article thumbnail

Magazine: CRISPR gene therapies cut through in 2023

Pharmaceutical Technology

If things go as per plan, in a few months, the US Food and Drug Administration (FDA) will deliberate on the first-of-its-kind CRISPR-based gene therapy for sickle cell disease (SCD) and transfusion-dependent beta thalassemia. We also explore how AI is being used to design digital twins for clinical trials.

article thumbnail

Novartis says £1.8m Zolgensma is model for gene therapy pricing

pharmaphorum

Novartis has said its gene therapy pricing model, involving a steep one-off payment, could be a template for the hundreds of similar treatments that could follow its Zolgensma for Spinal Muscular Atrophy (SMA). . There are around 350 gene therapies in various studies.”. The post Novartis says £1.8m

Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

The Unprecedented Growth of Cell and Gene Therapy (2021)

Cloudbyz

Introduction Cell and gene therapy is an upcoming wave of therapeutic innovation in the healthcare and life sciences industry and is being pragmatically accepted worldwide. The gene therapy market reported its first market approvals back in 2017 and the evolution has been extensive ever since.

article thumbnail

FDA Approves Gene Therapy Adstiladrin for the Treatment of Bladder Cancer

XTalks

Ferring Pharmaceuticals announced that the US Food and Drug Administration (FDA) approved Adstiladrin (nadofaragene firadenovec-vncg) for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

article thumbnail

Life Science Trends for 2023 – Xtalks Life Science Podcast Ep. 91

XTalks

With a handful of significant gene therapy approvals in 2022, the cell and gene therapy space continues to rapidly expand, and hence we can expect more ground-breaking gene therapy approvals in the coming year, particularly for rare diseases. Read the full article here: 4 Life Sciences Trends for 2023.

article thumbnail

Kyowa Kirin completes acquisition of Orchard Therapeutics

Drug Discovery World

Japan-based pharma company Kyowa Kirin has successfully completed the acquisition of gene therapy developer Orchard Therapeutics. Libmeldy is known as OTL-200 in the US, where it is currently an investigational drug under Priority Review by the Food and Drug Administration (FDA).

article thumbnail

Injectable opportunities for CMOs despite internal capacity of big pharma sponsors

Pharmaceutical Technology

Even giant pharma companies with large internal production capabilities are leaning on outsourcing to meet market demand for huge numbers of injectables, including high-value biologics and Covid-19 vaccines, according to a GlobalData analysis. Why do large and mega-cap sponsors outsource?