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Researchers Develop New GPCR Mapping Method to Uncover Hidden Drug Targets for Various Diseases

Researchers Develop New GPCR Mapping Method to Uncover Hidden Drug Targets for Various Diseases

Researchers at the Centre for Genomic Regulation, led by Taylor Mighell and Ben Lehner, have developed a novel method for identifying druggable sites on G protein-coupled receptors (GPCRs). GPCRs are a family of proteins located on cell surfaces and are targets for approximately one-third of all regulatory-approved medicines globally. The new method creates detailed functional maps of these receptors, aiming to facilitate the development of treatments with fewer side effects for conditions ranging from heart disease to depression. The team constructed a detailed map of the beta-2 adrenergic receptor, which is involved in relaxing airways and is a target for asthma and COPD inhalers. They synthesized 7,500 variants of this protein, introduced each into human cells, and evaluated them across different drug doses, generating over 150,000 measurements. This extensive dataset revealed previously hidden potential drug targets within the receptor, including adjustable regions that can be modified to fine-tune rec...

Mammoth Biosciences Presents Positive Preclinical Data for CRISPR Gene Editing Therapy MB-111 for Hypertriglyceridemia

Mammoth Biosciences Presents Positive Preclinical Data for CRISPR Gene Editing Therapy MB-111 for Hypertriglyceridemia

Mammoth Biosciences, a biotechnology company, recently presented new preclinical data for its lead developmental candidate, MB-111, at the American Heart Association Scientific Sessions held in Chicago. MB-111 is an in vivo CRISPR gene editing therapy designed as a potential single-dose treatment to durably reduce plasma triglyceride levels in patients with persistent chylomicronemia (PC). The therapy utilizes CasPhi, an ultracompact CRISPR nuclease, delivered via a lipid nanoparticle to disrupt the APOC3 gene, which regulates triglyceride metabolism in the liver. Key findings from the preclinical studies include dose-dependent reductions of apoC-III protein and serum triglycerides by up to 95% in a humanized transgenic mouse model. Additionally, a single intravenous dose in non-human primates (NHPs) achieved up to a 90% reduction in APOC3 mRNA and suppression of apoC-III protein for nine months post-treatment. The studies also indicated a favorable tolerability profile, with modest, transient elevations i...

Mount Sinai Researchers Uncover New Drug Targets for Rare Metabolic Disease GA1

Mount Sinai Researchers Uncover New Drug Targets for Rare Metabolic Disease GA1

Researchers at the Icahn School of Medicine at Mount Sinai have made a significant breakthrough in understanding glutaric aciduria type 1 (GA1), a rare inherited metabolic disorder. Their study, published in Science Advances, provides the first structural map of the complete AASS enzyme and identifies mechanisms to control its activity. GA1 is caused by mutations in the GCDH gene, leading to the accumulation of toxic metabolites that can cause irreversible brain damage. Current treatments, including newborn screening and dietary restrictions, have improved outcomes but still struggle to prevent neurological injury. The Mount Sinai team focused on substrate reduction therapy, aiming to prevent harmful metabolites from forming. They identified AASS (alpha-aminoadipic semialdehyde synthase) as a promising target because it controls the initial steps of lysine degradation, a key amino acid involved in GA1. Previous studies in 2020 and March 2026 demonstrated that genetically removing AASS reduced toxic metabol...

Regeneron's Baloncibart Antibody Shows Promise in Reducing Heart Rate Spikes for POTS Patients, Advancing to Phase 3 Trials

Regeneron's Baloncibart Antibody Shows Promise in Reducing Heart Rate Spikes for POTS Patients, Advancing to Phase 3 Trials

Regeneron Pharmaceuticals' investigational monoclonal antibody, baloncibart (REGN7544), has demonstrated effectiveness in reducing orthostatic heart rate in patients suffering from postural orthostatic tachycardia syndrome (POTS). Results from the Phase 2 Pinnacle trial, presented at the American Autonomic Society annual meeting in San Diego, showed that a single 450-mg injection of baloncibart led to an average heart rate drop of 11.4 beats per minute compared to placebo at day eight. A 150-mg injection also reduced heart rate by 9.5 beats per minute. The higher dose maintained a significant decrease in heartbeat at day 29, along with improvements in physical functioning and daily living activities as reported by patients. The drug was generally well tolerated, with most treatment-emergent adverse events being mild or moderate. No serious adverse events led to discontinuation from the study. Regeneron plans to initiate late-stage testing for baloncibart in the first half of next year, aiming to address th...

