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).

Gene Silencing 98

Gene Silencing Gene Clinical Trials Clinical Trials 98

Drug Discovery World

OCTOBER 4, 2022

This year’s event will cover some of the most exciting advancements in areas spanning screening, automation, high content imaging, disease models, cell and gene therapies and how innovation is being driven through partnerships and collaborations. Cell and gene therapies. billion, compared to $19.9

Gene Therapy 52

Gene Therapy Drugs Gene Manufacturing 52

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.

Development 246

Development Gene Therapy Protein Gene 246

XTalks

FEBRUARY 14, 2022

The drug’s approval in the EU was based on analysis of clinical trial data including both pre- and early-symptomatic, and early-onset MLD patients, which showed that a single-dose treatment is effective in modulating disease progression in most early-onset MLD patients.

Gene Therapy 98

Gene Therapy Drugs Gene Gene Silencing 98

Drug Discovery World

JANUARY 23, 2023

To avoid these histocompatibility challenges, early clinical trials often opt for an autologous graft-based approach 8,9 to subvert the need for prolonged immunosuppressive treatment and streamline the grafting process. Regenerative iPSC-derived therapies are not just limited to ophthalmic diseases. 2007;131:861-872.

Gene Editing 52

Gene Editing Gene Engineer DNA 52