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Gene Therapy News

Precision genetics: unlocking the secrets of mutation

Harvard University - 13-Nov-2024

A revolutionary tool targets specific genes, advancing our understanding of evolution and disease

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Dyno partners with Roche to enhance gene therapy for brain diseases

Longevity Technology - 25-Oct-2024

They aim to use AI-driven capsid design for delivery for complex treatments

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Nanoparticles deliver tools to fix gene mistakes in lung cells

The Scientist - 15-Oct-2024

Tests in mice and human cells show long-lasting gene correction

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Nanopillars unlock the nucleus without harming cells

Science Daily - 02-Oct-2024

A new breakthrough in nanotechnology offers a non-invasive way to access the cell's genetic material

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Rejuvenate Bio secures $4M to advance gene therapy for a devastating heart disease

BioProcess International - 27-Aug-2024

A promising one-time treatment approach for arrhythmogenic cardiomyopathy

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Gene-editing offers NHS patients potential cure for beta thalassemia

BBC - 08-Aug-2024

This revolutionary treatment could eliminate the need for lifelong blood transfusions

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Beacon Therapeutics secures $170M to develop gene therapies for retinal diseases

Longevity Technology - 05-Jul-2024

Company is particularly focusing on less invasive treatments for dry AMD and XLRP

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NHS offers novel gene therapy for haemophilia B, potentially curing patients

BBC - 27-Jun-2024

The therapy is initially available at eight centers across the UK

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New gene-editing tool, 'bridge editing,' surpasses CRISPR in precise DNA alterations

New Scientist - 26-Jun-2024

Potential for advancements in medicine, agriculture, and other fields

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Scientists discover key protein that could prevent age-related vision loss

Independent - 05-Jun-2024

Boosting IRAK-M protein protects retina in the eyes and paves way for potential AMD treatment

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ExpressionEdits secures $13M for developing AI-powered gene therapy

Longevity Technology - 03-Jun-2024

Company aims to improve protein production in genetic medicine

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Toddler born with hearing loss can now hear thanks to new gene therapy trial

The Guardian - 09-May-2024

Therapy offers hope for a potential cure for certain types of hearing loss

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Gene therapy triggers natural blood vessel growth in angina patients

EurekAlert! - 02-May-2024

Treatment shows improved blood flow, exercise tolerance and reduced symptoms

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Gordian Biotechnology raises $60M to accelerate gene therapy discovery

Longevity Technology - 26-Apr-2024

AI-powered platform predicts drugs for age-related diseases with 80% accuracy

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AI designs a more precise CRISPR gene editor for use in human cells

Singularity Hub - 25-Apr-2024

This could lead to safer and more effective treatments for genetic diseases

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Nanoscope's gene therapy offers hope for patients blinded by inherited retinal disease

FierceBiotech - 26-Mar-2024

Phase 2 trial results show significant vision improvement, plans for FDA approval

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Scientists overcome hurdles to design human artificial chromosomes

Singularity Hub - 26-Mar-2024

New technique delivers more genetic material, paving way for safer & effective gene therapies

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Columbia scientists use CRISPR gene editing to boost energy production in eye cells

Medical Xpress - 21-Mar-2024

This experimental therapy targets a common metabolic issue in Retinitis Pigmentosa

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Gene therapy for rare brain disease sets a new pricing record at $4.25 million

FiercePharma - 20-Mar-2024

Its record-breaking price raises questions about affordability and access

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Novel approach effectively silences cholesterol genes in mice for a year

News Medical - 01-Mar-2024

This could lead to long-lasting cholesterol treatments without genetic damages

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Invisible stem cells treat disease without triggering immune response

Nature - 28-Feb-2024

Genetically-modified stem cells avoid immune rejection, paving way for universal therapies for everyone

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Diabetic monkeys regain insulin independence after transplant of unique insulin cells

Live Forever Club - 23-Feb-2024

This could lead to a safer and better treatment for type 1 diabetes.

