5 questions facing gene therapy in 2021

Bio Pharma Dive

Gene therapy developers will need to address a series of setbacks that have shaded optimism in the sector's fast growth

Gene therapy for hemophilia: So close, yet so far away

Bio Pharma Dive

Significant setbacks for pace-setting programs from BioMarin and UniQure have renewed questions about gene therapy’s promise, and its safety

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Former AveXis executives launch gene therapy startup with uncommon targets

Bio Pharma Dive

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

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

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

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

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

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

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

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

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

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

The setback for Bluebird's LentiGlobin comes amid a string of manufacturing-related delays for gene therapy developers

Genes for face shape identified

Scienmag

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

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Lilly dives into gene editing

Bio Pharma Dive

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

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

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

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

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.

Gene 59

Axovant shares sink on manufacturing delay for Parkinson's gene therapy

Bio Pharma Dive

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

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

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

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

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

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

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

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

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 […].

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Sarepta gene therapy misses goal in key muscular dystrophy study

Bio Pharma Dive

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

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

Biology Biodiversity Bioinformatics Ecology/Environment Evolution Genes Genetics Mycology Population Biology Zoology/Veterinary Science

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FDA gene therapy holdups suggest closer scrutiny by agency

Bio Pharma Dive

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

Medicine & Health Gene Therapy Genes Genetics Medicine/Health Ophthalmology

Gene-editing produces tenfold increase in superbug slaying antibiotics

Scienmag

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

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

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.

Gene 59

Genes that dance to the circadian rhythm

Scienmag

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. […].

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Risk genes in schizophrenia – their importance in choosing appropriate antipsychotic drug

Scienmag

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 59

Gene Therapy Medical Affairs

Pharma Phorum

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

Taysha Gene Therapies Announces Collaborations to Advance Next-Generation Mini-Gene Payloads for AAV Gene Therapies for the Treatment of Genetic Epilepsies and Additional CNS Disorders

BioTech 365

Biologists create new genetic systems to neutralize gene drives

Scienmag

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

Gene therapies offer great reward in the form of treating various devastating diseases, but there are also significant risks