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RSS FeedPosted by Michael Wonder on 06 Nov 2023
AviadoBio announces FDA IND clearance and fast track designation for AVB-101 for the treatment of frontotemporal dementia with progranulin mutations
6 November 2023 - AVB-101 is an investigational gene therapy delivered as a one time infusion directly to the brain, enabling ...
Posted by Michael Wonder on 31 Oct 2023
US FDA awards both rare paediatric disease and orphan drug designations to HG204, a CRISPR RNA editing therapy, for the treatment of MECP2 duplication syndrome
31 October 2023 - HG204 is the world first CRISPR RNA-editing therapy for the treatment of MECP2 duplication syndrome granted ...
Posted by Michael Wonder on 09 Oct 2023
Alnylam announces receipt of complete response letter from US FDA for supplemental new drug application for patisiran for the treatment of the cardiomyopathy of ATTR amyloidosis
9 October 2023 - FDA cites insufficient evidence of clinical meaningfulness. ...
Posted by Michael Wonder on 03 Oct 2023
FDA approves Rivfloza for children ≥9 years old and adults living with primary hyperoxaluria type 1, a rare genetic condition
2 October 2023 - Novo Nordisk announced today that the US FDA has approved Rivfloza (nedosiran) injection 80 mg, 128 mg, ...
Posted by Michael Wonder on 03 Oct 2023
Rocket Pharmaceuticals announces FDA acceptance of biologics license application with priority review for RP-L201 (marnetegragene autotemcel) for the treatment of severe leukocyte adhesion deficiency-I
2 October 2023 - PDUFA target action date is 31 March 2024. ...
Posted by Michael Wonder on 19 Sep 2023
Orchard Therapeutics announces acceptance of biologics license application for OTL-200 in MLD and receives priority review
18 September 2023 - PDUFA date set for 18 March 2024. ...
Posted by Michael Wonder on 13 Sep 2023
First bladder cancer patient dosed with commercially available intravesical gene therapy Adstiladrin (nadofaragene firadenovec-vncg)
12 September 2023 - Ferring launches ABLE-41 Real World Evidence Study, a patient registry to explore early use, experiences and outcomes ...
Posted by Michael Wonder on 30 Aug 2023
Bloomsbury Genetic Therapies receives rare paediatric disease designation from the US FDA for BGT-INAD for the treatment of infantile neuroaxonal dystrophy
29 August 2023 - Bloomsbury Genetic Therapies Limited announced today that the US FDA has granted rare paediatric disease designation for ...
Posted by Michael Wonder on 24 Aug 2023
Taysha Gene Therapies announces fast track designation granted by US FDA for TSHA-102 in Rett syndrome
24 August 2023 - TSHA-102 has also received orphan drug and rare paediatric disease designations from the US FDA and ...
Posted 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 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 ...