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RSS FeedPosted by Michael Wonder on 21 Jan 2020
Genprex receives U.S. FDA fast track designation for gene therapy that targets lung cancer
21 January 2020 - Genprex today announced that the U.S FDA has granted fast track Designation for Genprex’s Oncoprex immunogene ...
Posted by Michael Wonder on 17 Jan 2020
BioMarin explores pricing experimental gene therapy at $2 million to $3 million
16 January 2020 - Valrox would be first gene therapy approved in U.S. for inherited type of haemophilia. ...
Posted by Michael Wonder on 14 Jan 2020
Roche aims to 'underwhelm' on SMA drug price to challenge rivals
14 January 2020 - Swiss drug maker Roche plans to price its oral spinal muscular atrophy drug risdiplam aggressively to challenge ...
Posted by Michael Wonder on 14 Jan 2020
PTC Therapeutics provides corporate update
13 January 2020 - PTC-AADC MAA submitted; BLA now expected to be submitted in 2Q 2020. ...
Posted by Michael Wonder on 12 Jan 2020
At 16, she’s a pioneer in the fight to cure sickle cell disease
11 January 2020 - Helen Obando is the youngest person ever to get a gene therapy that scientists hope will cure ...
Posted by Michael Wonder on 09 Jan 2020
Aruvant receives FDA rare paediatric disease designation for ARU-1801 for the treatment of sickle cell disease
8 January 2020 - ARU-1801 is being developed as a potential one-time treatment for patients suffering from sickle cell disease and ...
Posted by Michael Wonder on 24 Dec 2019
BioMarin submits biologics license application to U.S. FDA for valoctocogene roxaparvovec to treat haemophilia A
23 December 2019 - First marketing application submission in U.S. for gene therapy directed at any type of haemophilia. ...
Posted by Michael Wonder on 14 Dec 2019
FDA grants accelerated approval to first targeted treatment for rare Duchenne muscular dystrophy mutation
12 December 2019 - The U.S. FDA today granted accelerated approval to Vyondys 53 (golodirsen) injection to treat Duchenne muscular dystrophy ...
Posted by Michael Wonder on 13 Dec 2019
Sarepta Therapeutics announces FDA approval of Vyondys (golodirsen) injection for the treatment of Duchenne muscular dystrophy in patients amenable to skipping exon 53
12 December 2019 - Vyondys 53 is Sarepta’s second RNA exon-skipping treatment for DMD approved in the U.S. ...
Posted by Michael Wonder on 25 Nov 2019
FDA grants priority review to Roche’s risdiplam for spinal muscular atrophy
25 November 2019 - Filing submission includes 12-month data from pivotal FIREFISH and SUNFISH trials in a broad population of people ...
Posted by Michael Wonder on 21 Nov 2019
Drug for ultra rare disease comes with ultra high price tag
21 November 2019 - Givlaari’s net price will be $442,000 after discounts included. ...
Posted by Michael Wonder on 20 Nov 2019
FDA approves first treatment for inherited rare disease
20 November 2019 - Today, the U.S. FDA granted approval to Givlaari (givosiran) for the treatment of adult patients with acute ...
Posted by Michael Wonder on 06 Nov 2019
Novartis says delayed telling FDA of Zolgensma concern due to 'mistake'
2 November 2019 - Novartis scientists learnt months ago about safety concerns surrounding its gene therapy Zolgensma but delayed telling the ...
Posted by Michael Wonder on 06 Nov 2019
One shot drug to end Sicilian curse comes at $1.8 million cost
5 November 2019 - A new targeted gene therapy shows the promise and pitfalls of medical advances. ...
Posted by Michael Wonder on 22 Oct 2019
Moderna receives FDA fast track designation for propionic acidemia program (mRNA-3927)
22 October 2019 - mRNA-3927 is Moderna’s second rare disease program to receive fast track designation. ...