SRP-9001
Lilly and MeiraGTx partner in $475m ophthalmology deal
The companies will develop and commercialise therapies for genetic conditions
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
What do people look for in gene therapy materials? Results from our survey of patients and relatives
By Sam Fraser
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
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
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
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
Roche/Sarepta announce two-year results for Duchenne muscular dystrophy gene therapy
The muscle-wasting disorder is estimated to affect one in every 5,000 male births worldwide
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
MHRA approves Italfarmaco’s Duvyzat to treat Duchenne muscular dystrophy
The severe neuromuscular disease affects approximately 2,500 people in the UK
NICE recommends Santhera’s Duchenne muscular dystrophy drug Agamree
Approximately 1,700 patients with the muscle-wasting disorder in England are expected to benefit from the decision