Blog
RSS FeedPosted by Michael Wonder on 04 Mar 2025
Qalsody (tofersen injection) receives conditional marketing authorisation from Health Canada as the first ALS treatment targeting a genetic cause
3 March 2025 - Biogen's second rare disease therapy to be approved in Canada reflects a continued commitment to advancing ...
Posted by Michael Wonder on 02 Mar 2025
Krystal Biotech receives positive CHMP opinion for Vyjuvek for the treatment of dystrophic epidermolysis bullosa
28 February 2025 - EC approval decision anticipated in second quarter of 2025. ...
Posted by Michael Wonder on 26 Feb 2025
Exagamglogene autotemcel for the treatment of patients with severe sickle cell disease
26 February 2025 - NICE has published final evidence-based recommendations on the use of exagamglogene autotemcel (Casgevy) for the treatment ...
Posted by Michael Wonder on 25 Feb 2025
FDA grants priority review to Precigen's BLA for PRGN-2012 for the treatment of adults with recurrent respiratory papillomatosis with PDUFA target action date set for 27 August 2025
25 February 2025 - If approved, PRGN-2012 would be the first and only available FDA approved therapy for eligible patients ...
Posted by Michael Wonder on 20 Feb 2025
AskBio receives FDA regenerative medicine advanced therapy designation for Parkinson’s disease investigational gene therapy
19 February 2025 - Regenerative medicine advanced therapy designation follows Phase Ib 36 month data. ...
Posted by Michael Wonder on 10 Feb 2025
ICER releases draft evidence report on treatment for advanced retinitis pigmentosa
6 February 2025 - Public comment period now open until 6 March 2025; requests to make oral comment during public ...
Posted by Michael Wonder on 04 Feb 2025
Fondazione Telethon submits EU marketing authorisation application for etuvetidigene autotemcel gene therapy for the treatment of Wiskott-Aldrich syndrome
3 February 2025 - Fondazione Telethon announced that it has submitted the marketing authorisation application for the gene therapy - etuvetidigene ...
Posted by Michael Wonder on 04 Feb 2025
Comparison of clinical evidence submitted to the FDA and EMA for cell and gene therapies
3 February 2025 - Harmonisation in regulatory submissions across agencies may support timelier access to innovative treatments, including cell and gene ...
Posted by Michael Wonder on 02 Feb 2025
Cell and gene therapies - improving access and outcomes for Medicare and Medicaid beneficiaries
1 February 2025 - State Medicaid agencies can now apply to participate in a new model that supports voluntary, outcomes-based agreements ...
Posted by Michael Wonder on 01 Feb 2025
Groundbreaking one-off gene therapy approved for severe sickle cell disease
31 January 2025 - People in England with severe sickle cell disease will be among the first to receive treatment using ...
Posted by Michael Wonder on 31 Jan 2025
Vertex announces Casgevy reimbursement agreement for the treatment of sickle cell disease in England
31 January 2025 - Agreement means eligible sickle cell disease patients in England now have access to Casgevy. ...
Posted by Michael Wonder on 28 Jan 2025
Beacon Therapeutics granted FDA regenerative medicine advanced therapy designation for laru-zova (AGTC-501)
28 January 2025 - Recognises preliminary clinical evidence from the Phase 2 DAWN and SKYLINE trials of laru-zova in X-linked retinitis ...
Posted by Michael Wonder on 23 Jan 2025
FDA and EMA accept applications for higher dose regimen of nusinersen in spinal muscular atrophy
23 January 2025 - Applications are based on data from the DEVOTE study, which demonstrate the potential for the investigational higher ...
Posted by Michael Wonder on 22 Jan 2025
Replimune announces biologics license application acceptance and priority review for RP1 for the treatment of advanced melanoma
21 January 2025 - PDUFA action date of 22 July 2025, with priority review. ...
Posted by Michael Wonder on 21 Jan 2025
Dyne Therapeutics receives FDA fast track designation for DYNE-101 for the treatment of myotonic dystrophy type 1
21 January 2025 - Dyne Therapeutics today announced that the US FDA has granted fast track designation for DYNE-101 for ...