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Bluebird, playing catch-up, gets decision date for sickle cell gene therapy

Bio Pharma Dive

The regulator will issue a decision on lovo-cel by Dec. 20, roughly two weeks after a verdict is expected on a rival gene editing treatment from Vertex and CRISPR Therapeutics.

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Logistics in Focus: Orchestrating the Challenges of Cell Therapy

Worldwide Clinical Trials

By Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease Cell and gene therapies (CGTs) include cutting-edge approaches that offer the hope of a healthier, happier, and better tomorrow for a wide range of patient populations. Below, we discuss some of these challenges in cell therapy trials.

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England's NICE clears Vertex, CRISPR's Casgevy in beta thalassemia after passing on sickle cell use

Fierce Pharma

On the rocky road to ensuring access to gene therapy Casgevy in the U.K.,

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Regulatory Trends in Cell and Gene Therapies

Advarra

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

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ASH: CRISPR, Vertex’ CTX001 hits the mark in red cell disorders

pharmaphorum

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has achieved “remarkable” improvements in patients with beta thalassaemia and sickle cell disease in an early-stage trial reported at the American Society of Haematology (ASH) annual meeting.

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The Challenges and Promise of CAR T-Cell Therapy in Oncology

XTalks

Vittoria Biotherapeutics is a clinical-stage cell therapy company that incorporates the latest advancements in cell therapy and gene editing with a unique approach that potentially overcomes some of the issues with this therapeutic modality. Its therapeutic pipeline targets unmet needs in oncology and autoimmune disease.

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New Rare Disease Drugs and Research Advancements

XTalks

Technologies like CRISPR-Cas9 have revolutionized the potential for gene editing, offering hope for curative treatments for conditions like Duchenne muscular dystrophy (DMD) and certain types of inherited blindness. Both therapies were awarded their approvals on the same day in December 2023.