211 
5 questions facing gene therapy in 2021
Bio Pharma Dive
JANUARY 8, 2021
Gene therapy developers will need to address a series of setbacks that have shaded optimism in the sector's fast growth
Gene Related Topics 
Gene therapy for hemophilia: So close, yet so far away
Bio Pharma Dive
JANUARY 28, 2021
Significant setbacks for pace-setting programs from BioMarin and UniQure have renewed questions about gene therapy’s promise, and its safety
Join 5,000+
Insiders
Sign Up for our Newsletter
This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.
Trending Sources
Former AveXis executives launch gene therapy startup with uncommon targets
Bio Pharma Dive
FEBRUARY 25, 2021
Sean Nolan and Joe Nolan, former leaders at the Zolgensma developer, are behind Jaguar Gene Therapy, which plans to research treatments for a rare metabolic disease as well as genetically linked autism and diabetes
Sarepta, continuing its gene therapy push, helps launch a startup
Bio Pharma Dive
OCTOBER 22, 2020
The biotech has joined a group of investors to form a new gene therapy developer led by one of its longtime executives and backed with $107 million
Gene editing: beyond the hype
Pharma Phorum
JANUARY 29, 2021
Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome.
J&J pushes further into eye gene therapy
Bio Pharma Dive
DECEMBER 2, 2020
The pharma licensed a treatment for a severe type of age-related vision loss, building on past deals that gave it a foothold in gene therapy
Scientists use DNA origami to monitor CRISPR gene targeting
Scienmag
FEBRUARY 23, 2021
Technology and Engineering Biomechanics/Biophysics Biotechnology Chemistry/Physics/Materials Sciences Gene Therapy Genes Genetics
Beam, capitalizing on stock surge, buys gene therapy tools developer for $120M
Bio Pharma Dive
FEBRUARY 23, 2021
Guide Therapeutics, the company Beam acquired, is working on ways to more efficiently deliver gene editing therapies into the body
How 2 scientific pioneers teamed up to run AskBio, Bayer's new gene therapy division
Bio Pharma Dive
JANUARY 21, 2021
Katherine High and Jude Samulski, two well-known gene therapy researchers, were longtime collaborators and competitors. Now they're colleagues at one of the world's largest drugmakers
UniQure's gene therapy for hemophilia B meets first goal in key study
Bio Pharma Dive
NOVEMBER 19, 2020
The results are first to emerge from a late-stage test of a hemophilia B gene therapy, and show UniQure's treatment can restore clotting protein levels to mild or even near normal levels
Gene Therapy for Muscular Disorders
Pharma Phorum
JANUARY 21, 2021
With clinical holds lifting there has been more investment and clinical progress in the gene therapy for muscular disorders space than ever before, yet the consequences of toxicity are not a distant memory. The post Gene Therapy for Muscular Disorders appeared first on.
