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

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

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. 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

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

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

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

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

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 two therapies have been tipped to generate $1.5

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

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.

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.

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

Novartis’ bid to expand use of SMA gene therapy delayed after FDA trial request

Pharma Phorum

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

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

Investigational gene therapy was developed by professor Brian Bigger

Gene therapy company Taysha completes sprint from first funding to IPO

Bio Pharma Dive

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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®

On June 3, members of the research community gathered virtually to discuss updates on hot topics and emerging issues in gene therapy. General