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RSS FeedPosted by Michael Wonder on 26 Feb 2020
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 ...
Posted by Michael Wonder on 25 Feb 2020
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. ...
Posted by Michael Wonder on 22 Feb 2020
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 ...
Posted by Michael Wonder on 21 Feb 2020
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. ...
Posted by Michael Wonder on 19 Feb 2020
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 ...
Posted by Michael Wonder on 13 Feb 2020
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 ...
Posted by Michael Wonder on 09 Feb 2020
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 ...
Posted by Michael Wonder on 06 Feb 2020
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 ...
Posted by Michael Wonder on 05 Feb 2020
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. ...
Posted by Michael Wonder on 03 Feb 2020
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. ...
Posted by Michael Wonder on 01 Feb 2020
Highlights from the CHMP 27-30 January 2020 meeting
31 January 2020 - Fifteen new medicines recommended for approval. ...
Posted by Michael Wonder on 31 Jan 2020
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 ...
Posted by Michael Wonder on 30 Jan 2020
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 ...
Posted by Michael Wonder on 29 Jan 2020
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 ...
Posted by Michael Wonder on 28 Jan 2020
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 ...