XTalks

JUNE 28, 2023

million price tag of Elevidys, a one-time gene therapy. The progression of DMD is often rapid, and those affected usually face a significantly shortened lifespan with severe physical limitations. Options for managing the symptoms of DMD have been limited. In a media call, Sarepta CEO Douglas Ingram revealed the $3.2

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XTalks

AUGUST 19, 2022

Zynteglo’s hefty price tag of $2.8 What’s happening to them is not unique to bluebird,” said Nicole Paulk, an assistant professor of AAV gene therapy at the University of California, San Francisco in a Biopharma Dive report. The cells are prepared outside of the body ( ex vivo ) and then infused into the patient.

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pharmaphorum

FEBRUARY 22, 2021

That therapy uses an adeno-associated virus (AAV) as a vector rather than a lentivirus, a vector type that features in approved gene therapies such as Roche’s Luxturna and Novartis’ Zolgensma. The case comes just weeks after UniQure halted a trial of its haemophilia B gene therapy AMT-061 after an unexpected case of liver cancer.

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XTalks

JUNE 8, 2021

Zolgensma is an adeno-associated virus vector (AAV)-based gene therapy that delivers a functional copy of the SMN1 gene into target motor neurons to replace the mutant copy. With a price tag of over $2.5 million, Zolgensma is currently the world’s most expensive drug. Why is Zolgensma So Expensive?

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Gene Therapy Gene Protein Trials 110

Pharmaceutical Technology

JUNE 21, 2023

The high price tag may be hard to defend , but with lifetime treatment costs for haemophilia B reaching as high as $23m for some, the single-dose treatment could prove far more cost-effective. Despite the limited market, the price tags of haemophilia make it potentially lucrative. Haemophilia B is a rare condition.

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Delveinsight

DECEMBER 3, 2020

It has been speculated that modifications to the epigenome drive cell ageing, especially a process known as DNA methylation, by which methyl groups are tagged onto DNA. Shinya Yamanaka discovered that the factors could alter the epigenome, and in that way modify mature cells back to a stem cell-like state.

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Gene Therapy Gene Antibody Research 52

pharmaphorum

AUGUST 26, 2022

Roctavian delivers a working copy of the Factor VIII gene into cells via an adeno-associated virus (AAV) vector, and offers a one-shot, potentially curative therapy for haemophilia A. million spread over five years.

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