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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
Gene Related Topics 
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
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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
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
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 […].
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
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
Catalent to invest $130M in gene therapy plant near Baltimore airport
Bio Pharma Dive
SEPTEMBER 10, 2020
The contract manufacturer is riding a boom in demand for both its gene therapy and vaccine manufacturing services
Investigating host tolerance to genes that jump
Scienmag
AUGUST 31, 2020
million grant Credit: University of Houston Believe it or not, your genes are 50% parasite, and opportunistic at that. Medicine & Health Biochemistry Biology Biotechnology Cell Biology Developmental/Reproductive Biology Genes Genetics Medicine/Health Microbiology Molecular Biology
New gene implicated in neuron diseases
Scienmag
SEPTEMBER 16, 2020
Medicine & Health Biology Cell Biology Genes Genetics Molecular Biology Neurobiology
Janssen/MeiraGTx showcase nine-month data for sight loss gene therapy
Pharma Times
OCTOBER 5, 2020
Gene therapy demonstrated improvement in retinal sensitivity
Epilepsy gene therapy candidate moves closer to clinical study phase
BioPharma Reporter
SEPTEMBER 17, 2020
Gene therapy developer, CombiGene, and CDMO, Cobra Biologics, have signed off on a deal to secure Good Manufacturing Practice (GMP) production of two essential plasmids needed for the manufacture of CombiGene's gene therapy, CG01.
Pfizer: Expanding North Carolina Gene Therapy Facility and Adding Jobs
BioSpace
OCTOBER 18, 2020
Pfizer is continuing to invest in gene therapy research and development and other areas going on throughout the company, specifically in its North Carolina sites in Chapel Hill and Kit Creek
IPK scientists discover gene that ensures slim inflorescence shape of barley
Scienmag
OCTOBER 12, 2020
Biology Agricultural Production/Economics Biodiversity Genes Plant SciencesCredit: Photo: Nadja Sonntag The “spikelet meristem” (SM) plays a central role during the development of the grass inflorescence.
About 14% of cerebral palsy cases may be tied to brain wiring genes
Scienmag
SEPTEMBER 28, 2020
NIH-funded study points to genes that control the establishment of neural circuits during early development Credit: Courtesy of Dreamstime | ©Jarenwicklund. Medicine & Health Bioinformatics Genes Genetics Medicine/Health Neurobiology Pediatrics
FDA sets back Novartis plans to expand use of SMA gene therapy
Bio Pharma Dive
SEPTEMBER 23, 2020
The agency has asked Novartis to run a new study of the Zolgensma formulation that Novartis aims to bring to older patients with spinal muscular atrophy
New CEO Mortazavi takes UK biotech e-therapeutics into gene silencing
Pharma Phorum
OCTOBER 13, 2020
UK biotech e-therapeutics has a new CEO, with executive chairman and former Silence Therapeutics chief Ali Mortazavi chosen to spearhead the next stage in the company’s development into gene silencing and other areas. News R&D Biotech e-Therapeutics gene silencing inflammation people
Axovant pushes on with Parkinson’s gene therapy, despite investor cynicism
Pharma Phorum
OCTOBER 7, 2020
Axovant has said it plans to continue developing its Parkinson’s Disease gene therapy after reporting supportive data from a small cohort of patients from a phase 2 trial. Parkinson’s is not a true genetic disease, although certain genes have been linked to an increased likelihood of onset.
FDA puts Voyager study on hold in latest gene therapy speed bump
Bio Pharma Dive
OCTOBER 13, 2020
The biotech won't be able to start human testing of an experimental Huntington's disease treatment until regulators see more manufacturing data
BioMarin's hemophilia gene therapy could have warranted a record price tag, ICER finds
Bio Pharma Dive
AUGUST 28, 2020
A draft report from the drug pricing watchdog concluded that, under the right conditions, Roctavian may be cost-effective at a price of $2.5 million
Ancient gene family protects algae from salt and cold in an Antarctic lake
Scienmag
AUGUST 19, 2020
Chemistry AND Physics Biochemistry Biodiversity Biology Ecology/Environment Evolution Genes Marine/Freshwater Biology Microbiology Physiology
Pfizer and Sangamo, just after a rival's setback, kick off late-stage gene therapy trial
Bio Pharma Dive
OCTOBER 7, 2020
The FDA's recent rejection of a hemophilia therapy from BioMarin sets a high data standard for approval. Will Pfizer fare any better
New data bolsters Sarepta gene therapies for two deadly muscle diseases
Bio Pharma Dive
SEPTEMBER 28, 2020
The results disclosed at a medical meeting on Monday show that all patients tested have seen their motor skills stabilize or improve when history suggests they wouldn't
Mosquitos lost an essential gene with no ill effects
Scienmag
SEPTEMBER 30, 2020
UMD study suggests caution is needed for research that presumes closely related species use the same genes for similar functions Credit: Image credit: Alys Jarvela/University of Maryland.
