Blog
RSS FeedPosted by Michael Wonder on 13 May 2019
Novartis pitches discounts on pricey gene therapy for deadly muscle disorder
11 May 2019 - Novartis is offering price discounts in negotiations with U.S. health insurers on its gene therapy for spinal ...
Posted by Michael Wonder on 09 May 2019
Novartis confident of Zolgensma supply, calls $2 million price 'speculation'
8 May 2019 - Novartis is confident it has adequate production capacity for its Zolgensma gene therapy should regulators this ...
Posted by Michael Wonder on 08 May 2019
A $2 million drug is about to hit the market
8 May 2019 -Insurers, drug makers grapple with new payment models for gene therapies that can cure diseases in one ...
Posted by Michael Wonder on 02 May 2019
FDA's efforts to advance the development of gene therapy
1 May 2019 - Gene therapy has been on the horizon for several decades and has now become a reality in ...
Posted by Michael Wonder on 09 Apr 2019
uniQure receives FDA fast track designation for AMT-130 gene therapy for the treatment of Huntington’s disease
8 April 2019 - On track to treat first patient in Phase I/II study of AMT-130 in 2H19. ...
Posted by Michael Wonder on 07 Apr 2019
Abeona Therapeutics receives FDA fast track designation for ABO-101 for treatment of Sanfilippo syndrome type B
4 April 2019 - Ongoing Phase 1/2 study of ABO-101 enrolling eligible patients with MPS IIIB. ...
Posted by Michael Wonder on 25 Feb 2019
Roche has a $5 billion chance to lead on drug prices
25 February 2019 - A deal for gene-therapy developer Spark could help reset what’s now an exorbitant corner of the market. ...
Posted by Michael Wonder on 15 Feb 2019
Sarepta announces FDA acceptance of golodirsen (SRP-4053) new drug application for patients with Duchenne muscular dystrophy amenable to skipping exon 53
14 February 2019 - FDA grants priority review status. ...
Posted by Michael Wonder on 11 Feb 2019
RegenxBio receives rare paediatric disease designation for RGX-181 gene therapy for the treatment of CLN2 form of Batten disease
31 January 2019 - Novel, one-time investigational treatment for CLN2 disease designed to halt progression of this rare, paediatric, neurodegenerative disease. ...
Posted by Michael Wonder on 15 Jan 2019
Statement from FDA Commissioner and Director of the Center for Biologics Evaluation and Research on new policies to advance development of safe and effective cell and gene therapies
15 January 2019 - The FDA is witnessing a surge of cell and gene therapy products entering early development, evidenced ...
Posted by Michael Wonder on 14 Jan 2019
A revolutionary drug that could treat a rare and devastating disease is prohibitively expensive. But one state has a plan to pay for its potential $5 million price tag.
13 January 2019 - A one-time treatment for a devastating rare disease could be paid for with an instalment plan as ...
Posted by Michael Wonder on 02 Jan 2019
ProQR receives fast track designation from FDA for QR-421a for Usher Syndrome Type 2
2 January 2019 - ProQR Therapeutics today announced that it received fast track designation from the FDA for QR-421a. ...
Posted by Michael Wonder on 23 Dec 2018
Gritstone Oncology announces FDA fast track designation for GRANITE-001 for the treatment of colorectal cancer
20 December 2018 - Gritstone Oncology today announced that the U.S. FDA has granted fast track designation to GRANITE-001 for the ...
Posted by Michael Wonder on 03 Dec 2018
Novartis announces FDA filing acceptance and priority review of AVXS-101, a one-time treatment designed to address the genetic root cause of SMA Type 1
3 December 2018 - The AVXS-101, now known as Zolgensma (onasemnogene abeparvovec-xxxx), filing is supported by data from the START trial ...
Posted by Michael Wonder on 28 Nov 2018
Rocket Pharmaceuticals receives FDA regenerative medicine advanced therapy and fast track designations for RP-L102 gene therapy for Fanconi anaemia
27 November 2018 - Global trial of RP-L102 utilising no conditioning and “Process B” on track for early 2019. ...