Blog
RSS FeedPosted by Michael Wonder on 24 May 2019
AveXis announces innovative Zolgensma gene therapy access programs for US payers and families
24 May 2019 - Annualised cost of Zolgensma is USD 425,000 per year for 5 years; 50% less than multiple established ...
Posted by Michael Wonder on 24 May 2019
FDA approves innovative gene therapy to treat paediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality
24 May 2019 - The U.S. FDA today approved Zolgensma (onasemnogene abeparvovec-xioi), the first gene therapy approved to treat children less ...
Posted by Michael Wonder on 23 May 2019
Novartis CEO plans gene therapy price 'far lower' than $4 million to $5 million range
23 May 2019 - Novartis AG’s top executive said on Wednesday it expects to price its gene therapy for spinal ...
Posted by Michael Wonder on 16 May 2019
Instalment payments could be used across gene therapy space, Spark CEO says
16 May 2019 - In a fireside chat at the recently concluded Veeva Summit in Philadelphia, Jeff Marrazzo said payment over ...
Posted by Michael Wonder on 15 May 2019
Homology Medicines announces FDA fast track designation for HMI-102 gene therapy development candidate for adults with PKU
14 May 2019 - Company provides details of pheNIX, the first gene therapy trial for PKU, and remains on track to ...
Posted 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 ...