Reducing barriers to mainstream gene therapy
BioPharma Reporter
SEPTEMBER 3, 2020
AskBio, which develops adeno-associated virus (AAV) gene therapies for genetic disorders, has won an R&D grant valued at Â2m (around US$2.7m) from Scottish Enterprise.
American Gene Technologies Gets Go-Ahead to Test Possible HIV Cure
BioSpace
AUGUST 12, 2020
American Gene Technologies (AGT), based in Rockville, Maryland, received the go-ahead from the U.S. Food and Drug Administration (FDA) to begin a Phase I clinical trial of AGT103-T, a gene therapy, to eliminate HIV from infected individuals
New HIV Gene Therapy, CAR-T Treatments Could be on the Horizon for Patients
BioSpace
SEPTEMBER 2, 2020
Could gene therapy provide a solution to HIV? A new research project aims to find out
New gene therapy approach eliminates at least 90% latent herpes simplex virus 1
Scienmag
AUGUST 18, 2020
In Nature Communications, Fred Hutch scientists demonstrate in a mouse model how gene therapy could cure an infection that afflicts billions of people Infectious disease researchers at Fred Hutchinson Cancer Research Center have used a gene editing approach to remove latent herpes simplex virus 1, or HSV-1, also known as oral herpes. Medicine & Health Biotechnology Gene Therapy Infectious/Emerging Diseases Medicine/Health
Pfizer: Expanding North Carolina Gene Therapy Facility and Adding Jobs
The Pharma Data
OCTOBER 18, 2020
In late August, Pfizer announced it was investing an additional $500 million into its state-of-the-art gene therapy manufacturing facility in Sanford, North Carolina. Ricci seemed excited about gene therapy and the part it is playing increasingly in Pfizer’s core mission.
EMA starts rapid review of Bluebird’s gene therapy for rare disease CALD
Pharma Phorum
OCTOBER 4, 2020
Bluebird bio could be just a few months away from approval of its gene therapy for rare disease cerebral adrenoleukodystrophy (CALD) in the EU, after the EMA started an accelerated review. The post EMA starts rapid review of Bluebird’s gene therapy for rare disease CALD appeared first on.
New approach uses wild genes to improve biological nitrogen fixation in soybeans
Scienmag
AUGUST 20, 2020
Biology Agricultural Production/Economics Agriculture Ecology/Environment Food/Food Science Genes Genetics Molecular Biology Plant SciencesCredit: Thi Nguyet Minh Dao and Svetlana Y. Folimonova.
Newly discovered gene may give ‘sea pickles’ their glow
Scienmag
OCTOBER 20, 2020
Finding could be first bioluminescent gene identified in a chordate, may be common across tree of life Credit: OceanX A new study describes a bioluminescent gene that could be the reason that so-called “sea pickles,” or pyrosomes, an underwater free-floating colony of thousands of tiny animals, reverberate in blue-green light.
Uni of Manchester partners with AVROBIO for Hunter syndrome gene therapy
Pharma Times
OCTOBER 6, 2020
Investigational gene therapy was developed by professor Brian Bigger
Study discovers gene that helps us know when it’s time to urinate
Scienmag
OCTOBER 19, 2020
Results suggest ‘sixth sense’ PIEZO2 gene may help body sense a full urinary bladder Credit: Courtesy of Patapoutian lab, Scripps Researcher Institute, La Jolla, CA.
Small Biopharma Companies Emerging in Gene and Cell Therapy
BioSpace
SEPTEMBER 2, 2020
Biospace recently analyzed key emerging cell and gene therapy companies to get a handle on how this evolving science is beginning to make its way from research into clinical applications
Gene Pulser Xcell CE Module
BioTech 365
SEPTEMBER 2, 2020
£500.00End Date: Friday Sep-11-2020 15:51:41 BSTBuy It Now for only: £500.00Buy It Now | Add to watch list Biotech365 : Gene Pulser Xcell CE Module BioMarketplace You want to propose your products or a Biotech Company, a Biotech Tool or … Continue reading → #BioMarketplace #BioMarketplace_UK #Biorad
Scientists found genes that help cancer cells to penetrate the brain
Scienmag
AUGUST 11, 2020
An international team of scientists, including a researcher from Sechenov University, reviewed scientific articles on proteins (and genes encoding them) that help cancer cells enter the brain. Medicine & Health cancer Genes Medicine/HealthAn understanding of the processes that facilitate the formation of metastases in the brain will allow scientists to create new methods for cancer diagnosis and treatment. Details of […].
FDA Grants Fast Track Designation to Pfizer's DMD Gene Therapy
BioSpace
SEPTEMBER 30, 2020
PF-06939926 is currently being evaluated to determine the safety and efficacy of this gene therapy in boys with DMD
Solid gets all-clear from FDA to restart gene therapy trial
Bio Pharma Dive
OCTOBER 1, 2020
Worrisome immune responses had led the FDA to halt Solid's study. Now, the agency will permit Solid to continue after the biotech made adjustments to its manufacturing and patient enrollment procedures
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