Drug Discovery World

OCTOBER 26, 2022

The new method involves adding chemical tags to sugar molecules which are added to cells. The researchers genetically modify the cell type they want to study, so that only this type adds the sugar to its proteins. Click’ chemistry .

Protein 52

Protein Genetics Containment Research 52

Pharmaceutical Technology

MAY 2, 2023

Chimeric antigen receptor T-cell (CAR-T) therapies are the only genetically modified cell therapies to have received regulatory approval, and they are currently utilised in relapsed/refractory settings. Cost-related unmet needs also scored highly.

Genetic Engineering 130

Genetic Engineering Antibody Clinical Trials Clinical Trials 130

pharmaphorum

JANUARY 9, 2023

First introduced in 2020, the global Managed Access Programme (gMAP) has provided Zolgensma (onasemnogene abeparvovec) free of charge to nearly 300 children with the genetic disorder across 36 countries where the therapy has not yet received approval or in which no formal access pathway exists.

Gene Therapy 118

Gene Therapy Genetics Gene Clinical Trials 118

XTalks

DECEMBER 13, 2023

Both Casgevy and Lyfgenia involve modifying the hematopoietic stem cells of patients with the disorder (to produce normal hemoglobin) and then injecting them back into patients, leading to the generation of red blood cells with normal, functional hemoglobin. Vertex-CRISPR’s Casgevy has a US list price of $2.2

Gene Therapy 98

Gene Therapy FDA Approval Gene In-Vivo 98

XTalks

JUNE 8, 2021

SMA is a rare genetic condition that leads to a loss of motor neurons that results in progressive muscle weakness and wasting, paralysis and, when left untreated in its most severe form, breathing difficulties leading to permanent ventilation or death for most patients by the age of two. With a price tag of over $2.5

Gene Therapy 110

Gene Therapy Gene Protein Trials 110

Pharmaceutical Technology

APRIL 5, 2023

But access to these treatments continues to remain limited due to high price tags and variable availability across regions. Currently, all approved CAR-T therapies are autologous, where T cells taken from patients are modified and then re-infused. But there is that significant challenge. There has been extremely rapid progress.

Gene Therapy 147

Gene Therapy Gene Engineer Production 147

pharmaphorum

AUGUST 18, 2022

Zynteglo is a customised, one-time treatment created using a patient’s own bone marrow stem cells hat are genetically modified to produce functional beta-globin, which is mutated in the disease. million price tag attached to the therapy when it was first made available in Europe, and is also well above the proposed $2.1

Gene Therapy 52

Gene Therapy FDA Approval Gene Clinical Trials 52