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RSS FeedPosted 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 19 Nov 2020
Alnylam receives approval for Oxlumo (lumasiran) in the European Union for the treatment of primary hyperoxaluria type 1 in all age groups
19 November 2020 - Oxlumo is the first therapeutic approved for the treatment of primary hyperoxaluria type 1, and the ...
Posted by Michael Wonder on 03 Nov 2020
GenSight Biologics reports validation of Lumevoq marketing authorisation application by European Medicines Agency
3 November 2020 - GenSight Biologics today reported that the Lumevoq marketing authorisation application passed the validation checks required for submissions ...
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 18 Oct 2020
Highlights from 12-15 October CHMP meeting
16 October 2020 - Ten new medicines recommended for approval. ...
Posted by Michael Wonder on 18 Oct 2020
Novartis receives positive CHMP opinion for Leqvio (inclisiran), a potential first-in-class siRNA for the treatment of high cholesterol
16 October 2020 - If approved, inclisiran will be the first and only small interfering RNA (siRNA) in Europe for patients ...
Posted by Michael Wonder on 18 Oct 2020
First treatment for rare condition primary hyperoxaluria type 1
16 October 2020 - EMA has recommended granting a marketing authorisation in the European Union for Oxlumo (lumasiran) for the ...
Posted by Michael Wonder on 15 Oct 2020
Health Canada approves first-ever gene replacement therapy, Luxturna
15 October 2020 - Luxturna (voretigene neparvovec), is the first approved therapy for previously untreatable inherited retinal disease. ...
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
bluebird bio announces European Medicines Agency’s acceptance of marketing authorisation application for elivaldogene autotemcel (eli-cel, Lenti-D) gene therapy for cerebral adrenoleukodystrophy
2 October 2020 - European Medicines Agency will evaluate eli-cel application under accelerated assessment. ...
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
bluebird bio’s LentiGlobin for sickle cell disease gene therapy (bb1111) granted priority medicines (PRIME) designation by European Medicines Agency
23 September 2020 - EMA’s PRIME program designed to optimise development and expedite evaluation of innovative medicines for patients with high ...
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 ...