United States Leads Global CRISPR Gene Editing Trials with 356 Studies Underway

United States Leads Global CRISPR Gene Editing Trials with 356 Studies Underway

The United States is at the forefront of CRISPR gene editing research, leading globally in the number of clinical trials. According to the CMN Clinical Trials Q3 2026 Global Report, there are 356 gene-editing trials worldwide as of September 30, 2026. The U.S. and China collectively account for the majority of these trials, with other significant contributions from Australia, the United Kingdom, Canada, and New Zealand. This report, compiled by CMN Intelligence, utilizes a manually curated global database of clinical gene-editing trials to provide a comprehensive overview of the landscape. The data also delves into various aspects of these trials, including editing approaches, cell sources, and delivery technologies being employed across the global clinical gene-editing landscape. This robust activity signifies a rapid advancement in the application of gene-editing technologies for therapeutic purposes.

GeneDx Research Reveals New Microcephaly Gene Candidates Through Large-Scale Genomic Data Analysis

GeneDx Research Reveals New Microcephaly Gene Candidates Through Large-Scale Genomic Data Analysis

GeneDx researchers have utilized their extensive Infinity™ dataset, the world's largest rare disease genomic dataset, to conduct a large-scale gene-based burden analysis focusing on microcephaly. This study, presented at the 2026 American Society of Human Genetics (ASHG) Annual Meeting, involved analyzing genetic data from 25,779 individuals with microcephaly and comparing it against two control groups comprising 476,448 unrelated parents and 418,542 non-microcephaly probands. The research successfully identified several established microcephaly-associated genes, including DYRK1A, KIF11, ASPM, FOXG1, DDX3X, TUBA1A, and WDR62. Crucially, the analysis also pinpointed two new promising candidate genes, ETF1 and IGF2BP1, which showed strong statistical associations across both control comparisons. While these candidate associations require further validation, the study demonstrates the potential of using large-scale genomic data to systematically investigate gene-disease associations and uncover new genetic in...

Indiana Organizations Launch Provena Consortium for Clinical and Genomic Data Resource

Indiana Organizations Launch Provena Consortium for Clinical and Genomic Data Resource

A coalition of Indiana-based organizations has launched the Provena Consortium, a new clinical and genomic data resource. This consortium connects academic and industry researchers with genomic data linked to longitudinal clinical data from over 90,000 consented participants. These participants have also agreed to be re-contacted for future studies. The founding partners include Eli Lilly and Co., Indiana University Health (IU Health), Indiana University (IU), the Indiana Health Information Exchange, the CEOs of Indiana Corporate Partnership (CICP), the Indiana Biosciences Research Institute, Purdue University, and the Regenstrief Institute. The consortium is expanding rapidly, adding over 3,500 participants each month, with a goal to reach 250,000 genotyped, clinically characterized, and recallable participants. This initiative is supported by grants from Lilly Endowment Inc. and a significant investment from IU, including $6.8 million for advanced sequencing equipment and infrastructure.

Guardant Health Introduces New Epigenomic Applications for Enhanced Cancer Treatment Selection

Guardant Health Introduces New Epigenomic Applications for Enhanced Cancer Treatment Selection

Guardant Health, Inc. has announced new epigenomic applications for its Guardant360® Liquid blood test, designed to provide oncologists with deeper insights for cancer treatment decisions, particularly in the absence of traditional biomarkers. These expanded capabilities, powered by the company's InfinityAI platform, integrate genomics and epigenomics to uncover more signals of tumor biology. The new applications aim to inform therapy selection in breast and lung cancers and identify additional treatment opportunities. Specifically, they include two novel epigenomic predictors for advanced breast cancer patients who may benefit from selective estrogen receptor degraders (SERDs) and for advanced non-small cell lung cancer (NSCLC) patients considering immune checkpoint inhibitor (ICI) monotherapy. The Guardant360 Liquid will also offer epigenomic identification of PD-L1 negative patients without requiring a tissue sample. Furthermore, these epigenomic features can identify ALK fusions and MTAP deletions that...

