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RSS FeedPosted by Michael Wonder on 10 Sep 2025
Ionis receives US FDA breakthrough therapy designation for ION582 in Angelman syndrome
9 September 2025 - Pivotal Phase 3 REVEAL study enrollment on track to complete in 2026. ...
Posted by Michael Wonder on 21 Aug 2025
VeonGen Therapeutics receives FDA regenerative medicine advanced therapy designation for VG801 gene therapy for Stargardt disease
21 August 2025 - VeonGen Therapeutics today announced that the US FDA has granted regenerative medicine advanced therapy designation to VG801, ...
Posted by Michael Wonder on 19 Aug 2025
Ultragenyx initiates rolling submission of niologics license application to US FDA for DTX401 AAV gene therapy for the Treatment of glycogen storage disease type Ia
18 August 2025 - Ultragenyx today announced the initiation of a rolling submission of a niologics license application to the US ...
Posted by Michael Wonder on 16 Aug 2025
FDA approves first immunotherapy for recurrent respiratory papillomatosis
14 August 2025 - Today, the US FDA approved Papzimeos (zopapogene imadenovec-drba), a first of its kind non-replicating adenoviral vector-based immunotherapy ...
Posted by Michael Wonder on 13 Aug 2025
AAVantgarde Bio announces FDA fast track designation for AAVB-039 for the treatment of Stargardt disease
12 August 2025 - AAVantgarde Bio today announced that the US FDA has granted fast track designation for AAVB-039, the company’s ...
Posted by Michael Wonder on 29 Jul 2025
Tofersen approved by the MHRA to treat rare inherited form of motor neurone disease
28 July 2025 - New genetic therapy approved for SOD1-ALS brings targeted treatment option to patients in the UK. ...
Posted by Michael Wonder on 26 Jul 2025
Roche provides regulatory update on Elevidys gene therapy for Duchenne muscular dystrophy in the EU
25 July 2025 - Roche will continue its dialogue with the EMA to explore a potential path forward to make Elevidys ...
Posted by Michael Wonder on 24 Jul 2025
Avidity Biosciences receives FDA breakthrough therapy designation for delpacibart zotadirsen for the treatment of DMD in people with mutations amenable to exon 44 skipping
23 July 2025 - On track for planned BLA submission for delpacibart zotadirsen at year end 2025. ...
Posted by Michael Wonder on 22 Jul 2025
Replimune receives complete response letter from FDA for RP1 biologics license application for the treatment of advanced melanoma
23 July 2025 - Replimune Group today announced that the US FDA has issued a complete response letter regarding the ...
Posted by Michael Wonder on 20 Jul 2025
Rocket Pharmaceuticals receives FDA regenerative medicine advanced therapy designation for RP-A601 gene therapy for PKP2 arhythmogenic cardiomyopathy
17 July 2025 - -- Rocket Pharmaceuticals today announced that the US FDA has granted regenerative medicine advanced therapy designation to ...
Posted by Michael Wonder on 20 Jul 2025
Genascence announces US FDA grants regenerative medicine advanced therapy designation to GNSC-001 for knee osteoarthritis
16 July 2025 - Genascence Corporation today announced that the US FDA has granted the regenerative medicine advanced therapy designation ...
Posted by Michael Wonder on 16 Jul 2025
Nanoscope Therapeutics initiates rolling submission of biologics license application to FDA for MCO-010, the first gene-agnostic therapy to treat retinitis pigmentosa
14 July 2025 - First modules of biologics license application submitted to FDA under rolling review, with full submission anticipated ...
Posted by Michael Wonder on 13 Jul 2025
Ultragenyx receives complete response letter from FDA for UX111 AAV gene therapy to treat Sanfilippo syndrome type A
11 July 2025 - complete response letter cited specific chemistry, manufacturing and controls related observations that are resolvable. ...
Posted by Michael Wonder on 05 Jul 2025
Vitalgen's gene therapy drug VGN-R09b for primary Parkinson's disease receives FDA fast track designation
26 June 2025 - Shanghai Vitalgen announced that its gene therapy drug, VGN-R09b, for the treatment of primary Parkinson’s disease, has ...
Posted by Michael Wonder on 26 Jun 2025
Precision BioSciences receives FDA rare paediatric disease designation for PBGENE-DMD for the treatment of Duchenne muscular dystrophy
25 June 2025 - Precision BioSciences today announced that the US FDA has granted rare paediatric disease designation for PBGENE-DMD for ...