Genetics & Epigenetics Library
Every article, presentation, spotlight, and news item we've tagged to Genetics & Epigenetics.
Showing 49–72 of 176
Epigenetic therapy achieves durable muscle gains without editing DNA
Epicrispr closed a $90 million Series C funding round to advance EPI-321, an epigenetic therapy for facioscapulohumeral muscular dystrophy that silences disease-causing genes without altering DNA sequence. Early Phase 1/2 data showed durable muscle improvements following a single intravenous dose, positioning epigenetic modulation as a distinct therapeutic approach from gene editing.
GenSight Biologics raises nearly €1.7M to support gene therapy programs
GenSight Biologics secured €1.7 million in funding to advance gene therapy programs targeting inherited retinal and mitochondrial diseases, with lead support from existing shareholders Advent France Biotechnology and RA Capital Management. The capital will fund clinical development of GS010/LUMEVOQ for Leber hereditary optic neuropathy and operational milestones through 2026.
AIRNA doses first patient in RNA-editing trial
AIRNA has initiated Phase 1 testing of AIR-001, an RNA-editing therapy for alpha-1 antitrypsin deficiency (AATD), a genetic condition causing progressive lung and liver damage. Unlike permanent gene editing, RNA editing allows for repeated dosing and adjustment, positioning the approach as a reversible, titratable intervention aligned with how chronic disease and aging actually progress.
Life Biosciences lands $80m to push cell reset therapy
Life Biosciences secured $80 million to advance ER-100, the first partial epigenetic reprogramming therapy entering human trials, designed to restore cellular function in age-damaged neurons. This represents a shift in aging intervention from symptom management toward addressing the underlying cellular decline that drives multiple age-related diseases.
Select Small Non‐Coding RNAs Are Determinants of Survival in Older Adults
Circulating small RNAs, particularly nine piRNAs, predict two-year survival in older adults with greater accuracy than age and clinical factors alone, with experimental evidence suggesting reduced piRNA levels associate with extended lifespan. These findings identify specific small RNA signatures as measurable biomarkers and potential therapeutic targets for longevity interventions.
Cardiac Fibrosis Reversal: RNA Therapy Targets Aging Cell Behavior
HAYA Therapeutics received FDA Fast Track designation for HTX-001, an RNA-based therapy designed to reprogram fibroblasts and reverse cardiac fibrosis rather than manage heart disease symptoms. This regulatory milestone signals a shift toward cellular reprogramming as a mechanism for addressing age-related organ stiffening and dysfunction.
Macrophage Reprogramming via LNP-mRNA Targets Fibrotic Disease
Resolution Therapeutics is systematically evaluating lipid nanoparticle delivery systems paired with mRNA payloads designed to reprogram myeloid cells toward anti-inflammatory phenotypes. This dual-platform approach—combining in vivo LNP optimization with their Phase 1/2 autologous regenerative macrophage therapy—targets a fundamental driver of age-related disease: chronic inflammatory fibrosis.
Genflow reveals early data from SIRT6 gene therapy trial in dogs
Genflow's SIRT6 gene therapy demonstrated superior survival and functional improvements in aged beagles across multiple endpoints, including muscle preservation, frailty reduction, and quality of life measures. This interim data from a proof-of-concept trial establishes preliminary evidence that SIRT6 activation may mitigate age-related functional decline, with potential implications for human therapeutic development.
PST-611 Phase 1 data shows inflections in geographic atrophy growth
PST-611, a transferrin-encoding gene therapy targeting iron dysregulation in dry age-related macular degeneration, demonstrated safety and tolerability in a six-patient Phase 1 trial with encouraging signals of slowed geographic atrophy progression. The therapy addresses a mechanistic pathway implicated in retinal degeneration, with Phase 2a development initiated for 2026.
Rznomics receives FDA RMAT status for RNA-editing HCC candidate
Rznomics' RZ-001, an RNA-editing therapeutic targeting hepatocellular carcinoma, received FDA Regenerative Medicine Advanced Therapy designation based on Phase 1b/2a safety and efficacy data. This regulatory milestone accelerates development pathways for a precision oncology candidate using trans-splicing ribozyme technology to selectively target cancer cells.
Scribe Therapeutics reaches second milestone in Lilly collaboration
Scribe Therapeutics and Eli Lilly have achieved a second development milestone in their partnership to create in vivo CRISPR-based genetic medicines for neurological and neuromuscular diseases. The progress validates their X-Editor platform and data-driven optimization approach for precise gene editing, positioning these therapies to address conditions with limited treatment options.
Using mRNA to Fight Tau Aggregation in Alzheimer’s
Researchers developed a lipid nanoparticle that delivers mRNA encoding TRIM11, a natural ligase that disaggregates tau tangles, across the blood-brain barrier in mouse models of Alzheimer's disease. Treatment produced sustained cognitive and behavioral restoration comparable to wild-type mice, with reduction in tau pathology and neuroinflammatory markers.
