Blog
RSS FeedPosted 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. ...
Posted by Michael Wonder on 12 Sep 2025
Sanofi’s SAR402663 earns fast track designation in the US for neovascular age-related macular degeneration
11 September 2025 - Designation earned for a one time intravitreal gene therapy with the potential to eliminate treatment burden for ...
Posted 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 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 ...