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. ...

Read more →

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 ...

Read more →

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 ...

Read more →

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 ...

Read more →

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 ...

Read more →

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 ...

Read more →

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 ...

Read more →

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 ...

Read more →

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 ...

Read more →

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 ...

Read more →

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. ...

Read more →

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 ...

Read more →

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. ...

Read more →

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 ...

Read more →

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. ...

Read more →