Blog
RSS FeedPosted 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 ...
Posted by Michael Wonder on 03 Jun 2025
Amylyx Pharmaceuticals receives US FDA fast track designation for AMX0114 for the treatment of amyotrophic lateral sclerosis
3 June 2025 - AMX0114 is an Amylyx developed antisense oligonucleotide designed to target calpain-2, a key contributor to the ...
Posted by Michael Wonder on 29 May 2025
Capsida receives FDA fast track designation for its potential first in class IV administered gene therapy for STXBP1 developmental and epileptic encephalopathy
29 May 2025 - Capsida previously received FDA orphan drug designation and IND clearance to initiate the SYNRGY Phase 1/2a ...
Posted by Michael Wonder on 28 May 2025
Candel Therapeutics receives FDA regenerative medicine advanced therapy designation for CAN-2409 for the treatment of prostate cancer
28 May 2025 - Candel Therapeutics today announced that the US FDA has granted regenerative medicine advanced therapy designation to CAN-2409 ...
Posted by Michael Wonder on 28 May 2025
Ocugen announces rare paediatric disease designation granted for OCU410ST—modifier gene therapy for the treatment of Stargardt disease
27 May 2025 - Ocugen today announced that the US FDA has granted rare paediatric disease designation for OCU410ST for the ...
Posted by Michael Wonder on 13 May 2025
RegenxBio announces FDA acceptance and priority review of the BLA for RGX-121 for MPS II
13 May 2025 - FDA assigns PDUFA target action date of 9 November 2025. ...
Posted by Michael Wonder on 10 May 2025
MeiraGTx granted FDA regenerative medicine advanced therapy designation for AAV-GAD for the treatment of Parkinson’s disease
9 May 2025 - This RMAT designation is based on data from 3 clinical studies demonstrating the potential benefit of AAV-GAD ...
Posted by Michael Wonder on 30 Apr 2025
US FDA approves Zevaskyn (prademagene zamikeracel), the first and only cell-based gene therapy for patients with recessive dystrophic epidermolysis bullosa
29 April 2025 - Rare paediatric disease priority review voucher granted by FDA. ...