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RSS FeedPosted by Michael Wonder on 23 Aug 2023
Comanche Biopharma receives US FDA fast track designation for CBP-4888 for the treatment of sFlt-1 mediated pre-term preeclampsia
23 August 2023 - Comanche Biopharma today announced that they have received fast track designation from the US FDA for ...
Posted by Michael Wonder on 07 Aug 2023
HuidaGene Therapeutics receives FDA rare paediatric disease designation for HG004 to treat inherited blindness
7 August 2023 - HG004 is a one time, direct to RPE treatment of inherited retinal disease caused by mutations in ...
Posted by Michael Wonder on 25 Jul 2023
Peter Mac increases manufacturing centre for gene therapy bringing new hope to cancer patients
25 July 2023 - Australia’s first biomedical manufacturing facility has opened in Melbourne and will boost lifesaving therapy for cancer patients ...
Posted by Michael Wonder on 21 Jul 2023
Excision BioTherapeutics receives FDA fast track designation for EBT-101, a first in class CRISPR based gene therapy candidate to functionally cure HIV-1
20 July 2023 - EBT-101 is a potentially curative, one time CRISPR based treatment which makes two cuts in integrated retroviral ...
Posted by Michael Wonder on 11 Jul 2023
Synlogic granted fast track designation from FDA for labafenogene marselecobac (SYNB1934) for treatment of phenylketonuria
11 July 2023 - Synlogic today announced that the US FDA has granted fast track designation to labafenogene marselecobac (previously known ...
Posted by Michael Wonder on 07 Jul 2023
FDA grants rare paediatric disease designation to NS-089/NCNP-02 for the treatment of Duchenne muscular dystrophy
7 July 2023 - NS Pharma announced today the US FDA has granted rare paediatric disease designation to NS-089/NCNP-02 (brogidirsen) an ...
Posted by Michael Wonder on 03 Jul 2023
BioMarin’s next Roctavian challenge is how to successfully commercialise its gene therapy: warranties may be an option
1 July 2023 - After quite the regulatory process, with Roctavian (valoctocogene roxaparvovec) BioMarin has a potential blockbuster. On 29 ...
Posted by Michael Wonder on 30 Jun 2023
FDA approves ARUP Laboratories' AAV5 DetectCDx, a first-ever companion diagnostic immunoassay for a gene therapy
29 June 2023 - FDA issues simultaneous approval of Roctavian (valoctocogene roxaparvovec-rvox), BioMarin's gene therapy for severe haemophilia A. ...
Posted by Michael Wonder on 29 Jun 2023
US FDA approves BioMarin's Roctavian (valoctocogene roxaparvovec-rvox), the first and only gene therapy for adults with severe haemophilia A
29 June 2023 - Roctavian's approval was based on durability, efficacy and safety results from the largest and longest Phase 3 ...
Posted by Michael Wonder on 27 Jun 2023
FDA accepts Pfizer’s application for haemophilia B gene therapy fidanacogene elaparvovec
27 June 2023 - Submissions based on positive Phase 3 data from BENEGENE-2 trial. ...
Posted by Michael Wonder on 22 Jun 2023
FDA approves first gene therapy for treatment of certain patients with Duchenne muscular dystrophy
22 June 2023 - Today, the US FDA approved Elevidys, the first gene therapy for the treatment of pediatric patients 4 ...
Posted by Michael Wonder on 21 Jun 2023
bluebird bio announces FDA priority review of the biologics license application for lovotibeglogene autotemcel (lovo-cel) for patients with sickle cell disease 12 years and older with a history of vaso-occlusive events
21 June 2023 - PDUFA date set for 20 December 2023. ...
Posted by Michael Wonder on 20 Jun 2023
CSL Behring announces the first patient has received FDA approved Hemgenix (etranacogene dezaparvovec-drlb) for haemophilia B
20 June 2023 - Hemgenix, the first and only FDA approved gene therapy for haemophilia B, has been proven to ...
Posted by Michael Wonder on 15 Jun 2023
FDA should reject accelerated approval of gene therapy SRP-9001
15 June 2023 - The US FDA should reject the accelerated approval of the gene therapy SRP-9001 (Sarepta Therapeutics) for ...
Posted by Michael Wonder on 09 Jun 2023
FDA accepts biologics license applications for exagamglogene autotemcel (exa-cel) for severe sickle cell disease and transfusion-dependent beta thalassaemia
8 June 2023 - FDA grants priority review for severe sickle cell disease and standard review for transfusion-dependent beta thalassemia. ...