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RSS FeedPosted by Michael Wonder on 16 May 2023
FDA grants SiSaf’s innovative siRNA therapy SIS-101-ADO orphan drug designation and rare paediatric disease designation for the treatment of autosomal dominant osteopetrosis
15 May 2023 - SiSaf announces that SIS-101-ADO, its siRNA therapeutic, has been granted orphan drug designation and rare paediatric disease ...
Posted by Michael Wonder on 04 May 2023
Gene therapies are still hampered by substantial delays between approval and launch
3 May 2023 - Gene therapies hold promise for treating a wide range of diseases. Innovative companies such as bluebird bio ...
Posted by Michael Wonder on 25 Apr 2023
FDA grants accelerated approval for Qalsody (tofersen) for SOD1-ALS, a major scientific advancement as the first treatment to target a genetic cause of ALS
25 April 2023 - FDA granted accelerated approval of Qalsody based on a reduction of neurofilament, a marker of neurodegeneration. ...
Posted by Michael Wonder on 25 Apr 2023
bluebird bio submits biologics license application to FDA for lovotibeglogene autotemcel (lovo-cel) for patients with sickle cell disease 12 years and older with a history of vaso-occlusive events
24 April 2023 - BLA submission based on data from the largest and most mature clinical development program for any ...
Posted by Michael Wonder on 21 Apr 2023
GenSight Biologics withdraws its EMA application for Lumevoq
20 April 2023 - GenSight Biologics today announces that the Committee for Advanced Therapies (CAT) of the CHMP of the EMA ...
Posted by Michael Wonder on 19 Apr 2023
Onasemnogene abeparvovec for the treatment of patients with presymptomatic spinal muscular atrophy
19 April 2023 - NICE has published Evidence-based recommendations on onasemnogene abeparvovec (Zolgensma) for the treatment of babies aged 12 months ...
Posted by Michael Wonder on 19 Apr 2023
Eladocagene exuparvovec for the treatment of patients with aromatic L-amino acid decarboxylase deficiency
19 April 2023 - NICE has published evidence based recommendations on eladocagene exuparvovec (Upstaza) for the treatment of patients 18 ...
Posted by Michael Wonder on 12 Apr 2023
ICER releases draft evidence report on gene therapies for sickle cell disease
12 April 2023 - Public comment period now open until 9 May 2023; Requests to make oral comment during public ...
Posted by Michael Wonder on 11 Apr 2023
RegenxBio receives FDA fast track designation for RGX-202, a novel gene therapy candidate for the treatment of Duchenne muscular dystrophy
11 April 2023 - RGX-202 is a potential one-time AAV therapeutic for the treatment of Duchenne and includes an optimised transgene ...
Posted by Michael Wonder on 03 Apr 2023
Vertex and CRISPR Therapeutics complete submission of rolling biologics license applications to the US FDA for exa-cel for the treatment of sickle cell disease and transfusion-dependent beta thalassaemia
3 April 2023 - EU and UK filings completed in 2022; submissions validated by EMA and MHRA and the review ...
Posted by Michael Wonder on 25 Mar 2023
NICE recommends life-changing gene therapy for children with ultra rare genetic disorder in final draft guidance
23 March 2023 - The first and currently only gene therapy for children with an ultra-rare genetic disorder has been recommended ...
Posted by Michael Wonder on 21 Mar 2023
Top FDA official: agency needs to start using accelerated approval for gene therapies
20 March 2023 - A top FDA official said Monday that the agency needs to start using accelerated approval, a ...
Posted by Michael Wonder on 10 Mar 2023
ICER to assess gene therapy for metachromatic leukodystrophy
9 March 2023 - Report will be subject of CTAF meeting in September 2023; draft scoping document open to public ...
Posted by Michael Wonder on 09 Mar 2023
Use warranties, not outcomes-based agreements, to make payers whole when a gene therapy fails
9 March 2023 - A decade ago, I had pegged gene and cell therapies as the next frontier. As an ...
Posted by Michael Wonder on 08 Mar 2023
Genethon given PRIME status by EMA for gene therapy to treat Crigler-Najjar syndrome, a rare liver disease
7 March 2023 - Genethon today announced that the EMA has granted PRIME (Priority Medicines) status to the gene therapy, ...