Genetics & Epigenetics Library
Every article, presentation, spotlight, and news item we've tagged to Genetics & Epigenetics.
Showing 25–48 of 176
Adipose Gene Therapy Cuts Chronic Disease Dosing Burden
Remedium Bio secured $10 million in Series A funding to advance a platform technology that converts subcutaneous fat tissue into sustained protein-production sites, reducing treatment frequency from multiple injections to single administrations with adjustable dosing. The approach targets cardiometabolic and chronic disease management through controlled gene expression.
Voyager IV gene therapies for Alzheimer’s disease highlighted at ASGCT 2026
Voyager Therapeutics will present VY1706, an intravenously delivered gene therapy targeting tau pathology in Alzheimer's disease, at ASGCT 2026. Preliminary three-month toxicology data in non-human primates demonstrate pharmacological efficacy and safety, with first-in-human dosing anticipated in late 2026.
Engineered fat cells deliver multi-year gene therapies
Remedium Bio secured $10 million in Series A funding to advance gene therapies designed for durable, long-lasting protein production using engineered fat cells as a therapeutic depot. The platform targets chronic diseases across endocrinology, immunology, neurology, musculoskeletal, and cardiometabolic domains with single-injection, adjustable dosing.
Genflow teams with Acuitas on gene delivery in fully-funded deal
Genflow Biosciences and Acuitas Therapeutics have partnered to optimize delivery of a SIRT6 gene therapy targeting aging-related processes. The collaboration underscores that therapeutic efficacy depends equally on how genetic material reaches target tissues as on the genetic intervention itself.
One-Time Gene Therapy Reduces Tau Protein in Alzheimer's Disease
Voyager Therapeutics has obtained FDA clearance to initiate clinical trials for VY1706, a single-administration gene therapy targeting tau protein in early Alzheimer's disease. Preclinical data demonstrated tau reductions up to 75% in relevant brain regions with acceptable tolerability, positioning this approach as a potential disease-modifying intervention for neurodegeneration.
RNA therapies research lists 80+ companies, 100+ drugs
A comprehensive market analysis catalogs over 80 RNA therapy companies and 100+ drug candidates across multiple modalities—mRNA, siRNA, antisense oligonucleotides, aptamers, and CRISPR approaches. The research examines development stages, delivery mechanisms, and commercial landscapes, with established players like Novartis and Arrowhead Pharmaceuticals advancing candidates across multiple therapeutic areas.
RNA Editing Rewrites Cancer Cell Instructions Without Permanent DNA Change
RNA editing therapy RZ-001 received FDA fast-track designation for hepatocellular carcinoma, signaling regulatory confidence in programmable medicine approaches that correct cellular instructions rather than permanently altering DNA. This advancement reflects a broader shift toward precision interventions that address age-related disease mechanisms at the molecular level.
Unlimited Bio registers dual gene therapy trial
Unlimited Bio initiated a Phase 1/2a trial combining AAV9-Follistatin and VEGF plasmid gene therapies to address age-related muscle decline, marking the first registered clinical study to combine these two approaches. The dual-therapy strategy targets both muscle growth signaling and vascular support, with safety and functional outcomes tracked over 12 months in adults aged 45 to 75.
‘Age reversal is the only viable path for effective therapy’
Telocyte's founder argues that effective longevity intervention requires reversing aging at the cellular level rather than managing age-related diseases incrementally. The company is preparing a telomerase gene therapy trial in dogs, with the thesis that aging results from failed maintenance systems that can be reset rather than from inevitable biological decline.
VectorY doses first patient in ALS gene therapy trial
VectorY Therapeutics has dosed the first patient in a Phase 1/2 trial of VTx-002, a gene therapy designed to sustain antibody production against pathological TDP-43 protein in ALS. This represents a shift from symptomatic management toward addressing the underlying biology of neurodegeneration, with implications for protein aggregation disorders across aging.
In Vivo Macrophage Reprogramming Expands Regenerative Therapy Access
Resolution Therapeutics is developing an in vivo regenerative macrophage therapy that reprograms immune cells directly within the body using mRNA-carrying lipid nanoparticles, rather than extracting and modifying cells ex vivo. This approach could extend regenerative medicine access to patients with advanced liver disease and chronic inflammatory conditions by eliminating manufacturing complexity.
Gene therapy reprograms cartilage cells without grafting
FibroBiologics obtained European patent protection for a gene therapy approach that instructs fibroblast cells to differentiate into cartilage-producing cells using Sox9 and related genetic sequences. The technology addresses cartilage degradation through cellular reprogramming rather than surgical grafting, with potential application across orthopedic and spinal conditions.
