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RSS FeedPosted by Michael Wonder on 18 Apr 2024
AskBio receives FDA fast track designation for AB-1002 investigational gene therapy program in congestive heart failure
18 April 2024 - AB-1002 is being studied for the treatment of adults with non-ischaemic cardiomyopathy and New York Heart Association ...
Posted by Michael Wonder on 16 Apr 2024
Lexeo Therapeutics granted FDA fast track designation for LX2006, an AAV based gene therapy candidate for the treatment of Friedreich’s ataxia cardiomyopathy
16 April 2024 - Lexeo Therapeutics today announced the US FDA has granted fast track designation to LX2006, the company’s AAVrh.10hFXN ...
Posted by Michael Wonder on 04 Apr 2024
Rocket Pharmaceuticals announces EMA acceptance of RP-L102 marketing authorisation application for the treatment of Fanconi anaemia
2 April 2024 - Positive, previously disclosed results from the global Phase 1/2 trial demonstrated genetic and phenotypic correction combined ...
Posted by Michael Wonder on 03 Apr 2024
Vertex announces new drug submission for exagamglogene autotemcel (exa-cel) has been accepted for priority review by Health Canada for the treatment of sickle cell disease and transfusion-dependent beta thalassaemia
1 April 2024 - Exa-cel is the first CRISPR-based gene-edited therapy to be submitted for Health Canada review. ...
Posted by Michael Wonder on 02 Apr 2024
Solid Biosciences receives rare paediatric disease designation from the FDA for Duchenne muscular dystrophy gene therapy candidate SGT-003
1 April 2024 - Site initiations scheduled for April; patient dosing expected to begin in Q2 ,2024. ...
Posted by Michael Wonder on 21 Mar 2024
Health Canada accepts for review new drug submission for tofersen for treatment of rare, genetic form of ALS
19 March 2024 - Health Canada regulatory decision on tofersen new drug submission expected in early 2025. ...
Posted by Michael Wonder on 20 Mar 2024
A lifesaving therapy for children with a rare disease is now the world’s most expensive drug, raising questions about access
20 March 2024 - A new gene therapy for the fatal genetic disorder metachromatic leukodystrophy, or MLD, will carry a ...
Posted by Michael Wonder on 20 Mar 2024
PTC Therapeutics provides key regulatory updates (March 2024)
19 March 2024 - BLA submitted to FDA for Upstaza for the treatment of AADC deficiency - ...
Posted by Michael Wonder on 18 Mar 2024
FDA approves first gene therapy for children with metachromatic leukodystrophy
18 March 2024 - Today, the US FDA approved Lenmeldy (atidarsagene autotemcel), the first FDA approved gene therapy indicated for the ...
Posted by Michael Wonder on 14 Mar 2024
PepGen receives US FDA orphan drug and rare paediatric disease designations for PGN-EDO51 for the treatment of Duchenne muscular dystrophy
13 March 2024 - PepGen today announced that the US FDA granted both orphan drug and rare paediatric disease designations for ...
Posted by Michael Wonder on 24 Feb 2024
New treatment for rare motor neurone disease recommended for approval
23 February 2024 - The EMA has recommended granting a marketing authorisation in the European Union for a new therapy ...
Posted by Michael Wonder on 20 Feb 2024
Medera’s Sardocor announces fast track designation and dosing of 3 patients in first in human HFpEF gene therapy trial
14 February 2024 - In the Phase 1/2A clinical trial for SRD-001, an adeno-associated virus mediated first in human gene therapy ...
Posted by Michael Wonder on 18 Feb 2024
Sarepta Therapeutics announces US FDA acceptance of an efficacy supplement to expand the Elevidys indication
16 February 2024 - Priority review granted, with a review goal date of 21 June 2024. ...
Posted by Michael Wonder on 14 Feb 2024
Rocket Pharmaceuticals announces update on FDA review timeline of Kresladi (marnetegragene autotemcel) for the treatment of severe leukocyte adhesion deficiency-I
13 February 2024 - New Prescription Drug User Fee Act date of 30 June 2024. ...
Posted by Michael Wonder on 13 Feb 2024
European Commission approves first CRISPR/Cas9 gene-edited therapy, Casgevy (exagamglogene autotemcel), for the treatment of sickle cell disease and transfusion-dependent beta thalassaemia
13 February 2024 - Over 8,000 patients 12 years of age and older with severe sickle cell disease or transfusion-dependent ...