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Astellas to support development of Taysha’s gene therapy programmes

Pharmaceutical Technology

Ast ellas Pharma has announced plans to make a strategic investment to back the development of Taysha Gene Therapies’ adeno-associated virus (AAV) development programmes for Rett syndrome and giant axonal neuropathy (GAN). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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EC grants approval for BioMarin’s gene therapy to treat Hemophilia A

Pharmaceutical Technology

The European Commission (EC) has granted conditional marketing authorisation (CMA) for BioMarin Pharmaceutical ’s gene therapy, Roctavian (valoctocogene roxaparvovec), to treat adults with severe haemophilia A (congenital Factor VIII deficiency). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Can gene therapies for haemophilia defend their high price tags?

Pharmaceutical Technology

On November 2, the Institute for Clinical and Economic Review (ICER) released its updated evidence aimed at measuring the clinical effectiveness and cost of the two haemophilia gene therapies. Known by the brand name Roctavian, BioMarin’s haemophilia A therapy valoctocogene roxaparvovec could be fairly priced in the range of $1.95–1.96

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GenScript ProBio and CCS partner for cell and gene therapies development

Pharmaceutical Technology

GenScript ProBio has collaborated with the New York Blood Center Enterprises’ business unit, called Comprehensive Cell Solutions (CCS), to accelerate the development and manufacture of cell and gene therapies. The core objective of the collaboration is to make the therapies affordable and accessible to patients.

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Hansa Biopharma and Genethon collaborate to test imlifidase as gene therapy pre-treatment

BioPharma Reporter

Hansa Biopharma and Genethon have entered a research and development collaboration to test imlifidase as a gene therapy treatment in patients with pre-existing neutralizing antibodies.

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Roctavian Becomes First Gene Therapy for Severe Hemophilia A to Get FDA Nod

XTalks

It’s been a big week for cell and gene therapy approvals in the US, including a much-awaited approval for one to treat hemophilia A, the most common form of hemophilia. To be eligible for the therapy, patients must test negative for AAV5-targeted antibodies. Roctavian, a $2.9 SVB has forecasted around $2.2

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Can vertical AI advance cell and gene therapies? 

Drug Discovery World

Andrew Busey , Co-Founder, Form Bio discusses why vertical AI is set to reshape the cell and gene therapy sector. The cell and gene therapy industry is no exception to this digital transformation. Here’s what this means for pharmaceutical leaders and patients.