Bayer to buy AskBio for $2B in bid to become a gene therapy player
Bio Pharma Dive
OCTOBER 26, 2020
Founded by gene therapy pioneer Jude Samulski, AskBio boasts a pipeline of experimental gene therapies as well as its own manufacturing capabilities
Bluebird faces delay for sickle cell gene therapy as rivals inch closer
Bio Pharma Dive
NOVEMBER 5, 2020
The setback for Bluebird's LentiGlobin comes amid a string of manufacturing-related delays for gene therapy developers
Genes for face shape identified
Scienmag
FEBRUARY 8, 2021
Genes that determine the shape of a person’s facial profile have been discovered by a UCL-led research team. The researchers identified 32 gene regions that influenced facial features such as nose, lip, jaw, and brow shape, nine of which were entirely new discoveries while the others validated genes with prior limited evidence. Archaeology Anthropology Biology Developmental/Reproductive Biology Evolution Genes Genetics New World
Lilly dives into gene editing
Bio Pharma Dive
NOVEMBER 20, 2020
Through a deal with Precision Biosciences, the pharma aims to develop therapies for genetic disorders, including Duchenne muscular dystrophy
Preclinical done right: The importance of using relevant animal models in gene therapy studies
Bio Pharma Dive
OCTOBER 1, 2020
Gene therapy is no longer an approach for the future. It's a technique used now
Ultragenyx expands into Duchenne gene therapy with Solid Bio deal
Bio Pharma Dive
OCTOBER 23, 2020
The rare disease drug developer is betting it can create a better DMD treatment by combining Solid's research with its manufacturing and gene delivery technology
FDA wants more details on Sarepta gene therapy, potentially delaying final test
Bio Pharma Dive
SEPTEMBER 10, 2020
A new request from the regulator could tighten the race between Sarepta and Pfizer to prove out a gene therapy for Duchenne muscular dystrophy
Larger panel finds more gene mutations, treatment targets for leukemia
Scienmag
JANUARY 31, 2021
1, 2020) – A gene panel that looks for about 10 times the number of cancer-causing genes as panels currently used to diagnose and fine tune treatment for a variety of cancers is effective at identifying problematic genes in the most common leukemia, investigators report.
Axovant shares sink on manufacturing delay for Parkinson's gene therapy
Bio Pharma Dive
OCTOBER 30, 2020
A longer-than-expected timeline for developing a new manufacturing process means a key study for Axovant's top gene therapy likely won't begin until 2022
AbbVie exploring how CRISPR gene editing can improve cell therapies
Bio Pharma Dive
FEBRUARY 10, 2021
Using technology from Caribou Biosciences, AbbVie hopes to engineer "off-the-shelf" CAR-T cells that can better withstand attacks from the immune system
Gene therapy solution: The value of a CDMO as your end-to-end partner
Bio Pharma Dive
SEPTEMBER 29, 2020
As gene therapy research continues to expand, innovators in this space will need CDMOs with highly specific expertise, facilities, and equipment
Roche, aiming to make better gene therapies, turns to an emerging startup
Bio Pharma Dive
OCTOBER 14, 2020
The Swiss pharma is the third large drugmaker to partner with Harvard spinout Dyno Therapeutics, a small biotech aiming to improve gene therapy technology
Cancer case puts UniQure's hemophilia gene therapy on hold, raising alarms
Bio Pharma Dive
DECEMBER 21, 2020
The FDA halted the AMT-061 program, which could be the first approved gene therapy for hemophilia B, because a patient who received the treatment appears to have developed liver cancer
Many genes associated with the risk of coronary artery disease act through the liver
Scienmag
FEBRUARY 24, 2021
According to a new study published in The American Journal of Human Genetics, more than one third of genetic variants that increase the risk of coronary artery disease regulate the expression of genes in the liver. Credit: UEF/Raija Törrönen.
Editas, AbbVie rework gene editing deal as pioneering CRISPR trial resumes
Bio Pharma Dive
AUGUST 7, 2020
Development of EDIT-101, the first CRISPR-based therapy to be used in a company-led trial to alter genes within a person's body, will now move forward under Editas' sole ownership
Gene therapy for placental insufficiency moves toward the clinic
Scienmag
DECEMBER 15, 2020
publishers New Rochelle, NY, December 15, 2020–A new study identified an adenovirus gene therapy vector carrying a VEGF isoform. It can improve uterine blood flow in placental insufficiency, as reported in the peer-reviewed journal Human Gene Therapy.
Are male genes from Mars, female genes from Venus?
Scienmag
SEPTEMBER 11, 2020
Only a sprinkling of genes, located on the so-called X and Y sex chromosomes, differ between the sexes. Nevertheless, the activities of our genes–their expression in cells and tissues–generate […].
