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BioMarin

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Lilly and MeiraGTx partner in $475m ophthalmology deal

The companies will develop and commercialise therapies for genetic conditions

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FDA unveils new voucher programme to accelerate drug review process

The programme is designed to reduce the review time for companies supporting US national interests

The (clinical) trials and tribulations of gene therapy – why meaningful patient engagement strategies matter

By Sarah Mohamad and Lauren Coleman-Bennett

Amiculum

What do people look for in gene therapy materials? Results from our survey of patients and relatives

By Sam Fraser

Amiculum

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Sobi receives NICE recommendation for Altuvoct in severe haemophilia A

Approximately 7,700 people in England are living with the genetic bleeding disorder

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FDA approves Bavarian Nordic’s chikungunya vaccine for individuals aged from 12 years

The mosquito-borne viral disease has been reported across many popular travel destinations

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Sobi receives MHRA approval for Altuvoct in severe or moderate haemophilia A

The factor VIII replacement therapy has been authorised to treat and prevent bleeding in children and adults

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Roche’s Evrysdi tablet formulation granted FDA approval for spinal muscular atrophy

The progressive neuromuscular disease affects approximately one in every 10,000 babies globally

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CSL Behring announces four-year results for haemophilia B gene therapy Hemgenix

The genetic bleeding disorder affects an estimated one per every 19,283 male births in the US

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Novo Nordisk’s haemophilia A therapy Mim8 shows promise in late-stage study

The inherited bleeding disorder accounts for up to 85% of the 1,125,000 global haemophilia cases

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NICE approves Vertex’s Casgevy one-time gene therapy for sickle cell disease

About 15,000 people in England are living with the inherited blood disorder

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FDA accepts updated protocol for Annovis’ phase 3 Alzheimer’s disease study

The company hopes the revised protocol will accelerate the development timeline of buntanetap

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