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.

Gene Therapy 289

Gene Therapy FDA Approval Gene Genetic Disease 289

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.

Drugs 246

Drugs FDA Approval Marketing Development 246

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.

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Gene Therapy Gene Marketing Trials 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

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Gene Therapy Gene Genetic Disease Manufacturing 60

XTalks

DECEMBER 13, 2023

In 2022, bluebird won approvals for two gene therapies — Skysona for the treatment of the rare neurological disorder cerebral adrenoleukodystrophy (CALD) and Zynteglo for beta-thalassemia. The company has been facing financial woes despite the big approvals. Vertex-CRISPR’s Casgevy has a US list price of $2.2

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Gene Therapy FDA Approval Gene In-Vivo 98

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.

Gene Therapy 52

Gene Therapy FDA Approval Gene Clinical Trials 52

XTalks

SEPTEMBER 27, 2022

Skysona received FDA approval for the treatment of the rare neurological disorder cerebral adrenoleukodystrophy (CALD). The record-breaking price tag of Skysona is not surprising for one-time gene therapies like it. Related: Bluebird’s $2.8M Gene Therapy Zynteglo Wins Landmark FDA Approval for Beta Thalassemia.

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FDA Approval Gene Therapy Drugs Gene 52