Sarepta gene therapy misses goal in key muscular dystrophy study
Bio Pharma Dive
JANUARY 7, 2021
Company executives, however, blamed "improbably bad luck" for study enrollment that resulted in more older patients with milder disease receiving placebo, and pledged to press on
Gene Therapy Comparability
Pharma Phorum
JANUARY 21, 2021
The inaugural Gene Therapy Comparability Summit is the only industry dedicated meeting to ensuring your manufacturing processes are effective and comparable at every stage of development, supporting a seamless transition to the larger scales required for commercialization.
Deadly white-nose syndrome changed genes in surviving bats
Scienmag
FEBRUARY 4, 2021
Biology Biodiversity Bioinformatics Ecology/Environment Evolution Genes Genetics Mycology Population Biology Zoology/Veterinary Science
FDA gene therapy holdups suggest closer scrutiny by agency
Bio Pharma Dive
NOVEMBER 5, 2020
Requests for more data on manufacturing, clinical holds and a surprise rejection have led to questions of a tougher standard at the agency. Officials, however, point to a large and fast-advancing pipeline
Scientists develop new gene therapy for eye disease
Scienmag
NOVEMBER 25, 2020
Medicine & Health Gene Therapy Genes Genetics Medicine/Health Ophthalmology
Gene-editing produces tenfold increase in superbug slaying antibiotics
Scienmag
JANUARY 12, 2021
Martins Scientists have used gene-editing advances to achieve a tenfold increase in the production of super-bug targeting formicamycin antibiotics. Technology and Engineering Bacteriology Biochemistry Biodiversity Biology Biotechnology Entomology Genes Genetics Microbiology Molecular Biology
BioMarin's gene therapy rejection didn't shock everyone
Bio Pharma Dive
AUGUST 20, 2020
The FDA's decision not to approve Roctavian raises the question of why BioMarin was able to submit the hemophilia A therapy in the first place
Winner-takes-all synthetic gene circuit process opens new pathways to disease treatment
Scienmag
FEBRUARY 8, 2021
Division of synthetic gene circuit workloads will make therapy more effective. Cancer Biology Biomedical/Environmental/Chemical Engineering Biotechnology cancer Cell Biology Gene Therapy MicrobiologyCredit: Graphic created by Xiaojun Tian/ASU.
Genes that dance to the circadian rhythm
Scienmag
JANUARY 29, 2021
Technology and Engineering Bioinformatics Biology Biotechnology Circadian Rhythm Genes GeneticsIn 2017, the Nobel Prize in Physiology or Medicine went to three scientists who uncovered the molecular mechanisms that control the circadian rhythm, otherwise known as the “wake-sleep” cycle. To carry out their work, the scientists used the common fruit fly Drosophila melanogaster, making this the sixth Nobel to be awarded to research involving it. […].
Risk genes in schizophrenia – their importance in choosing appropriate antipsychotic drug
Scienmag
FEBRUARY 25, 2021
Chemistry AND Physics Biotechnology Cell Biology Genes Genetics Medicine/Health Neurobiology Neurochemistry Pharmaceutical Science Pharmaceutical SciencesThis article by Dr. Felix-Martin Werner and Prof.
Gene Therapy Medical Affairs
Pharma Phorum
NOVEMBER 20, 2020
The inaugural Gene Therapy Medical Affairs Summit is dedicated to exploring the pivotal internal and external role that Medical Affairs plays in delivering gene therapies to patients. The post Gene Therapy Medical Affairs appeared first on.
Taysha Gene Therapies Announces New cGMP Gene Therapy Manufacturing Facility
BioTech 365
DECEMBER 17, 2020
Biologists create new genetic systems to neutralize gene drives
Scienmag
SEPTEMBER 18, 2020
Two active genetics strategies help address concerns about gene-drive releases into the wild Credit: Ana Silva In the past decade, researchers have engineered an array of new tools that control the balance of genetic inheritance.
Are Gene Therapies the Medicine of the Future?
BioSpace
JANUARY 25, 2021
Gene therapies offer great reward in the form of treating various devastating diseases, but there are also significant risks
Let's personalize your content