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RNA editing therapies for genetic diseases signify medical research progress

Nature - 16-Feb-2024

Unlike editing DNA itself, this new approach makes temporary changes to RNA

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Rejuvenate Bio partners with animal company to develop gene therapy for canine osteoarthritis

Biospace - 14-Feb-2024

Single dose injection targets root causes, offering long-term relief & improved quality of life

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Fractyl raised $110 million to develop treatments for obesity and diabetes

Longevity Technology - 05-Feb-2024

They picked RJVA-001, a gene therapy, as their first clinical candidate

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Dresden merges recombinases & DNA-binding domains for tailored gene editing

Medical Xpress - 31-Jan-2024

The research offers safer and more precise ways to treat genetic disorders

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A CRISPR gene editing technique has cured hereditary angioedema in a single-dose

New Scientist - 31-Jan-2024

It demonstrated a remarkable cure rate, rather than merely offering a treatment

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Regenxbio-AbbVie gene therapy reduces eye injections for AMD patients

FierceBiotech - 17-Jan-2024

Patients’ vision and retinal health remained stable after six months

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MeiraGTx at the forefront of gene therapy for cancer, obesity, and vision loss

Evening Standard - 21-Dec-2023

London biotech promises revolutionary therapies by replacing faulty genes & flipping gene switches

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Revolutionizing gene editing: Tome Biosciences unveils groundbreaking DNA integration technology

Genetic Engineering & Biotechnology News (GEN) - 19-Dec-2023

Tome Biosciences secures $213m to advance next-gen gene editing

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ARCUS gene editing technology effectively eliminates mutant mitochondrial DNA

Biospace - 04-Dec-2023

Potential therapeutic approach for mitochondrial and other genetic diseases

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UK's approval of casgevy paves the way for gene-edited treatment of blood disorders

BBC - 16-Nov-2023

Casgevy offers hope for patients with sickle cell disease and beta thalassemia

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Single-dose gene-editing therapy offers permanent solution to high cholesterol levels

New Scientist - 14-Nov-2023

Early results show promise in eliminating the need for lifelong statins

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A new gene therapy treatment has helped deaf children hear for the first time

MIT Technology Review - 27-Oct-2023

Treatment delivers a working copy of a missing gene to the inner ear

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Beyond CRISPR: Fanzors, a new era of programmable DNA editing tools

Massachusetts Institute of Technology (MIT) - 13-Oct-2023

Scientists discover a vast array of RNA-guided enzymes in diverse organisms

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Broken string secures $15m to make cell and gene therapy safer, more accessible

Longevity Technology - 19-Sep-2023

Company's INDUCE-seq platform could revolutionise cell & gene therapy efficiency

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Amber Bio secures $26M funding to advance new gene editing technologies

Longevity Technology - 07-Aug-2023

By editing RNA instead of DNA, the company aims to treat multiple undruggable genetic diseases safely

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Voyager's new gene therapy targets amyloid plaques in Alzheimer's disease

FierceBiotech - 04-Aug-2023

New treatment approach could be more effective in lowering the risk of serious side effects

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Novel method facilitates single-cell expansion of gene edited stem cells

Lifespan.io (LEAF) - 17-Jul-2023

Its polymer-supplemented culture system enables cell expansion without harmful mutations

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Gene therapy restores partial red colour vision in total colour blindness patients

New Scientist - 10-Jul-2023

Despite the limited improvement, gene therapy offers hope for future breakthroughs in restoring vision

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Game-changing bacteriophage could revolutionize gene therapy

New Scientist - 30-May-2023

Ability to carry vast amounts of DNA and other molecules promises substantial advancements in gene and cell therapies

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Therapeutic applications of base editing clears girl's leukemia

BBC - 11-Dec-2022

Base editing can soon be a part in taking control of our genomes to fight genetic diseases

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CRISPR based PASTE (Programmable Addition via Site-specific Targeting Elements) tool designed

MIT Media Lab - 24-Nov-2022

The new PASTE tool replaces faulty genes in a safer and more efficient way

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Jeune Aesthetics moves to the Phase 2 trial for fine lines and wrinkles

Biospace - 17-Nov-2022

Targeted gene-delivery hold potential in replenishing the skins key protein, to treat fine lines

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Breakthrough gene therapy offers cure for a rare bleeding disorder heamophilia

BBC - 21-Jul-2022

Trial results were positive but cautioned the use until proven safe and effective

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HTR in Drosophila somatic cells using nikase and CRISPR genetic editing tools

Genetic Engineering & Biotechnology News (GEN) - 05-Jul-2022

CRISPR genetic editing tools may be helpful in genetic repair

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Lifespan of middle-aged mice increased by a third with telomerase gene therapy

CISION PR Newswire - 19-May-2022

Treatment used proprietary CMV vector which is able to deliver multiple genes into the nucleus

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Viral-vector gene therapies could unlock healthy life extension in humans

Lifespan.io (LEAF) - 12-May-2022

Lifespan of mice greatly extended even when treatment only started at the equivalent of 56 years old

