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After nearly 2.
A twist and turn on the road to listing
A twist and turn on the road to listingRoctavian is an AA5 gene therapy that delivers a functional copy of the gene encoding coagulation factor VIII to patients through AAV5, thereby helping patients restore their own coagulation factor VIII production capacity, aiming to achieve the effect
BioMarin filed a Marketing Authorization Application (MAA) with the EMA as early as November 21, 2019, and a month later (December 23) with the FDA
However, compared with the FDA, the EMA's regulatory approval of new drugs is more relaxed
Disrupt the $10 billion market
Disrupt the $10 billion marketHemophilia is an ancient disease, the progress of medical methods has made great progress in the treatment of hemophilia, and today's hemophilia patients have almost achieved a survival time
Hemophilia is mainly divided into A and B types, of which most patients with hemophilia A have an incidence of about 4 times
As gene therapy technology continues to make breakthroughs in the clinical treatment of hemophilia, hemophilia has gradually become one of the hottest tracks of gene therapy
summary
summaryIn recent years, the development of gene therapy has always been accompanied
References:
References:1.
1.
first-gene-therapy-for-adults-with-severe-hemophilia-a-biomarins-roctavian-valoctocogene-roxaparvovec-approved-by-european-commission-ec-301611980.
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