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RSS FeedPosted 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 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 ...
Posted by Michael Wonder on 21 Nov 2025
Sangamo Therapeutics announces FDA acceptance of BLA rolling submission request for ST-920 in Fabry disease
21 November 2025 - Sangamo Therapeutics today announced that the US FDA has accepted Sangamo’s request for a rolling submission and ...
Posted by Michael Wonder on 15 Nov 2025
FDA’s new plausible mechanism pathway
12 November 2025 - Personalised therapies hold tremendous promise but challenge traditional models of drug and biologic development. ...
Posted by Michael Wonder on 15 Nov 2025
Sarepta announces FDA’s approval of updated Elevidys Prescribing Information
14 November 2025 - Sarepta Therapeutics today announced an update to the prescribing information for Elevidys (delandistrogene moxeparvovec-rokl), the only ...
Posted by Michael Wonder on 16 Oct 2025
Rocket Pharmaceuticals announces FDA acceptance of BLA resubmission of Kresladi for the treatment of severe leukocyte adhesion deficiency-I
14 October 2025 - PDUFA target action date is 28 March 2026. ...
Posted by Michael Wonder on 07 Oct 2025
EG 427 receives US FDA fast track designation for EG110A DNA medicine in neurogenic bladder patients
6 October 2025 - EG 427 announced today that the US FDA granted fast track designation to EG110A, its novel DNA ...
Posted by Michael Wonder on 03 Oct 2025
Taysha Gene Therapies announces FDA breakthrough therapy designation and provides positive regulatory update on TSHA-102 in Rett syndrome
2 October 2025 - Breakthrough therapy designation granted based on FDA’s review of available clinical evidence of safety and efficacy from ...
Posted by Michael Wonder on 24 Sep 2025
Biogen provides regulatory update on high dose regimen of nusinersen
23 September 2023 - Biogen today announced that the US FDA issued a complete response letter for the Company’s supplemental ...
Posted by Michael Wonder on 23 Sep 2025
Sanofi’s SAR446268 earns US fast track designation for the treatment of non-congenital myotonic dystrophy type 1
23 September 2025 - Designation earned for one time AAV gene therapy SAR446268, designed to silence DMPK expression. ...