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

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

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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

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

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 […].

Gene 56

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

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

Catalent to invest $130M in gene therapy plant near Baltimore airport

Bio Pharma Dive

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

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

Gene 56

New gene implicated in neuron diseases

Scienmag

Medicine & Health Biology Cell Biology Genes Genetics Molecular Biology Neurobiology

Gene 56

Janssen/MeiraGTx showcase nine-month data for sight loss gene therapy

Pharma Times

Gene therapy demonstrated improvement in retinal sensitivity

Epilepsy gene therapy candidate moves closer to clinical study phase

BioPharma Reporter

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

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

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.

Gene 56

About 14% of cerebral palsy cases may be tied to brain wiring genes

Scienmag

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

Gene 56

FDA sets back Novartis plans to expand use of SMA gene therapy

Bio Pharma Dive

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

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

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

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

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

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

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

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

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.

Gene 56

Reducing barriers to mainstream gene therapy

BioPharma Reporter

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

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

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

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

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

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

Biology Agricultural Production/Economics Agriculture Ecology/Environment Food/Food Science Genes Genetics Molecular Biology Plant SciencesCredit: Thi Nguyet Minh Dao and Svetlana Y. Folimonova.

Gene 56

Newly discovered gene may give ‘sea pickles’ their glow

Scienmag

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.

Gene 40

Scientists identify gene family key to unlocking vertebrate evolution

Scienmag

Uni of Manchester partners with AVROBIO for Hunter syndrome gene therapy

Pharma Times

Investigational gene therapy was developed by professor Brian Bigger

Study discovers gene that helps us know when it’s time to urinate

Scienmag

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

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

£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

Gene 40

Scientists found genes that help cancer cells to penetrate the brain

Scienmag

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

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

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