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RSS FeedPosted 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 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 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 ...
Posted by Michael Wonder on 21 Jan 2025
Solid Biosciences receives FDA fast track designation for SGT-212 dual route of administration gene therapy for Friedreich’s ataxia
21 January 2025 - Only dual route gene transfer therapy in development to treat Friedreich’s ataxia with FDA IND clearance and ...
Posted by Michael Wonder on 18 Jan 2025
Arrowhead Pharmaceuticals announces acceptance of new drug application by US FDA of plozasiran for the treatment of familial chylomicronaemia syndrome
17 January 2025 - The new drug application is based on positive results from the Phase 3 PALISADE study. ...
Posted by Michael Wonder on 14 Jan 2025
Adaptimmune announces US FDA breakthrough therapy designation granted to letetresgene autoleucel for treatment of myxoid/round cell liposarcoma
13 January 2025 - Adaptimmune Therapeutics today announced that letetresgene autoleucel has been granted breakthrough therapy designation by the US ...
Posted by Michael Wonder on 10 Jan 2025
ViGeneron announces FDA rare paediatric disease designation for VG901
8 January 2025 - ViGeneron today announced two important milestones for its novel gene therapy candidate VG901, to treat patients ...
Posted by Michael Wonder on 21 Dec 2024
Ultragenyx submits biologics license application to the US FDA for UX111 AAV gene therapy for the treatment of Sanfilippo syndrome type A
19 December 2024 - If approved, UX111 would be the first approved therapy in the US for Sanfilippo syndrome type ...
Posted by Michael Wonder on 13 Dec 2024
Stoke Therapeutics receives FDA breakthrough therapy designation for zorevunersen for the treatment of Dravet syndrome
4 December 2024 - Supported by evidence from clinical studies that indicate that zorevunersen may demonstrate substantial improvement over available therapies. ...
Posted by Michael Wonder on 10 Dec 2024
MeiraGTx granted FDA regenerative medicine advanced therapy designation for AAV2-hAQP1 for the treatment of grade 2/3 radiation-induced xerostomia
9 December 2024 - MeiraGTx today announced that the US FDA has granted regenerative medicine advanced therapy designation to AAV2-hAQP1 for ...
Posted by Michael Wonder on 03 Dec 2024
Intellia Therapeutics announces FDA regenerative medicine advanced therapy designation granted to nexiguran ziclumeran for the treatment of hereditary transthyretin amyloidosis with polyneuropathy
25 November 2024 - Intellia Therapeutics today announced that the US FDA has granted regenerative medicine advanced therapy designation to nexiguran ...
Posted by Michael Wonder on 29 Nov 2024
Replimune receives breakthrough therapy designation for RP1 and submits RP1 biologics license application to the FDA under the accelerated approval pathway
21 November 2024 - Replimune today announced that it has submitted a biologics license application to the FDA for RP1 (vusolimogene ...