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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 Therapy 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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Trending Sources
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
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
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. The post Axovant pushes on with Parkinson’s gene therapy, despite investor cynicism appeared first on.
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
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
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
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
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 two therapies have been tipped to generate $1.5
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
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.
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.
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
Novartis’ bid to expand use of SMA gene therapy delayed after FDA trial request
Pharma Phorum
SEPTEMBER 23, 2020
Analysts have said the FDA’s request for a new trial will delay Novartis’ intrathecal formulation of its spinal muscular atrophy (SMA) gene therapy in older patients until 2023, potentially benefiting a rival from Roche.
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
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
Gene therapy company Taysha completes sprint from first funding to IPO
Bio Pharma Dive
SEPTEMBER 24, 2020
Led by alumni of Zolgensma developer AveXis, the university spinout could have four experimental therapies in the clinic by the end of 2021
With latest data, a gene therapy for a deadly brain disease inches closer to market
Bio Pharma Dive
AUGUST 27, 2020
The results keep Bluebird Bio on track to seek approval of the experimental treatment, known as eli-cel, in Europe later this year and in the U.S. in 2021
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
Roche deal latest in AAV gene therapy partnership plan for Dyno
BioPharma Reporter
OCTOBER 19, 2020
US company, Dyno Therapeutics, has inked a deal with Roche to develop next-generation AAV gene therapy vectors for central nervous system (CNS) diseases and liver-directed therapies.
AVROBIO Expands Lentiviral Gene Therapy Pipeline with Program for Hunter Syndrome
BioTech 365
OCTOBER 5, 2020
Investigational gene therapy licensed from The University of Manchester, UK aims to address both physical and CNS manifestations of Hunter syndrome Expands leading pipeline targeting lysosomal disorders with 5th investigational gene therapy Program expected to enter Phase 1/2 clinical trial … Continue reading → Business Wire
Taysha Gene Therapies Receives Rare Pediatric Disease Designation and Orphan Drug Designation for TSHA-102 as a Treatment for Rett Syndrome
BioTech 365
OCTOBER 14, 2020
Program leverages novel miRARE platform technology used to control transgene expression on a cellular basis TSHA-102 anticipated to submit Investigational New Drug application in 2021 DALLAS–(BUSINESS WIRE)–Taysha Gene Therapies Inc.
#news #biotech Passage Bio slapped with gene therapy clinical hold
BioTech 365
AUGUST 13, 2020
Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Passage Bio slapped with gene therapy clinical hold.Passage Bio slapped with gene therapy clinical hold ntaylor Thu, 08/13/2020 – 08:31 from FierceBiotech: Biotech [link].
Fujifilm expands its gene therapy drug CDMO business
BioPharma Reporter
OCTOBER 7, 2020
Fujifilm is boosting its gene therapy offering, taking the first steps to expand its viral vector contract development and manufacturing organization (CDMO) services to Europe, at the site of Fujifilm Diosynth Biotechnologies (FDB) in the North East of the UK.
FDA delays BioMarin’s gene therapy for haemophilia A
The Pharma Data
AUGUST 20, 2020
The FDA has delayed Roctavin, BioMarin’s gene therapy for haemophilia A, by a year to wait for more extensive results, it has been revealed. . We are confident in Roctavian gene therapy and its potential to redefine the treatment paradigm for people with haemophilia A.
#news #biotech SparingVision raises €44.5M to develop mutation-agnostic gene therapy
BioTech 365
OCTOBER 20, 2020
to develop mutation-agnostic gene therapy.SparingVision raises €44.5M to develop mutation-agnostic gene therapy ntaylor Tue, 10/20/2020 – 11:36 from FierceBiotech: Biotech [link]. Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: SparingVision raises €44.5M
FDA rejection of BioMarin gene therapy raises durability questions
BioPharma Reporter
AUGUST 20, 2020
The US Food and Drug Administration (FDA) has rejected approval of BioMarinâs hemophilia A gene therapy amid durability doubts. Bio Developments
EU review extended for BioMarin's gene therapy Valrox
The Pharma Data
SEPTEMBER 10, 2020
European regulators have requested more data to review the marketing application for BioMarin’s haemophilia A gene therapy Valrox (valoctocogene roxaparvove).
FDA approves Phase 1 trial for HIV gene therapy
The Pharma Data
AUGUST 24, 2020
The FDA has approved a request from American Gene Technologies to begin a clinical study into its HIV gene therapy. In a paper published in Molecular Therapy: Methods & Clinical Development , the team discussed why they choose this type of gene therapy to treat HIV.
Vertex, CRISPR Therapeutics’ sickle cell gene therapy wins PRIME designation
Pharma Times
SEPTEMBER 23, 2020
European Medicines Agency grants designation based on phase I/II data
Disruptor in cell and gene therapy space secures US$30m in funding
BioPharma Reporter
OCTOBER 20, 2020
Ori Biotech Ltd (Ori), a London and New Jersey based innovator in the cell and gene therapy (CGT) manufacturing sphere, last week announced the successful close of a US$30m Series A financing round, bringing the companyâs total funding to date to US$41m.
#news #biotech Avrobio bags gene therapy to join Regenxbio and Sangamo in race
BioTech 365
OCTOBER 5, 2020
Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Avrobio bags gene therapy to join Regenxbio and Sangamo in race.Avrobio bags gene therapy to join Regenxbio and Sangamo in race ntaylor Mon, 10/05/2020 – 08:43 from … Continue reading → #news
#news #biotech FDA lifts clinical hold on Solid Bio gene therapy trial
BioTech 365
OCTOBER 2, 2020
Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: FDA lifts clinical hold on Solid Bio gene therapy trial.FDA lifts clinical hold on Solid Bio gene therapy trial ntaylor Fri, 10/02/2020 – 06:07 from FierceBiotech: Biotech … Continue reading → #news
Bringing the Gene Therapy Conversation to You – Virtually
Forte Research Systems®
JUNE 11, 2020
On June 3, members of the research community gathered virtually to discuss updates on hot topics and emerging issues in gene therapy. General
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