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Verve Therapeutics' gene editing therapy may cure heart attacks once and for all

Bloomberg - 06-May-2022

Trials underway to test the safety, but affordability and acceptance of gene therapy are major concerns

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Gene therapy gel heals wounds in people with rare genetic skin condition

New Scientist - 28-Mar-2022

Positive results in clinical trials provide hope for conditions that currently have no specific treatments

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Long-term partial reprogramming reversed ageing process in mice

The Guardian - 07-Mar-2022

Yamanaka factors restored youthfullness in aged cells, raised hope for new anti-ageing therapies

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NHS offers the world's most expensive drug for children with a rare disease

BBC - 04-Feb-2022

Libmeldy is life-saving gene therapy now available for use in England

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Gene-editing technology, CRISPR, selectively kills drug-resistant superbugs

Deutsche Welle (DW) - 10-Nov-2021

A single dose of CRISPR Cas system cleared 99.9% of harmful bacterial in mice

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Turning off abnormal genes may cure crippling genetic disease

BBC - 21-Oct-2021

Potential gene silencing approach in porphyria and possibly for many other diseases in the future

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FDA puts clinical hold on Allogenic CAR T therapy due to safety concerns

FierceBiotech - 08-Oct-2021

Chromosome abnormality reported in a clinical trial raises doubts on patient safety

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OMEGA, a new powerful gene-editing system similar to CRISPR or so much better

New Atlas - 16-Sep-2021

Potential RNA-guiding enzymes that are capable of editing and modifying DNA in human cells

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Centaura's human artificial chromosome to treat complex diseases, including ageing

Medium - 10-Sep-2021

A breakthrough technology focusing on effective gene therapies for healthy longevity

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Lifespan.io interviews George Church about gene therapy and longevity

Lifespan.io (LEAF) - 28-Jul-2021

Discusses animal trials, potential human trials, reeducating the FDA and improvements in technology

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New CRISPR gene-editing drug for a rare protein-misfolding disorder

pharmaphorum - 28-Jun-2021

Phase 1 trial reports, single dose of the drug cuts protein levels by 87% in ATTR amyloidosis

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Brain can learn to see again using algae proteins

BBC - 24-May-2021

Optogenetics bring back vision partially with light sensing proteins - new hope for the blind

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Gene therapy preserved memory and learning in mice with Alzheimer's

Lifespan.io (LEAF) - 11-May-2021

Boosting a vital protein, Caveolin-1, this new technique offers hope against neurodegenerative diseases

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New gene therapy technique, CopyCatcher, more accurate in fruit flies

SciTechDaily - 11-May-2021

Targets precise location of mutated gene rather than tagging on anywhere

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Inherited blindness can now be cured with gene therapy

BBC - 29-Apr-2021

A breakthrough gene therapy restores eyesight in patients with rare eye disease

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Latest CRISPR editing method can turn on/off gene expression

Phys.org - 09-Apr-2021

Allows to silence bad genes without modifying the DNA sequence and it is fully reversible

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Gene therapy for Alzheimer's disease enters human clinical trial

Forbes - 12-Mar-2021

Virus carries gene encoding protein into the brain -possible breakthrough treatment for neurodegenerative diseases

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Single gene therapy injection halts spinal muscular atrophy

BBC - 08-Mar-2021

Becomes most expensive drug ever approved for the NHS - but no lifetime costs

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CRISPR cuts cholesterol by 57% with just one shot

Phys.org - 01-Mar-2021

Tiny lipid particles with gene editing machinery selectively targets liver and reduces cholesterol in mice

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A look at the government consultation on genetic technologies in agriculture

Nuffield Council on Bioethics - 04-Feb-2021

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Deactivating ageing gene KAT7 to slow down ageing

Reuters - 20-Jan-2021

A potential therapeutic target for ageing, but still a long way to human trials

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A gene editing tool used to treat progeria in mice

Science Magazine - 06-Jan-2021

Hope to launch a clinical trial in kids soon using this approach

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Gene therapy prevented deafness in mice with genetic defects

ISRAEL21c - 04-Jan-2021

Scientists to use this approach & treat deafness due to other mutations also

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CRISPR's gene editing therapy finds cure for blood cancer

STAT - 05-Dec-2020

Early data from Vertex & CRISPR showed promising results to cure blood disorders

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MIT professor Richard Young explains how Omega regulates gene expression

Cell and gene - 01-Dec-2020

Using Epigenomic Controllers to alter DNA transcription rather than the genes themselves

