Blog
RSS FeedPosted 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 ...
Posted by Michael Wonder on 21 Aug 2018
MeiraGTx announces AAV-CNGB3 granted fast track designation by U.S. FDA for treatment of achromatopsia
20 August 2018 - MeiraGTx today announced that the U.S. FDA has granted fast track designation for its AAV-CNGB3 gene therapy ...
Posted by Michael Wonder on 12 Aug 2018
Alnylam prices first gene silencing drug at $450,000 per patient, but offers money-back guarantee
10 August 2018 - This morning, after 16 years and $2.5 billion in investment, the Cambridge, Massachusetts-based company Alnylam finally ...
Posted by Michael Wonder on 11 Aug 2018
FDA approves first-of-its kind targeted RNA-based therapy to treat a rare disease
10 August 2018 - The U.S. FDA today approved Onpattro (patisiran) infusion for the treatment of peripheral nerve disease (polyneuropathy) caused ...
Posted by Michael Wonder on 08 Aug 2018
The FDA is poised to approve the first-ever drug that mutes disease-causing genes
7 August 2018 - After a decades-long wait, the FDA is on the brink of approving a landmark rare disease ...
Posted by Michael Wonder on 29 Jul 2018
Ultragenyx announces first patient dosed in Phase 1/2 study of DTX401, a gene therapy for the treatment of glycogen storage disease Type Ia; FDA grants fast track designation to gene therapy program
26 July 2018 - Ultragenyx today announced that the first patient has been dosed in the Phase 1/2 study of DTX401, ...
Posted by Michael Wonder on 11 Jul 2018
Statement from FDA Commissioner on agency’s efforts to advance development of gene therapies
11 July 2018 - Once just a theory, gene therapies are now a therapeutic reality for some patients. ...
Posted by Michael Wonder on 23 Jun 2018
Voyager Therapeutics announces FDA regenerative medicine advanced therapy designation granted for VY-AADC for the treatment of Parkinson’s Disease
21 June 2018 - Voyager Therapeutics today announced that the U.S. FDA granted regenerative medicine advanced therapy designation for Voyager’s VY-AADC ...
Posted by Michael Wonder on 17 Jun 2018
Nightstar Therapeutics receives regenerative medicine advanced therapy designation for NSR-REP1 in choroideremia
14 June 2018 - First gene therapy RMAT designation for an inherited retinal disease. ...