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.

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119

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.

Immunization 52

Immunization Vaccines 52

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

FDA 52

FDA Insurance 52

pharmaphorum

DECEMBER 23, 2022

price tag of $475,000 when it was first launched in 2017 – and it becomes apparent that these may not be desirable treatment options for every patient and in every setting. Add to this the considerable cost of these medications – the first approved CAR-T, Novartis’ Kymriah (tisagenlecleucel), had a U.S.

Immunization 52

Immunization Hospitals Vaccines 52

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.

FDA 59

FDA Immunization 59

European Pharmaceutical Review

DECEMBER 27, 2023

This is in addition to its considerable price tag. He established the first public cord blood bank in Israel and performed the first cord blood transplantations from related and unrelated donors in genetic and malignant haematological diseases. Chimeric antigen receptor–modified T cells for acute lymphoid leukemia. Cytotherapy.

Adverse Reactions 85

Adverse Reactions Immunization Medical Schools Chemotherapy 85

European Pharmaceutical Review

NOVEMBER 10, 2022

Modifying or developing targeting moieties is necessary to improve extrahepatic delivery. BT-474 cell line was gene-edited to incorporate a green fluorescent protein (GFP) tag into late endosomes/lysosomes (green). Physical characterisation. Lorna has over ten years working in an academic and industrial setting. During her PhD at St.

Labelling 97

Labelling Compounding 97