Blog
RSS FeedPosted by Michael Wonder on 17 Jul 2019
LogicBio Therapeutics receives rare paediatric disease designation for LB-001 for the treatment of methylmalonic acidaemia
16 July 2019 - LogicBio Therapeutics today announced the U.S. FDA has granted rare paediatric disease designation to LB-001, a ...
Posted by Michael Wonder on 16 Jul 2019
Dicerna receives breakthrough therapy designation for DCR-PHXC for treatment of primary hyperoxaluria type 1
15 July 2019 - FDA recognises primary hyperoxaluria Types 2 and 3 as meeting criteria for a serious or life-threatening disease ...
Posted by Michael Wonder on 09 Jul 2019
'Amazing' gene-silencing drugs reach NHS
9 July 2019 - A new form of medicine called "gene-silencing" has been approved for use by the NHS in ...
Posted by Michael Wonder on 09 Jul 2019
NICE Onpattro approval puts Alnylam back in hATTR running
9 July 2019 - NICE has recommended the use of Alynylam’s Onpattro (patisiran), after issuing a preliminary ‘no’, back in ...
Posted by Michael Wonder on 08 Jul 2019
Report calls for European funding reform for gene therapies
8 July 2019 - New payment models, dedicated investment, and better health technology assessment are among a raft of changes ...
Posted by Michael Wonder on 07 Jul 2019
Life-changing drug Spinraza still not covered for many Canadians with spinal muscular atrophy
4 July 2019 - For several hundred Canadians with spinal muscular atrophy, Spinraza is a drug that means the difference ...
Posted by Michael Wonder on 04 Jul 2019
Spinraza access agreement extended
3 July 2019 - More children with the rare genetic disorder spinal muscular atrophy can now be treated with Spinraza after ...
Posted by Michael Wonder on 03 Jul 2019
Some insurers are pushing back on the high price for a Novartis gene therapy
3 July 2019 - Barely a month after Novartis won U.S. regulatory approval for its gene therapy — the most ...
Posted by Michael Wonder on 03 Jul 2019
These two brothers are living healthier lives, thanks to gene therapy
3 July 2019 - It has taken decades of work and billions of dollars, but Australia is now entering an ...
Posted by Michael Wonder on 02 Jul 2019
Innovation prizes to support cell and gene therapy
2 July 2019 - Personalised cell and gene therapy represent a fundamental therapeutic revolution. ...
Posted by Michael Wonder on 02 Jul 2019
Alnylam submits marketing authorisation application to the EMA for givosiran for the treatment of acute hepatic porphyria
1 July 2019 - Patients receiving givosiran in pivotal Phase 3 ENVISION study had a 74% mean reduction in annualised rate ...
Posted by Michael Wonder on 25 Jun 2019
Krystal Biotech receives regenerative medicine advanced therapy designation from FDA for KB103
24 June 2019 - RMAT designation granted to KB103 based on positive interim data from GEM-1 and GEM-2 study. ...
Posted by Michael Wonder on 19 Jun 2019
Abeona Therapeutics receives FDA fast track designation for ABO-202 AAV9 gene therapy in CLN1 disease
18 June 2019 - Abeona Therapeutics today announced that the U.S. Food and FDA has granted fast track designation to its ...
Posted by Michael Wonder on 18 Jun 2019
Zolgensma: a remarkable new treatment, an ICER analysis, and a poorly justified price
18 June 2019 - Defects in both sets of alleles in the coding region for the survival motor neuron 1 ...
Posted by Michael Wonder on 15 Jun 2019
New cost-effectiveness methods to determine value-based prices for potential cures: what are the options?
12 June 2019 - Evaluating different approaches to assessing the clinical effectiveness and value of potential cures will be essential to ...