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RSS FeedPosted by Michael Wonder on 08 Mar 2021
U.S. FDA grants fast track designation to three Passage Bio gene therapy candidates targeting rare CNS disorders
8 March 2021 - FDA designation will facilitate development and expedited review of company’s lead gene therapy product candidates for ...
Posted by Michael Wonder on 08 Mar 2021
BioMarin announces FDA regenerative medicine advanced therapy designation granted to valoctocogene roxaparvovec, investigational gene therapy for haemophilia A
8 March 2021 - RMAT designation granted by FDA during bleeding disorders awareness month. ...
Posted by Michael Wonder on 25 Feb 2021
FDA approves targeted treatment for rare Duchenne muscular dystrophy mutation
25 February 2021 - Today, the U.S. FDA granted approval for Amondys 45 (casimersen) injection for the treatment of Duchenne muscular ...
Posted by Michael Wonder on 26 Jan 2021
MeiraGTx announces AAV-CNGA3 granted fast track designation by U.S. FDA for treatment of achromatopsia
26 January 2021 - MeiraGTx today announced that the U.S. FDA has granted fast track designation to its AAV-CNGA3 gene therapy ...
Posted by Michael Wonder on 19 Jan 2021
Taysha Gene Therapies receives rare paediatric disease and orphan drug designations for TSHA-105 for the treatment of epilepsy caused by SLC13A5 deficiency
19 January 2021 - Designations reinforce unmet need for treatment options for patients with rare form of genetic epilepsy. ...
Posted by Michael Wonder on 21 Dec 2020
Sesen Bio submits completed biologics license application to the FDA for Vicineum and has requested priority review
21 December 2020 - BLA supported by strong Phase 3 VISTA trial data and positive analytical comparability data. ...
Posted by Michael Wonder on 18 Dec 2020
Novartis receives complete response letter from U.S. FDA for inclisiran
18 December 2020 - The U.S. FDA has not raised any concerns related to the efficacy or safety of inclisiran. The ...
Posted by Michael Wonder on 04 Dec 2020
enGene receives fast track designation for EG-70 for the treatment of non-muscle invasive bladder cancer
3 December 2020 - enGene announced today that the U.S. FDA has granted fast track designation to enGene for EG-70, the ...
Posted by Michael Wonder on 28 Oct 2020
Taysha Gene Therapies receives rare paediatric disease designation and orphan drug designation for TSHA-104 to treat SURF1 associated Leigh syndrome
27 October 2020 - Taysha anticipated to submit Investigational new drug application for TSHA-104 to FDA in 2021. ...
Posted by Michael Wonder on 15 Oct 2020
Taysha Gene Therapies receives rare paediatric disease designation and orphan drug designation for TSHA-102 as a treatment for Rett syndrome
14 October 2020 - Program leverages novel miRARE platform technology used to control transgene expression on a cellular basis. ...
Posted by Michael Wonder on 11 Oct 2020
Axovant Gene Therapies receives rare paediatric disease designation for AXO-AAV-GM1 for GM1 gangliosidosis
9 October 2020 - Company on-track to report data from on-going Phase 1/2 clinical study in Q4 2020. ...
Posted by Michael Wonder on 04 Oct 2020
BioMarin receives FDA fast track designation for investigational gene therapy, BMN 307
2 October 2020 - Second investigational gene therapy in clinic, potential third therapy in PKU franchise. ...
Posted by Michael Wonder on 02 Oct 2020
Pfizer receives FDA fast track designation for Duchenne muscular dystrophy investigational gene therapy
1 October 2020 - Pfizer today announced that its investigational gene therapy candidate (PF-06939926) being developed to treat Duchenne muscular dystrophy ...
Posted by Michael Wonder on 24 Sep 2020
FDA grants Spirovant Sciences orphan drug and rare paediatric disease designations for SPIRO-2101 for treatment of cystic fibrosis
24 September 2020 - Spirovant Sciences today announced that the U.S. FDA has granted orphan drug and rare paediatric disease ...
Posted by Michael Wonder on 22 Sep 2020
Gyroscope Therapeutics granted FDA fast track designation for GT005, an investigational gene therapy for dry age-related macular degeneration
22 September 2020 - Gyroscope Therapeutics today announced that the U.S. FDA has granted fast track designation to GT005 for the ...