Pharmaceutical Technology

JANUARY 31, 2023

Gene therapy company uniQure has entered into a global licensing agreement with Apic Bio for APB-102 to treat patients with amyotrophic lateral sclerosis (ALS) caused by mutations in superoxide dismutase 1 (SOD1). APB-102 is designed to be a one-time, intrathecally administered gene therapy for ALS patients.

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Gene Therapy Licensing Licensing Gene 278

XTalks

OCTOBER 26, 2021

After initial rejection from the National Institute for Health and Care Excellence (NICE) last year, the non-departmental public body of the Department of Health in England has now given the green light to the gene silencing treatment Givlaari (givosiran) for the treatment of the rare metabolic disorder, acute intermittent porphyria (AIP).

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Gene Silencing Gene Clinical Trials Clinical Trials 98

BioSpace

JUNE 16, 2024

The plethora of genes involved in obesity presents an intriguing opportunity for both gene silencing and ex vivo gene therapy approaches.

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Gene Silencing In-Vivo Genetics Gene Therapy 93

Pharmaceutical Technology

FEBRUARY 20, 2023

Different approaches that are studied include antisense oligonucleotides (ASOs), and gene therapies, which are in early clinical trials. In August 2022, Passage Bio dosed the first subject in a Phase I/II trial for their FTD gene therapy PBFT02. The company dosed its first patient in this study in December 2020.

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Development Gene Therapy Protein Gene 246

Scienmag

JULY 2, 2021

A newly developed CRISPR-Cas9-based tool carries out efficient and long-term gene silencing by epigenetic editing Credit: BioDesign Research Originally discovered as a bacterial mode of defense against invading viruses, the remarkable ability of CRISPR-Cas9 to modify specific locations of DNA has made it a researcher favorite among gene editing tools. (..)

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Gene Silencing Gene Editing Gene DNA 75

pharmaphorum

OCTOBER 29, 2020

Already a major player in gene therapy, Novartis has swooped on US startup Vedere Bio in a $280 million deal that builds its position in inherited eye diseases that can lead to blindness. Novartis’ big move in the gene therapy market came when it bought AveXis for $8.7

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Gene Therapy Gene Gene Silencing Protein 40

XTalks

MAY 4, 2021

The tool could also prove to be safer than conventional CRISPR-based gene therapies as it does not involve DNA editing, and thus would not cause potentially harmful off-target genomic changes.

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DNA Gene Gene Silencing Genome 98