gene therapy

Showing 15 posts of 41 posts found.

Rocket Pharmaceuticals granted FDA RMAT designation for gene therapy

May 24, 2023
Medical Communications FDA, Haematology, RMAT, Rocket Pharmaceuticals, gene therapy

Rocket Pharmaceuticals, a US-based biotechnology company, has announced that it has received US Food and Drug Administration (FDA) Regenerative Medicine …

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Krystal Biotech secures FDA approval for redosable topical gene therapy

May 22, 2023
Medical Communications FDA, Krystal Biotech, Paediatrics, dystrophic epidermolysis bullosa, gene therapy

US-based biotech company Krystal Biotech has announced that the US Food and Drug Administration (FDA) has approved its topical gene …

Nemours Children’s Health selected to conduct first gene therapy for Morquio A syndrome

May 17, 2023
Research and Development Morquio A syndrome, NIH, Nemours Children's Health, Paediatrics, gene therapy

Nemours Children’s Health, based in Delaware, US, has been chosen by the Foundation for the National Institutes of Health (FNIH) …

FDA pre-meeting report raises concerns over Sarepta’s gene therapy for Duchenne muscular dystrophy

May 11, 2023
Medical Communications Duchenne Muscular Dystrophy, FDA, Musculo-skeletal disorder, Sarepta Therapeutics, gene therapy

The US Food and Drug Administration (FDA) has released a report in preparation for its 12 May decision on Sarepta …

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Biogen and Capsigen to collaborate on novel gene therapies

May 11, 2021
Manufacturing and Production gene therapy

Biogen and Capsigen have announced a research collaboration to engineer novel adeno-associated virus (AAV) capsids that have the potential to …

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Novartis reveals hard-hitting five-year data for its gene therapy Zolgensma in spinal muscular atrophy

March 25, 2020
Research and Development Novartis, Zolgensma, gene therapy, pharma, spinal muscular atrophy

Novartis has revealed five-year data on its gene therapy Zolgensma, boasting that the one-time infusion demonstrated “rapid, significant and clinically …

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Bluebird bio’s Lenti-D gene therapy shows promise in cerebral adrenoleukodystrophy

September 23, 2019
Research and Development, Sales and Marketing Bluebird Bio, cerebral adrenoleukodystrophy, gene therapy, pharma

Bluebird bio has revealed updated data for its investigational Lenti-D gene therapy in the treatment of cerebral adrenoleukodystrophy (CALD), a …

NICE recommends Novartis’ gene therapy Luxturna for inherited retinal dystrophy

September 4, 2019
Research and Development, Sales and Marketing Luxturna, NHS, NICE, UK, gene therapy, pharma

NICE has chosen to recommend the use of Novartis’ one-time gene therapy Luxturna (voretigene neparvovec) for use on the NHS …

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Novartis hid faulty data from FDA over $2.1 million gene therapy Zolgensma

August 7, 2019
Research and Development AveXis, FDA, Novartis, Zolgensma, gene therapy, pharma

Novartis hid faulty data from the FDA until their drug was approved, according to a statement from Dr Peter Marks, …

EMA approves Bluebird Bio’s gene therapy Zynteglo

June 4, 2019
Manufacturing and Production Bluebird Bio, European comission, gene therapy, health, pharma

The European Commission has granted a conditional marketing authorisation to bluebird bio’s gene therapy Zynteglo. The Massachusetts-based firm’s one time …

Doctors cure ‘bubble boy syndrome’ using modified version of HIV

April 18, 2019
Research and Development HIV, SCID-X1, gene therapy, immunodeficiency, immunology

Doctors at St. Jude Children’s Research Hospital have been able to treat patients with X-linked severe combined immunodeficiency (SCID-X1) – …

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CHMP recommendation for bluebird bio’s gene therapy in β-thalassaemia

April 1, 2019
Research and Development, Sales and Marketing Bluebird Bio, CHMP, EMA, Zynteglo, gene therapy, β-thalassaemia

Massachusetts-based bluebird bio has taken a significant leap forward in its pursuit of regulatory authorisation for its first gene therapy, …

Roche set to acquire Spark Therapeutics in $4.8 billion deal

February 25, 2019
Sales and Marketing Roche, Spark Therapeutics, acquisition, gene therapy, pharma

Roche is set to fully acquire gene therapy Spark Therapeutics after entering into a definitive merger agreement, after the Boards …

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Janssen seals global inherited retinal disease partnership with gene therapy firm MeiraGTx

February 1, 2019
Medical Communications, Research and Development, Sales and Marketing Janssen, MeiraGTx, eye care, gene therapy, pharma

In a bid to boost its portfolio in the eye care space, Janssen has announced a global partnership with clinical-stage …

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Scientists prevent axon self-destruct mechanism using gene therapy

January 17, 2019
Research and Development Axon, gene therapy, health, neuropathy

Scientists at the University of Washington have developed a gene therapy that prevents the process through which axons (nerve fibres) …

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