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RSS FeedPosted 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. ...
Posted by Michael Wonder on 19 Nov 2018
Gottlieb pushes for funding to speed gene therapy reviews
16 November 2018 - The FDA is working to increase its investment in reviewing gene therapy products, Commissioner Scott Gottlieb ...
Posted by Michael Wonder on 05 Nov 2018
Novartis says SMA gene therapy is cost-effective at $4-5 million per patient
5 November 2018 - Novartis, which is shifting into rare diseases, said on Monday it believes its new gene therapy ...
Posted by Michael Wonder on 08 Oct 2018
Akcea announces its access and distribution strategy for Tegsedi (inotersen)
5 October 2018 - Express Scripts’ Accredo selected as specialty pharmacy for its expertise in providing timely access and support services ...
Posted by Michael Wonder on 05 Oct 2018
Akcea and Ionis receive FDA approval of Tegsedi (inotersen) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults
5 October 2018 - Tegsedi provides powerful knockdown of TTR protein, delivering significant and sustained benefits in neuropathy and quality of ...
Posted by Michael Wonder on 21 Sep 2018
Adverum Biotechnologies receives fast track designation for ADVM-022 gene therapy for the treatment of wAMD
19 September 2018 - ADVM-022 is a unique single-administration gene therapy delivered intra-vitreally for the treatment of wAMD. ...
Posted by Michael Wonder on 11 Sep 2018
Amicus Therapeutics announces regulatory and clinical updates for AT-GAA in Pompe disease
10 September 2018 - Amicus Therapeutics announced today regulatory and clinical advancements in its development program AT-GAA for Pompe disease. ...
Posted by Michael Wonder on 07 Sep 2018
Fibrocell announces FDA fast track designation of FCX-013 for treatment of moderate to severe localised scleroderma
5 September 2018 - Fibrocell Science today announced that the U.S. FDA has granted fast track designation to FCX-013, the Company’s ...