Blog
RSS FeedPosted by Michael Wonder on 03 May 2022
First million dollar medicine listed on the PBS
4 May 2022 - Zolgensma has the great honour of being the first medicine listed on the PBS with a ...
Posted by Michael Wonder on 02 May 2022
TLV submits proposals for increased access to new gene therapies
2 May 2022 - How should society handle new gene therapies for serious diseases, where the effect of the drugs is ...
Posted by Michael Wonder on 13 Apr 2022
ICER releases draft evidence report on gene therapy for beta thalassaemia
13 April 2022 - Public comment period now open until 10 May 2022; requests to make oral comment during public ...
Posted by Michael Wonder on 11 Apr 2022
Orchard Therapeutics announces reimbursement agreement making Libmeldy available for all eligible MLD patients in Italy
11 April 2022 - Orchard Therapeutics today announced it has reached another historic reimbursement agreement with the Italian Medicines Agency, also ...
Posted by Michael Wonder on 04 Apr 2022
JW Therapeutics announces receipt of breakthrough therapy designation for Carteyva in mantle cell lymphoma in China
3 April 2022 - JW Therapeutics announced that the Center for Drug Evaluation of the National Medical Products Administration of China ...
Posted by Michael Wonder on 04 Apr 2022
Bluebird Bio has serious cash flow concerns; inability to strike reimbursement deals in Europe partly to blame
3 April 2022 - Bluebird bio is a highly innovative biotechnology company which develops gene therapies for several rare genetic ...
Posted by Michael Wonder on 31 Mar 2022
ASC Therapeutics receives key regulatory designations in U.S. and Europe to advance its second generation gene therapy for haemophilia A
29 March 2022 - ASC Therapeutics has received from the U.S. FDA the fast track designation for ASC618, a second-generation gene ...
Posted by Michael Wonder on 29 Mar 2022
European Medicines Agency commences review of novel gene therapy candidate etranacogene dezaparvovec for people with haemophilia B
29 March 2022 - Marketing authorisation application for etranacogene dezaparvovec will be reviewed under accelerated assessment and has the potential to ...
Posted by Michael Wonder on 28 Mar 2022
Atidarsagene autotemcel for treating metachromatic leukodystrophy
28 March 2022 - NICE has published evidence-based recommendations on atidarsagene autotemcel (Libmeldy) for treating metachromatic leukodystrophy in children. ...
Posted by Michael Wonder on 25 Mar 2022
New gene therapy to treat adult patients with multiple myeloma
25 March 2022 - EMA has recommended a conditional marketing authorisation in the European Union for Carvykti (ciltacabtagene autoleucel) for the ...
Posted by Michael Wonder on 21 Mar 2022
Genomics Australia to guide the future of genomic health and medicine over the coming decade
21 March 2022 - Through our 10 year Medical Research Future Fund (MRFF) plan, the Morrison Government is committed to ...
Posted by Michael Wonder on 16 Mar 2022
FDA drafts guidance on genome editing, CAR T cell therapies
16 March 2022 - The U.S. FDA has issued two draft guidances addressing the development of human gene therapy products that ...
Posted by Michael Wonder on 01 Mar 2022
Kiwis call on Government to increase PHARMAC funding to help fund Spinraza treatment
1 March 2022 - A group of Kiwis are calling on the Government to increase PHARMAC funding to provide better ...
Posted by Michael Wonder on 17 Feb 2022
FDA grants fast track designation to SBT101, the first investigational AAV based gene therapy for patients with adrenomyeloneuropathy
16 February 2022 - Initiation of Phase 1/2 clinical trial expected in the second half of 2022. ...
Posted by Michael Wonder on 04 Feb 2022
First gene therapy for rare genetic neurodegenerative disorder in children, recommended in NICE draft guidance
4 February 2022 - Children with a rare, fatal, genetic disorder will be able to benefit from a new one-off treatment ...