Gene Therapy
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News
ACCESS TO INNOVATION | UK Greenlights Gene-Editing Therapy Casgevy for β-Thalassaemia Treatment
This development opens new treatment avenues for an estimated 460 patients aged 12 and above in England who rely on regular transfusions due to β-thalassaemia, leveraging innovative CRISPR gene-editing technology.…
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News
BREAKING | Vertex and CRISPR’s Gene-editing Therapy Casgevy Receives Early FDA Approval for β-Thalassaemia Treatment
Just one month prior, Casgevy made history as the first CRISPR gene editing therapy to enter the US market when it was approved for sickle cell disease. Now, the FDA…
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News
SICKLE CELL DISEASE | FDA Approves Two Landmark Gene Therapies for the Treatment of the Disorder
The two therapies are Casgevy (exa-cel) by Vertex Pharmaceuticals and CRISPR Therapeutics – the world’s first drug to utilize the revolutionary CRISPR gene-editing system which earned its creators the 2020…
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News
GENE THERAPY | FDA Decision on Lovo-cel for SCD Now Expected by Year’s End
The regulatory agency has agreed to review an application from lovo-cel’s developer, Bluebird Bio, requesting the gene therapy’s approval for SCD patients ages 12 and older. Moreover, the FDA has given the application priority…
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News
NEW TREATMENTS | Gene Editing, mRNA Hold Promise as Fanconi Anemia Therapies
That is the major finding of a newly published review that examined how a better understanding in the scientific and molecular underpinnings of the disease has translated into potential new…
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News
TOP STORY | EMA Validates Vertex’s/CRISPR Submission Of Exa-Cel For β-Τhal And SCD
The Marketing Authorization Application (MAA) for exagamglogene autotemcel (exa-cel), marks the first regulatory submission in the EU for a CRISPR-based medicine. Through the validation, exa-cel is indicated for the…
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News
NEW | Bluebirdbio Releases Resources For Patients On Gene Therapy Zynteglo®
To help people who are living with or caring for someone who has β-thalassaemia major get substantial information about Zynteglo®, bluebird bio, the company behind this ground-breaking therapy, has recently…
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Clinical News
BREAKING NEWS | Bluebird’s Gene Therapy For β-Thalassaemia Wins US Approval
The green light is “monumental” for both patients and the company itself, Tom Klima, Bluebird’s Chief Commercial Officer, said in an interview. Bluebird has endured its share of “bumps and…
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News
Juggling Between The Cost And Value of New Therapies: Does Science Still Serve Patient Needs?
In the aftermath of the withdrawal of Zynteglo, the first gene therapy for patients with β-thalassaemia, from Europe, TIF´s Executive Director, Dr Androulla Eleftheriou, reflects on the viability of the…
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News
Bluebird Bio Resumes Marketing Of Gene Therapy Zynteglo For β-Thalassaemia In Europe
The European Medicines Agency (EMA)’s safety committee (PRAC) has concluded that there is no evidence Zynteglo causes a blood cancer known as acute myeloid leukaemia (AML). Zynteglo, a gene therapy…
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