and reproducible model of paclitaxel-induced axon degeneration and neurotoxicity in human induced pluripotent stem cell (iPSC)-derived sensory neurons.

Two-step HIV vaccine induces broadly neutralizing antibodies

Fri, 08 May 2026 09:00:00 -0400

A designed chimeric virus induced broadly neutralizing antibodies (bNAbs) against the macaque equivalent of HIV. The strategy works in two steps: first it uses an envelope protein (Env) with a mutation that reduces the glycan shield that makes it invisible to the immune system, and then it exposes the part of the protein most likely to generate these antibodies capable of blocking many variants of the virus. The macaques developed potent and diverse antibodies with this approach, which pave the way for the development of an HIV-1 vaccine.

First measles treatment advances as vaccination rates drop

Thu, 07 May 2026 11:00:00 -0400

Scientists at the La Jolla Institute for Immunology have identified and characterized human antibodies that neutralize the measles virus by blocking its entry into the cell. This is the first time that antibodies have been shown to bind effectively to two essential viral proteins, creating a dual blockade that prevents infection. Unlike the current vaccine, which is based on an attenuated virus and is not recommended for immunocompromised individuals, these monoclonal antibodies could be used both as a new vaccine approach and as a treatment for the entire population.

MYO technology enables DNA delivery of incretin agonists for durable weight loss

Wed, 06 May 2026 09:00:00 -0400

Researchers from Renbio Inc. and Louisiana State University investigated the delivery of plasmid DNA encoding new glucagon-like peptide-1(GLP-1)-based biologics with MYO (Make Your Own) technology as a new therapeutic strategy. This approach ensures the continuous production of GLP-1-based biologics for an extended period, overcoming the need for weekly dosing.

Liposomes displaying Env trimers drive HIV apex-focused responses

Mon, 04 May 2026 09:00:00 -0400

A new vaccination strategy designed to induce antibodies that recognize the apex of the HIV Env protein uses Env trimers displayed on liposomes to increase their density and orient them correctly. This presentation enhanced apex-focused antibody responses in macaques, and the monoclonal antibodies isolated after immunization showed binding modes and structural features resembling human broadly neutralizing antibodies (bNAbs), indicating that the vaccine can steer the antibody response toward this vulnerable site.

Quintuple GLP-1-GIP-PPAR agonist for obesity and diabetes control

Thu, 30 Apr 2026 09:00:00 -0400

A new molecule combines the action of two incretins, GLP-1 and GIP, hormones that regulate glucose and appetite, with lanifibranor, a triple agonist of peroxisome proliferator activated receptors (PPAR α/γ/δ). GLP-1-GIP-Lani enables targeted delivery of the PPAR agonist to cells that express incretin receptors, enhancing weight loss, improving glucose control and reducing inflammation in obese mice. In these models, it surpassed the effects of GLP-1 receptor agonists such as semaglutide and GLP-1-GIP co-agonists such as tirzepatide in reducing body weight, improving glycemic control and enhancing metabolic outcomes during active treatment.