Pharmaceutical Technology

APRIL 15, 2024

Discover the groundbreaking gene therapy patent by Ginkgo BioWorks Inc for treating NPC1 using AAV vectors. Learn how this innovative approach offers hope for rare genetic disorders like Neimann-Pick Disease Type C.

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Gene Sequencing Gene Gene Therapy Genetics 130

pharmaphorum

AUGUST 10, 2021

Europe market has proved so hostile to gene therapies when it comes to pricing and reimbursement for gene therapies that bluebird bio has decided to quit the market altogether, according to Andrew Obenshain, president of its severe genetic diseases unit. — Brad Loncar (@bradloncar) August 9, 2021.

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Gene Therapy Gene Marketing Genetic Disease 135

pharmaphorum

FEBRUARY 23, 2022

Takeda has forged another alliance as it continues a push into gene therapy, agreeing a deal worth up to $2 billion with Code Biotherapeutics for opt-in rights to four candidates for rare diseases. The post Takeda grows in gene therapies again with $2bn Code Bio deal appeared first on. Last October it signed a $3.6

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Gene Therapy Gene Gene Sequencing Immune Response 104

pharmaphorum

SEPTEMBER 14, 2022

SparingVision has raised €75 million in second-round funding that will be used to fund clinical trials of gene therapies for ocular diseases retinitis pigmentosa (RP) and dry age-related macular degeneration (AMD). The post SparingVision raises €75m for eye disease gene therapies appeared first on.

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Gene Therapy Gene Gene Sequencing Gene Editing 52

pharmaphorum

JUNE 23, 2022

Systemic administration of gene-editing drugs would avoid that process with an “off-the-shelf” option for a one-shot, potentially curative treatment for diseases associated with genetic mutations. billion agreement that started in 2020.

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Gene Editing In-Vivo Gene Sequencing Gene 91

pharmaphorum

FEBRUARY 22, 2021

Bluebird Bio’s decision to hit pause on the launch of Zynteglo for beta thalassaemia after two cases of cancer were seen in a clinical trial could see fears over the safety of viral vectors used to deliver gene therapies resurface. . million in Europe.

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Gene Therapy Gene Gene Sequencing Gene Expression 52

XTalks

DECEMBER 14, 2022

We wanted to work toward what gene therapy was supposed to do at its original inception, which is to replace genes, not just correct individual mutations.”. The PASTE gene editing technique was recently published in Nature Biotechnology.

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