Genethon
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Latest From Genethon
France: A European Leader In Rare Diseases
France has long been a European leader in rare diseases, from its scientific research and academic hospitals, through to patient advocacy and regulatory reforms that have encouraged R&D. Its biotech and pharmaceutical industries are now leading the way, discovering the next generation of therapies for patients that are often overlooked.
Genethon Works Toward Gene Therapy For Crigler-Najjar Syndrome
The French non-profit, whose IP is incorporated in Zolgensma, is taking its Crigler-Najjar gene therapy into a pivotal study on its own for now. It hopes to be ready for an EU filing in two years.
Moderna & Merck’s mRNA Cancer Vaccine Secures EMA PRIME Designation
A total of four investigational products targeting unmet medical needs have made it onto the European Medicines Agency’s priority medicines scheme so far this year.
Tech Transfer Roundup: Tiba And CEPI Strategize Together On ‘Disease X’
Immix collaborates with Israeli research institutions on BCMA-targeted CAR-T therapy. NeoImmuneTech/NIAID alliance could pave way for “animal rule” approval in acute radiation syndrome.
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