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RSS FeedPosted by Michael Wonder on 02 Apr 2026
Ultragenyx announces US FDA acceptance of BLA resubmission for UX111 AAV gene therapy to treat Sanfilippo syndrome type A
2 April 2026 - Ultragenyx Pharmaceutical today announced the US FDA has accepted for review the resubmitted biologics license application seeking ...
Posted by Michael Wonder on 31 Mar 2026
FDA approves new high dose regimen of Spinraza (nusinersen) for spinal muscular atrophy
30 March 2026 - The approval was anchored on data from the pivotal DEVOTE study that investigated the efficacy and safety ...
Posted by Michael Wonder on 27 Mar 2026
MeiraGTx announces FDA breakthrough therapy designation for AAV2-hAQP1 for the treatment of grade 2 and grade 3 radiation-induced xerostomia
26 March 2026 - FDA granted breakthrough therapy designation for AAV2-hAQP1 for the treatment of Grade 2 and Grade 3 late ...
Posted by Michael Wonder on 27 Mar 2026
FDA approves first gene therapy for severe leukocyte adhesion deficiency type I
26 March 2026 - The US FDA today approved Kresladi (marnetegragene autotemcel), the first gene therapy for the treatment of severe ...
Posted by Michael Wonder on 23 Mar 2026
Ionis announces zilganersen new drug application for Alexander disease accepted by FDA for priority review
23 March 2026 - Ionis Pharmaceuticals today announced that the US FDA has accepted for priority review the new drug application ...
Posted by Michael Wonder on 10 Mar 2026
Sangamo Therapeutics advances rolling submission of BLA to US FDA for ST-920 in Fabry disease
9 March 2026 - Sangamo Therapeutics today announced advancement of the rolling submission of a BLA to the FDA seeking ...
Posted by Michael Wonder on 09 Mar 2026
Precision BioSciences receives FDA fast track sesignation for PBGENE-DMD
9 March 2026 - Precision BioSciences today announced that the US FDA has granted fast track designation to PBGENE-DMD for the ...
Posted by Michael Wonder on 24 Feb 2026
Ultragenyx announces US FDA acceptance and priority review of the biologics license application for DTX401 AAV gene therapy for glycogen storage disease type Ia
23 February 2026 - Ultragenyx today announced the US FDA has accepted for review the biologics license application seeking approval of ...
Posted by Michael Wonder on 23 Feb 2026
Tessera Therapeutics receives US FDA fast track and orphan drug designations for its lead in vivo gene editing program TSRA-196 for the treatment of adults with AATD
23 February 2026 - Tessera Therapeutics today announced that the US FDA has granted fast track and orphan drug designations to ...
Posted by Michael Wonder on 10 Feb 2026
RegenxBio announces regulatory update on RGX-121 BLA for MPS II
9 February 2026 - RegenXBio today announced that the US FDA has issued a complete response letter regarding its biologics ...
Posted by Michael Wonder on 21 Jan 2026
iECURE receives FDA regenerative medicine advanced therapy designation for ECUR-506 for neonatal onset ornithine transcarbamylase deficiency
7 January 2026 - iECURE today announced that the US FDA has granted regenerative medicine advanced therapy designation to ECUR-506, ...
Posted by Michael Wonder on 17 Jan 2026
Fondazione Telethon receives european marketing authorisation for Waskyra (etuvetidigene autotemcel), a gene therapy for the treatment of Wiskott-Aldrich syndrome
16 January 2026 - The European Commission’s decision follows the positive opinion issued by the EMA’s CHMP in November 2025. ...
Posted by Michael Wonder on 10 Dec 2025
FDA approves first gene therapy treatment for Wiskott-Aldrich syndrome
9 December 2025 - Agency exercises regulatory flexibility to address unmet need for rare, life-threatening disease. ...
Posted by Michael Wonder on 02 Dec 2025
Solid Biosciences receives FDA rare paediatric disease designation for SGT-212 dual route of administration gene therapy for Friedreich’s ataxia
1 December 2025 - Solid Biosciences today announced that it received rare paediatric disease designation from the US FDA for SGT-212, ...
Posted by Michael Wonder on 24 Nov 2025
Novartis receives FDA approval for Itvisma, the only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy
24 November 2024 - Novartis today announced that the US FDA has approved Itvisma (onasemnogene abeparvovec-brve) for the treatment of children ...