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RSS FeedPosted by Michael Wonder on 17 Apr 2025
uniQure announces FDA breakthrough therapy designation granted to AMT-130 for the treatment of Huntington’s disease
17 April 2025 - Breakthrough therapy designation based on clinical evidence from Phase I/II trials showing meaningful slowing of disease progression. ...
Posted by Michael Wonder on 15 Apr 2025
Precision BioSciences receives US FDA fast track designation for PBGENE-HBV, a first in class gene editing therapy designed to eliminate the root cause of chronic hepatitis B
15 April 2025 - Precision BioSciences today announced that the US FDA has granted fast track designation for PBGENE-HBV, the Company’s ...
Posted by Michael Wonder on 11 Apr 2025
Verve Therapeutics receives US FDA fast track designation for VERVE-102, an in vivo base editing medicine targeting PCSK9
11 April 2025 - Verve Therapeutics today announced that the US FDA has granted fast track designation for VERVE-102 for ...
Posted by Michael Wonder on 21 Mar 2025
Alnylam announces FDA approval of Amvuttra (vutrisiran), the first RNAi therapeutic to reduce cardiovascular death, hospitalisations and urgent heart failure visits in adults with ATTR amyloidosis with cardiomyopathy
20 March 2025 - Only therapeutic approved in the US to address both cardiomyopathy and polyneuropathy manifestations of ATTR amyloidosis. ...
Posted by Michael Wonder on 12 Mar 2025
Atsena Therapeutics granted US FDA fast track designation for ATSN-201 gene therapy to treat X-linked retinoschisis
12 March 2025 - Marks third FDA designation for ATSN-201, which has also received rare paediatric disease designation and orphan drug ...
Posted by Michael Wonder on 07 Mar 2025
Neurotech’s Encelto (revakinagene taroretcel-lwey) approved by the FDA for the treatment of macular telangiectasia type 2
6 March 2025 - Encelto is the first and only FDA approved treatment for macular telangiectasia type 2. ...
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 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. ...