Blog
RSS FeedPosted by Michael Wonder on 22 May 2020
Passage Bio receives rare paediatric disease designation for PBGM01 for patients with GM1 gangliosidosis
21 May 2020 - PBGM01 to enter the clinic in fourth quarter of 2020. ...
Posted 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 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 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 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 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 ...
Posted 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 ...