gene therapy

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BioMarin seeks haemophilia gene therapy approval after promi...

BioMarin is set to file for approval for its haemophilia gene therapy valoctocogene roxaparvovec, after it achieved pre-specified clinical criteria for regulatory review.

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SMi's 9th Annual Orphan Drugs & Rare Diseases Conference

Orphan Drugs & Rare Diseases

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Former Alexion CEO Hallal makes comeback at cell therapy hol...

Massachusetts holding company ElevateBio has launched with $150m in initial funding from a range of investors on a mission to build a portfolio of cell and gene therapy companies.<

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Second death in gene therapy trial prompts investigation

A second death in a study of Novartis’ spinal muscular atrophy gene therapy Zolgensma (onasemnogene abeparvovec-xioi; AVXS-101) – a potential blockbuster for this rare disease – has prompte

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Catalent acquires gene therapy specialist Paragon for $1.2bn

Drug delivery technology provider Catalent is to acquire gene therapy company Paragon for $1.2 billion.

News

Roche insists Spark deal is still on, despite lacklustre tak...

Shareholders in Spark Therapeutics haven’t been queueing up to take advantage of Roche’s $114.50-per-share offer, forcing an extension to the tender process.

BioMarin seeks haemophilia gene therapy approval after promi...

SMi's 9th Annual Orphan Drugs & Rare Diseases Conference

Former Alexion CEO Hallal makes comeback at cell therapy hol...

Second death in gene therapy trial prompts investigation

Catalent acquires gene therapy specialist Paragon for $1.2bn

Roche insists Spark deal is still on, despite lacklustre tak...

Ambrosia's sweet $100m round, and other biofinancings

Using AI-driven synthetic personas to take your insights fur...

gene therapy

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