Gene Therapy Corrects Hearing Loss at Genetic Source
MED-EL acquired gene therapy programs targeting MYO7A and STRC mutations, which cause genetic hearing loss and balance disorders. This represents a strategic shift toward addressing the genetic substrate of sensory dysfunction rather than managing symptoms alone, positioning corrective therapy alongside existing implantable solutions.
CRISPR In Vivo Gene Editing Achieves Phase 3 Success
In vivo CRISPR gene editing achieved its first Phase 3 clinical success, with lonvoguran reducing hereditary angioedema attacks by 87% in treated patients versus placebo, with 62% remaining attack-free. This represents a proof-of-concept that permanent genetic intervention can supplant lifelong symptomatic management for monogenic disease.
SIRT6 Gene Therapy Improves Survival in Aging Dogs
Genflow's SIRT6-targeted gene therapy in aging dogs showed improved survival at three months post-treatment with no adverse events reported. The trial, enrolling 24 beagles over age 10, represents an early-stage assessment of whether genetic interventions can reverse hallmarks of aging in a mammalian model relevant to human longevity research.
Flagship launches Serif Biomedicines to develop modified DNA medicines
Flagship Pioneering launched Serif Biomedicines to develop Modified DNA medicines—a hybrid platform combining DNA and mRNA delivery with reduced immune activation and enhanced durability. The approach targets rare genetic diseases and immune reprogramming with preclinical evidence of tolerability and sustained gene expression in primates.
Epigenetic CRISPR therapy cuts LDL 68% in single dose
STX-1150, a liver-targeted CRISPR-based epigenetic therapy, achieved up to 68% LDL-C reduction from a single dose in non-human primates, with effects sustained beyond 22 months. The approach modulates gene expression without permanent DNA alteration, advancing toward human trials with regulatory clearance in Australia.
Age‐Like Methylation Changes of HSCs in GADD45B Knockout Mice Define Methylation Sites Associated With Loss of Function
GADD45B deletion induces DNA methylation patterns resembling age-associated changes in hematopoietic stem cells, yet these methylation alterations occur without functional decline. The research distinguishes between methylation signatures and actual loss of HSC capacity, providing a resource to identify which methylation sites causally drive age-related hematopoietic dysfunction.
RNA gene therapy achieves durable glioblastoma control
RZ-001, an RNA editing-based gene therapy, demonstrated tolerability and disease control in 10 glioblastoma patients during Phase 1/2a trials, with no dose-limiting toxicity and several patients achieving tumor recurrence inhibition beyond six months. Early safety and efficacy signals support continued development of this targeted approach to a treatment-resistant malignancy.
Rafael Holdings gains exclusive MIT Alzheimer’s patent
Rafael Holdings secured exclusive MIT patent rights for cyclodextrin-based molecules targeting ApoE4-positive Alzheimer's disease by restoring cholesterol transport between brain cells. This represents a shift from amyloid-clearance paradigms toward addressing the cellular infrastructure that prevents pathological buildup in the first place.
Durable CRISPR cholesterol silencing lasts 18 months
Scribe Therapeutics presented engineered CRISPR platforms—ELXR for epigenetic silencing and XE for genome editing—designed to lower LDL cholesterol through sustained PCSK9 knockdown with improved specificity and reduced off-target effects. Preclinical data in non-human primates demonstrated durable LDL-C reduction over 18 months, positioning these platforms as potential therapeutic alternatives to current cholesterol management approaches.
Gordian Bio unveils scalable in‑vivo screening platform preprint
Gordian Bio describes a scalable in-vivo mosaic screening platform that uses barcoded CRISPR libraries and single-cell readouts to establish causal gene-disease relationships in living organisms. This approach accelerates therapeutic target discovery by testing multiple genetic perturbations simultaneously, reducing experimental variation and improving efficiency compared to traditional methods.
Cell and gene therapy market shows $1.2B Zolgensma and $1.5B Yescarta sales
The cell and gene therapy market demonstrates commercial maturation with Zolgensma generating $1.2 billion and Yescarta $1.5 billion in annual sales, reflecting regulatory acceptance of 46 FDA-approved CGT products. This market trajectory indicates that genetic and cellular interventions are transitioning from experimental to established therapeutic options for previously intractable genetic disorders and certain cancers.
Epigenetic Restoration Enters Human Trial for Vision Loss
Life Biosciences has dosed the first patient in a Phase 1 trial of ER-100, an epigenetic restoration therapy targeting retinal ganglion cell dysfunction in glaucoma and NAION. The trial represents a pivotal clinical test of whether partial reprogramming of gene expression patterns can reverse age-related cellular decline in humans.