Novel haemophilia B gene therapy approved
Pfizer has attained its first approval for a gene therapy from the US Food and Drug Administration (FDA), which is indicated to treat the rare blood disorder haemophilia B.
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Pfizer has attained its first approval for a gene therapy from the US Food and Drug Administration (FDA), which is indicated to treat the rare blood disorder haemophilia B.
In late October, we learned that an exciting and ambitious new entity – the Bespoke Gene Therapy Consortium (BGTC) – had come into being in the US, designed to encourage the delivery of more gene therapies for rare diseases. The consortium is an interesting construct, headed and funded by the…
The consortium also consists of 10 pharmaceutical companies and five organisations to accelerate gene therapies to treat rare diseases.
The CMA has cleared the anticipated purchase of gene therapy company Spark Therapeutics by pharmaceutical company Roche Holdings.