Drug Discovery World

APRIL 10, 2024

iECURE has been given the go-ahead to begin US trials of its investigational gene editing-based therapy in newborn males with neonatal onset Ornithine Transcarbamylase (OTC) deficiency. The post FDA approves first in vivo gene insertion programme in infants appeared first on Drug Discovery World (DDW).

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In-Vivo FDA Approval Gene Gene Editing 80

Drug Discovery World

AUGUST 14, 2023

Lung-I Cheng, Vice President and Head of Cell & Gene Therapy Service Line at AmerisourceBergen, and Cori Gorman, Senior Director of CMC and Regulatory Affairs at Biopharma Excellence, offer advice on navigating the different and sometimes contradictory regulatory requirements in the US and EU. billion in 2020 to $15.5

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Gene Therapy Marketing Gene DNA 52

pharmaphorum

SEPTEMBER 28, 2022

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. The time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market.

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Gene Editing Gene In-Vivo Clinical Trials 57

XTalks

AUGUST 19, 2022

Bluebird bio’s gene therapy Zynteglo (betibeglogene autotemcel, beti-cel) has been awarded a much anticipated approval from the US Food and Drug Administration (FDA) for the treatment of adult and pediatric patients with beta thalassemia who need regular blood transfusions. Zynteglo’s hefty price tag of $2.8

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Gene Therapy FDA Approval Gene Clinical Trials 95

Pharmaceutical Technology

SEPTEMBER 19, 2022

The US Food and Drug Administration (FDA) has awarded Accelerated Approval for bluebird bio ’s Skysona (elivaldogene autotemcel; eli-cel) for slowing neurologic dysfunction progression in early, active cerebral adrenoleukodystrophy (CALD) patients. The treatment is indicated for usage in boys aged four to 17 years with CALD.

FDA Approval 246

FDA Approval In-Vivo Gene Therapy Clinical Trials 246

Drug Discovery World

SEPTEMBER 14, 2023

Also in July 2023, Alexion, AstraZeneca Rare Disease (Alexion) and Pfizer entered into an agreement for Alexion to purchase and license the assets of Pfizer’s early-stage rare disease gene therapy portfolio for a total consideration of up to $1bn, plus tiered royalties on sales. billion, a decrease of $15.0 Vaccines were up 9.1%

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Drugs Vaccination Vaccine Medicine 59

Pharmaceutical Technology

AUGUST 18, 2022

The US Food and Drug Administration (FDA) has granted approval for bluebird bio ’s Zynteglo (betibeglogene autotemcel, beti-cel) for the treatment of the underlying genetic cause of beta?thalassemia Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy Gene Genotype Genetics 246

Roots Analysis

JANUARY 14, 2024

Given the ongoing scientific advancements and the rise of FDA-approved biologics, the pharmaceutical industry seems to be approaching the era of biologics. It is worth mentioning that in vitro gene expression requires a suitable host for the production of a specific gene product.

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Contract Manufacturing Manufacturing Protein Bacteria 40

XTalks

DECEMBER 20, 2023

In 2023, Dr. Varshney notes that the US Food and Drug Administration (FDA) issued a request for industry comment on the integration of AI in drug and biologics development. This year, the very first CRISPR-based therapy received FDA approval for sickle cell disease.

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Life Science Gene Editing Development Clinical Trials 119

The Pharma Data

APRIL 27, 2021

Strong increase in Q1 2021 business EPS ( 1) at CER. Q1 2021 sales increase of 2.4% at CER driven by growth drivers Dupixent ® and Vaccines. Specialty Care sales grew 15.3%, due to strong Dupixent ® performance (+45.6% to €1,047 million) and oncology launches. General Medicines core assets grew 4.4%, while GBU sales were down 3.8%.

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Sales Vaccination Vaccine Medicine 52

Drug Discovery World

FEBRUARY 22, 2024

According to Medicine Discovery Catapult, these include: “Navigating the complexity of the CNS; understanding the disease pathology, and accessing predictable in vitro and in vivo cell models 2 ”. Dementia is the only major cause of death without a treatment to prevent, slow or stop disease progression. “In In 2019, dementia accounted for 1.8

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Drugs Gene Clinical Trials Clinical Trials 59