PharmaShots

JANUARY 27, 2023

Magenta Therapeutics Pauses the P-I/II Study in AML Patients Date: Jan 27, 2023 | Tags: Magenta Therapeutics, MGTA-117, AML, Clinical Trial, P-I/II Ipsen Receives CHMP Negative Opinion for Palovarotene to Treat Fibrodysplasia Ossificans Progressiva Date: Jan 27, 2023 | Tags: Ipsen, Palovarotene, Fibrodysplasia Ossificans Progressiva, Regulatory, CHMP, (..)

FDA 40

FDA Immunization 40

Pharmaceutical Technology

SEPTEMBER 30, 2022

Skysona is indicated as a one-time gene therapy to slow the progression of cerebral adrenoleukodystrophy (CALD), a rare paediatric neurodegenerative disease in boys aged 4–17 years diagnosed with early-stage CALD. These approvals represent crucial milestones for bluebird bio, the gene therapy field, and patients with rare genetic diseases.

FDA 115

FDA Insurance 115

pharmaphorum

JUNE 12, 2022

Thursday saw the Cellular, Tissue and Gene Therapies advisory committee back elivaldogene autotemcel (eli-cel) for cerebral adrenoleukodystrophy (CALD) – see our report here – and the following day it was the turn of betibeglogene autotemcel (beti-cel) for beta thalassaemia.

FDA 59

FDA 59

pharmaphorum

JANUARY 11, 2021

The gene therapy firm will be focused on its most important therapies in beta-thalassemia, cerebral adrenoleukodystrophy and sickle cell disease in the US And Europe. million price tag. Zynteglo is already in beta-thalassemia in Europe, where the company will seek to expand access despite its hefty $1.8

FDA 66

FDA 66

pharmaphorum

AUGUST 18, 2022

million price tag attached to the therapy when it was first made available in Europe, and is also well above the proposed $2.1 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.

FDA 52

FDA Insurance 52

Pharmaceutical Technology

JUNE 29, 2022

The US Food and Drug Administration (FDA) put a high-profile bluebird bio trial for sickle cell disease on partial clinical hold, and advisory panels deliberated over decisions involving gene therapies for amyotrophic lateral sclerosis (ALS), cerebral adrenoleukodystrophy (CALD), and beta-thalassemia.

Pharmaceutical Companies 98

Pharmaceutical Companies Pharmaceutical Companies FDA 98