Translate Bio receives FDA fast track designation for MRT5005 for the treatment of cystic fibrosis

26 February 2020 - First inhaled mRNA therapeutic remains on track to report results from its ongoing multiple-ascending dose portion of ...

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Lysogene receives FDA fast track designation for LYS-SAF302 gene therapy in MPS IIIA

25 February 2020 - 17th patient dosed in the ongoing phase 2/3 AAVance study. ...

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Health ministers condemn Novartis lottery for Zolgensma, the world’s most expensive drug

12 February 2020 - Novartis has held the first draw to choose four babies who will receive its one-shot treatment ...

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BioMarin's biologics license application for valoctocogene roxaparvovec accepted for priority review by FDA with review action date of 21 August 2020

20 February 2020 - No advisory committee meeting currently planned to review the application. ...

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New institute launched to ensure the U.S. health care system is ready for gene therapies

20 February 2020 - Industry leaders, patient advocates, researchers unite to maximise the incredible potential of transformative gene therapies ...

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NDA accepted for filing by the FDA for antisense oligonucleotide viltolarsen

7 February 2020 - Nippon Shinyaku announced today that the U.S. FDA has accepted the filing of our new drug ...

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How this gene therapy drug earned its $2.1 million price tag

8 February 2020 - Swiss drugmaker Novartis recently launched a lottery-style program to allocate free doses of Zolgensma, its groundbreaking ...

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Call to reduce ‘scary’ gene therapy costs

28 November 2019 - Health care funders have been urged to overcome the “scary” cost of emerging gene therapies that ...

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Orsini Pharmaceutical Services now providing FDA approved Vyondys 53 (golodirsen) injection for Duchenne muscular dystrophy

5 February 2020 - Commercial distribution of Vyondys 53 in the U.S. is currently underway. ...

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Alnylam receives positive CHMP opinion for Givlaari (givosiran) for the treatment of acute hepatic porphyria in adults and adolescents

31 January 2020 - Positive opinion is based on data from the pivotal ENVISION Phase 3 study. ...

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Highlights from the CHMP 27-30 January 2020 meeting

31 January 2020 - Fifteen new medicines recommended for approval. ...

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When a lottery 'wins' sick babies life-saving drugs

30 January 2020 - Eva was diagnosed with spinal muscular atrophy, a motor neuron disease, just a few weeks after ...

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ProQR receives rare paediatric disease designation from FDA for QR-421a

30 January 2020 - Designation is for the treatment of patients with retinitis pigmentosa caused by mutations in exon 13 ...

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With new rules, FDA aims to spark competition, lower prices for expensive gene therapies

28 January 2020 - The FDA released a slew of gene therapy policies Tuesday aimed at encouraging drug makers to ...

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FDA continues strong support of innovation in development of gene therapy products

28 January 2020 - This is a pivotal time in the field of gene therapy as the FDA continues its ...

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