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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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Tenaya’s cardiomyopathy gene therapy gets fast track boost

Pharmaceutical Technology

The US Food and Drug Administration (FDA) has granted Tenaya Therapeutics’ gene therapy TN-201 a fast track designation for the treatment of myosin binding protein C3 (MYBPC3)-associated hypertrophic cardiomyopathy (HCM). If successful, TN-201 would be a first-in-class disease-modifying gene therapy for MYBPC3-associated HCM.

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Regeneron, UPenn to use gene therapy tools to deliver a COVID-19 antibody drug

Bio Pharma Dive

Working with University of Pennsylvania researcher Jim Wilson, Regeneron aims to develop a nasal spray version of the coronavirus treatment recently cleared by the Food and Drug Administration.

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Roctavian okay sets up another gene therapy test for Europe

pharmaphorum

BioMarin Pharma has its landmark first approval for Roctavian – the first for a haemophilia gene therapy – and will now have to see if that can convert that into a viable business in Europe. For now, the list price hasn’t been set and will depend on discussions with reimbursement authorities, according to the biotech.

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BioMarin gene therapy for haemophilia on course for EU approval

pharmaphorum

The EMA’s human medicines advisory committee has recommended that BioMarin Pharmaceutical’s haemophilia A gene therapy Roctavian be approved in the EU, removing one of the last barriers to launch. The post BioMarin gene therapy for haemophilia on course for EU approval appeared first on.