Aerobic Capacity Encodes Distinct Brain Methylation
Rats with naturally high aerobic capacity exhibit distinct DNA methylation patterns in the hippocampus and elevated signaling through MAPK, AKT-mTOR, and synaptic pathways in cortical tissue—a molecular signature present independent of exercise training. This suggests intrinsic aerobic capacity creates a measurable biochemical state in aged brain tissue that may support cognitive resilience.
Oligonucleotide therapeutics market forecast to reach $17.7B by 2030
The oligonucleotide therapeutics market is projected to reach $17.7 billion by 2030, growing at 19.7 percent annually from 2025. Advances in RNA-targeting medicines and delivery technologies are enabling precision approaches to neurological disorders and rare diseases where conventional pharmaceuticals have limited efficacy.
IFN-γ emerges as a simple blood clue to Alzheimer’s
Interferon-gamma (IFN-γ), a common immune signaling molecule, emerges as a blood-based biomarker capable of distinguishing Alzheimer's disease from healthy aging with high accuracy, particularly in individuals carrying the APOE ε4 genetic variant. The finding suggests a scalable pathway for early detection and monitoring, shifting the disease profile toward immune-driven pathology.
Electrocardiogram derived heart age models agreement, accuracy and predictive ability in the Tromsø study
Electrocardiogram-derived heart age models show moderate agreement with chronological age but demonstrate significant individual variation, with predictive accuracy for cardiovascular outcomes varying substantially depending on the specific model used. This suggests ECG-based age estimates require careful interpretation and cannot yet replace traditional risk assessment tools.
Enzymatic RNA synthesis cuts manufacturing toxicity 50%
Codexis has advanced enzymatic RNA manufacturing with precise stereochemical control and full-length siRNA synthesis capability, while reducing environmental impact by over 50% through aqueous processing and minimal organic solvent use. This development accelerates the scalability and sustainability of RNA therapeutics, a critical class of drugs targeting aging-related diseases and tissue regeneration.
Dendritic Cell Reprogramming Drives Durable Tumor Rejection
Researchers engineered mRNA therapies that reprogram dendritic cells to enhance T cell activation against cancer, achieving complete tumor regression in preclinical models while establishing durable immune memory. This approach targets intracellular signaling pathways rather than relying on external cytokine signals, addressing a fundamental limitation in current immunotherapy.
Oligomer targeting reshapes Alzheimer's safety profile
ProMIS Neurosciences reported interim Phase 1b data showing PMN310, which targets toxic amyloid oligomers rather than large plaques, produced no cases of amyloid-related imaging abnormalities (ARIA-E) in a cohort with high APOE4 representation. This selective approach may expand treatment access for genetically high-risk populations previously limited by safety concerns with conventional anti-amyloid therapies.
Rafael Holdings secures MIT patent license for cyclodextrins
Cyclo Therapeutics secured an exclusive MIT patent license for cyclodextrin use in ApoE4-positive Alzheimer's disease patients, a population representing 50-70% of Alzheimer's cases. The mechanism targets cholesterol transport in the central nervous system to address amyloid and tau pathology, with Phase 3 results expected in Q3 2026.
Sickle cell disease competitive landscape highlights broad drug pipeline
Over 40 companies are developing more than 50 drug candidates for sickle cell disease across gene therapy, gene editing, and small molecule approaches, with several candidates in late-stage clinical trials. This expansion of therapeutic options addresses a genetic blood disorder that directly compromises oxygen delivery, energy production, and tissue regeneration—core drivers of age-related pathology.
Airna doses first patient in Phase 1 trial of AIR-001 for AATD
Airna initiated a Phase 1 trial of AIR-001, an RNA-editing therapeutic designed to correct the genetic mutation underlying alpha-1 antitrypsin deficiency by restoring functional AAT protein production. This approach addresses a monogenic disorder affecting both pulmonary and hepatic function, representing a mechanistic advance in treating a condition with significant longevity impact across multiple organ systems.
Ocular immunity gene therapy targets age-related vision loss
Cirrus Therapeutics secured funding and established a research collaboration with Singapore Eye Research Institute to advance ocular immunology therapies, including an IRAK-M targeting gene therapy and retinal pigment epithelium cell therapy for geographic atrophy. The partnership accelerates preclinical development and positions the company in the expanding ocular immunology sector.
Dyno Therapeutics license exercised by Astellas for muscle delivery
Astellas exercised a licensing option for a Dyno Therapeutics engineered AAV capsid designed for skeletal muscle gene delivery, marking validation of AI-driven capsid engineering for tissue-targeted viral vectors. The technology addresses a fundamental limitation in gene therapy: achieving efficient delivery to specific tissues while maintaining manufacturability.