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A novel gene editing tool to fight aggressive cancers

Medical Xpress - 18-Nov-2020

Uses CRISPR technology to target and destroy cancer cells

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A potential therapy for blinding retinal diseases

National Institutes of Health (NIH) - 03-Nov-2020

Researchers engineer novel protein that restores vision in blind mice via gene therapy

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Tiny lipid bubbles could deliver gene editing kit to your tissues

Phys.org - 08-Oct-2020

Will make it easier to target treatment to specific locations in the body

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Old mice show improved muscle function with gene therapy

Lifespan.io (LEAF) - 27-Aug-2020

A single injection could treat motor diseases of the aged but needs more human studies

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90 % of herpes simplex virus 1 eliminated in infected cells

Lifespan.io (LEAF) - 19-Aug-2020

Successful in mice, new approach may ease immune burden of herpes in humans in the future

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$95M raised by Taysha for clinical trials of four new gene therapies

FierceBiotech - 05-Aug-2020

Company has gathered support from investors to quickly lead therapies to clinic then market

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Scientists go where no gene-editing tool has gone before

Lifespan.io (LEAF) - 14-Jul-2020

DddA able to target mitochondrial DNA (mtDNA) by splitting in half and hitching a ride

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A gene editing tool to treat mitochondrial mutations

The Scientist - 09-Jul-2020

Derived from bacterial toxin - can make single base pair changes

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Company introduces 'gene writing', to surpass CRISPR's limitations

Wired - 07-Jul-2020

New class of gene editors set to remove hurdles of traditional gene therapy

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Gene editing lowered blood level cholesterol and triglycerides in monkeys

New York Times - 27-Jun-2020

Could prevent not only the complications but the heart disease per se in the future

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CRISPR gene editing to cure beta thalassaemia and sickle cell disease

New Scientist - 12-Jun-2020

Didn't change the inherited faulty gene, but gene to switch on fetal haemoglobin production

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Phase 1 clinical trial for type 1 diabetes cell therapy comes closer

Medical Xpress - 03-Jun-2020

A world-first novel technology to treat the disease is now within reach

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Automated manufacturing of custom-engineered cell lines

News Medical - 20-May-2020

This technology is very promising and has an enormous potential

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Follistatin gene therapy to combat osteoarthritis and obesity

Next Big Future - 16-May-2020

This new approach doubled muscle mass in mice, reduced its fat

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Mapping gene expression regulation in yeast to understand ageing

Google AI Blog - 29-Apr-2020

Made possible by Calico's research expertise and Google AI's machine learning tech

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CRISPR-Cas9 variants, key to overcoming major limitation in gene editing system

Lifespan.io (LEAF) - 31-Mar-2020

Entire genome now targetable, open to interventions for use in combating diseases

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A global commission laid out roadmap for gene-editing trials on human embryo

Wired - 09-Mar-2020

Strict ethical guidelines to avoid undesirable outcomes in gene-edited babies

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Fixing the genes to cure inherited blindness

Guardian - 04-Mar-2020

Crispr gene editing is a great but a dangerous tool

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First gene therapy for a rare eye disease, now NHS-approved

BBC - 17-Feb-2020

This life-changing new gene therapy will halt sight loss and improve vision

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CRISPR gene editing to help immune cells target and kill cancer

New Scientist - 06-Feb-2020

First CRISPR cancer trial in the world publish its promising findings

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Chinese scientist tried genome editing on embryos

Guardian - 31-Dec-2019

Research of He Jiankui was a violation of medical regulations

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Scientists identify multiple genes responsible for keeping cancer cells alive

Independent - 20-Dec-2019

If combined they can form the largest database of genetic information on cancer

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NIH and Gates Foundation to invest $100 million each to develop gene therapies

Time Magazine - 24-Nov-2019

New hope for the 37.9 million people worldwide living with HIV/AIDS

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CRISPR technology edited plant cells after being sprayed on their leaves

New Scientist - 01-Nov-2019

Can get into animal cells, so I wonder if this could be used with humans in future?

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Prime editing as a way of "editing" genes

BBC - 21-Oct-2019

There are around 75,000 mutations caused by DNA errors and prime editing can correct 89% of them

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Direct cell to cell transformation without a stem cell stage

TechCrunch - 14-Oct-2019

Analyses a huge amount of data without the help of an artificial intelligence

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Drug invented for a girl with Batten disease

BBC - 12-Oct-2019

Doctors from Boston sequenced in detail whole genome of Mila and found the disease-causing mutation

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