Scribe Therapeutics Reports Q3 2026 Financial Results and Advances Gene-Editing Pipeline

Scribe Therapeutics Reports Q3 2026 Financial Results and Advances Gene-Editing Pipeline

Scribe Therapeutics Inc. (Nasdaq: SCTX), a clinical-stage biotechnology company, has released its financial results for the third quarter ended September 30, 2026, alongside updates on its corporate and pipeline developments. The company reported cash, cash equivalents, and marketable securities totaling $174.2 million as of September 30, 2026, an increase from $43.0 million on June 30, 2026. This rise was primarily driven by approximately $140.2 million in net proceeds from its July 2026 Initial Public Offering (IPO) and a concurrent private placement. Scribe Therapeutics also announced continued enrollment in the first-in-human Phase 1 clinical trial for STX-1150, its epigenetic silencing therapy aimed at lowering LDL-C, with initial clinical data anticipated in the first half of 2027. Additionally, the company expanded its strategic collaboration with Sanofi to include Scribe’s ELXR epigenetic silencing technology, further validating its platform innovation. Benjamin Oakes, Ph.D., co-founder and Chief E...

Precision Medicine Advances with Focus on Individualized Treatments and Genetic Data

Precision Medicine Advances with Focus on Individualized Treatments and Genetic Data

Precision medicine is an evolving field that utilizes advanced tools and technologies to customize medical treatments for individual patients. This approach considers a patient's unique genetic, environmental, and lifestyle profiles. By analyzing genetic data, medical researchers are developing targeted treatments that aim to be more effective than conventional, generalized medicines. Researchers are actively exploring new avenues within precision medicine, including genetic testing and personal genomics. The field is also addressing the challenge of scaling highly personalized therapies beyond single patients, drawing parallels with existing precision medicine models in oncology. The Precision Medicine World Conference (PMWC) Pioneer Award highlights research contributing to new cancer treatment options, emphasizing a continuous cycle of measurement, learning, and refinement to enhance the safety, practicality, and patient burden of new treatments.

Helix Launches Research Access Program to Provide Clinicogenomic Data to Academic Researchers

Helix Launches Research Access Program to Provide Clinicogenomic Data to Academic Researchers

Helix, a leading enterprise precision medicine company, has launched its Research Access Program, inviting academic and nonprofit researchers to apply for secure access to linked clinicogenomic data from the Helix Research Network®. This program provides access to North America’s largest available clinicogenomic dataset through Helix’s Research Workspaces, a Trusted Research Environment (TRE). The launch follows new clinical research presented at the American Society of Human Genetics (ASHG) 2026 Annual Meeting, which utilized data from the same network. One study demonstrated that population genomic screening can drive earlier preventive care for common, actionable conditions like Hereditary Breast and Ovarian Cancer (HBOC), Lynch Syndrome, and Familial Hypercholesterolemia. Another study highlighted how rare genetic variants might influence responses to GLP-1 medications.

Human Phenotype Ontology Improves Genetic Diagnosis in Neurodevelopmental Disorders

Human Phenotype Ontology Improves Genetic Diagnosis in Neurodevelopmental Disorders

A human phenotype ontology (HPO) approach is being utilized to enhance genetic diagnosis in neurodevelopmental disorders (NDDs). Genetic testing is a powerful tool, but its limitations necessitate careful selection and re-evaluation to ensure accurate diagnoses. Despite advancements in DNA analysis, thousands of rare genetic diseases remain undiagnosable. The HPO system provides a structured vocabulary for describing clinical features, improving diagnostic precision by integrating standardized phenotypic descriptions with genomic data. HPO terms are incorporated into web-based gene prioritization tools to help rank candidate genes for Mendelian disorders. The aim is to provide clinically interpretable evidence that HPO terms differ in their potential to narrow diagnostic yield and to support more deliberate selection and communication of HPO terms. This approach helps classify phenotypic features systematically, prioritizing major phenotypic variants over minor ones to streamline the diagnostic process and...