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RSS FeedPosted by Michael Wonder on 12 Sep 2024
Ilya Pharma receives rare paediatric disease designation from the US FDA for emilimogene sigulactibac for the treatment of skin wounds in SAVI patients
11 September 2024 - Ilya Pharma today announced that it has received rare paediatric disease designation by the US FDA. ...
Posted by Michael Wonder on 11 Sep 2024
Exagamglogene autotemcel for the treatment of patients 12 years of age and older with transfusion-dependent beta-thalassaemia
11 September 2024 - NICE has published final evidence-based recommendations on the use of exagamglogene autotemcel (Casgevy) for the treatment ...
Posted by Michael Wonder on 10 Sep 2024
BridgeBio receives FDA’s regenerative medicine advanced therapy designation for BBP-812 Canavan disease gene therapy program
10 September 2024 - Receipt of RMAT designation is based on preliminary clinical evidence from the CANaspire Phase 1/2 clinical trial, ...
Posted by Michael Wonder on 08 Sep 2024
YolTech Therapeutics receives US FDA rare paediatric disease designation for YOLT-203 in treating primary hyperoxaluria type 1
4 September 2024 - YolTech Therapeutics today announced that the US FDA has granted rare paediatric disease designation to YOLT-203 for ...
Posted by Michael Wonder on 22 Aug 2024
Opus Genetics receives rare paediatric disease designation from the US FDA for ocular gene therapy OPGx-LCA5 to treat rare inherited retinal disease LCA5
20 August 2024 - Opus Genetics today announced the US FDA has granted rare paediatric disease designation for its ocular gene ...
Posted by Michael Wonder on 13 Aug 2024
Wave Life Sciences receives FDA rare paediatric disease designation for WVE-N531 for the treatment of Duchenne muscular dystrophy
12 August 2024 - Wave Life Sciences today announced that the US FDA has granted rare paediatric disease designation to WVE-N531 ...
Posted by Michael Wonder on 09 Aug 2024
BioMarin announces updated strategy for Roctavian to focus on US, Germany and Italy
5 August 2024 - Strategic focus will reduce operating expenses with the goal of achieving Roctavian profitability by end of 2025. ...
Posted by Michael Wonder on 08 Aug 2024
World’s first gene editing therapy for blood disorder to be available to hundreds of patients in England
8 August 2024 - Patients in England with severe beta-thalassaemia will be amongst the first in Europe to benefit from one-time ...
Posted by Michael Wonder on 08 Aug 2024
Vertex announces Casgevy reimbursement agreement for the treatment of transfusion-dependent beta thalassemia in England
7 August 2024 - Eligible transfusion-dependent beta thalassaemia patients in England will be able to access the therapy from today. ...
Posted by Michael Wonder on 08 Aug 2024
Neurogene announces RMAT designation for NGN-401 investigational gene therapy for Rett syndrome
7 August 2024 - Designation based on preliminary clinical evidence from ongoing NGN-401 clinical trial that shows potential to address unmet ...
Posted by Michael Wonder on 06 Aug 2024
PBS listing for Australians with hereditary transthyretin-mediated amyloidosis with polyneuropathy
1 August 2024 - Medison Pharma, alongside their partner Alnylam Pharmaceuticals, are pleased to announce that from 1 August 2024, ...
Posted by Michael Wonder on 04 Aug 2024
Genomic medicines can cost $3 million a dose. How to make them affordable.
1 August 2024 - The treatments are marvels of innovation. Their pricing must be inventive, too. ...
Posted by Michael Wonder on 02 Aug 2024
FDA approves first gene therapy to treat adults with metastatic synovial sarcoma
2 August 2024 - The US FDA approved Tecelra (afamitresgene autoleucel), a gene therapy indicated for the treatment of adults with ...
Posted by Michael Wonder on 02 Aug 2024
Adverum Biotechnologies announces FDA regenerative medicine advanced therapy designation granted for ixo-vec for the treatment of wet AMD
1 August 2024 - Adverum Biotechnologies today announced that the US FDA has granted regenerative medicine advanced therapy designation for ...
Posted by Michael Wonder on 26 Jul 2024
European Commission approves Pfizer’s Durveqtix (fidanacogene elaparvovec), a one-time gene therapy for adults with haemophilia B
25 July 2024 - A one-time dose of Durveqtix has reduced bleeds post-treatment compared to standard of care with a median ...